Scholars@Duke
Joanne Kurtzberg

Joanne Kurtzberg

Jerome S. Harris Distinguished Professor of Pediatrics
Pediatrics, Transplant and Cellular Therapy
joanne.kurtzberg@duke.edu
Box 102501, Durham, NC 27710
2400 Pratt Street, DUMC Box 102501, Durham, NC 27710

Selected Publications

Safety and feasibility of umbilical cord blood transplantation in children with neuronal ceroid lipofuscinosis: a retrospective study.

Journal Article Stem Cells Transl Med · October 29, 2024 Ceroid lipofuscinosis neuronal (CLN) encompasses rare inherited neurodegenerative disorders that present in childhood with clinical features including epilepsy, psychomotor delay, progressive vision loss, and premature death. Published experience utilizing ... Full text Link to item Cite

Mitigation of supply chain challenges in cell therapy manufacturing: perspectives from the cord blood alliance.

Journal Article Stem Cells Transl Med · September 10, 2024 Cellular therapies rely on highly specialized supply chains that often depend on single source providers. Public cord blood banks (CBB) manufacturing the first cell therapy to be highly regulated by the FDA and related international agencies are a prime ex ... Full text Link to item Cite

Impact of FDA's HCT/P ZIKV Recommendations on Cord Blood Unit Eligibility and Utilization in a Large Public Cord Blood Bank.

Journal Article Stem Cells Transl Med · May 14, 2024 BACKGROUND: Cord blood units (CBUs) that are ineligible for licensure due to incomplete compliance with FDA recommendations may be used for hematopoietic stem cell transplantation under urgent medical need and an Investigational Drug Application. The large ... Full text Link to item Cite

A Randomized, Placebo-Controlled, Phase II Trial of Intravenous Allogeneic Non-HLA Matched, Unrelated Donor, Cord Blood Infusion for Ischemic Stroke.

Journal Article Stem Cells Transl Med · February 14, 2024 Stroke remains a leading cause of death and disability in the US, and time-limited reperfusion strategies remain the only approved treatment options. To address this unmet clinical need, we conducted a phase II randomized clinical trial to determine whethe ... Full text Open Access Link to item Cite

Mural cells interact with macrophages in the dura mater to regulate CNS immune surveillance.

Journal Article J Exp Med · February 5, 2024 The central nervous system (CNS) tightly regulates access of circulating immune cells. Immunosurveillance is therefore managed in the meninges at the borders of the CNS. Here, we demonstrated that mural cells, which include pericytes and smooth muscle cell ... Full text Link to item Cite

Mesenchymal stromal cells suppress microglial activation and tumor necrosis factor production.

Journal Article Cytotherapy · February 2024 BACKGROUND AIMS: White matter diseases are commonly associated with microglial activation and neuroinflammation. Mesenchymal stromal cells (MSCs) have immunomodulatory properties and thus have the potential to be developed as cell therapy for white matter ... Full text Link to item Cite

Newborn Screening for Krabbe Disease: Status Quo and Recommendations for Improvements.

Journal Article Int J Neonatal Screen · January 28, 2024 Krabbe disease (KD) is part of newborn screening (NBS) in 11 states with at least one additional state preparing to screen. In July 2021, KD was re-nominated for addition to the federal Recommended Uniform Screening Panel (RUSP) in the USA with a two-tiere ... Full text Link to item Cite

Cell-based versus corticosteroid injections for knee pain in osteoarthritis: a randomized phase 3 trial.

Journal Article Nat Med · December 2023 Various types of cellular injection have become a popular and costly treatment option for patients with knee osteoarthritis despite a paucity of literature establishing relative efficacy to each other or corticosteroid injections. Here we aimed to identify ... Full text Link to item Cite

A Pilot Phase I Trial of Allogeneic Umbilical Cord Tissue-Derived Mesenchymal Stromal Cells in Neonates With Hypoxic-Ischemic Encephalopathy.

Journal Article Stem Cells Transl Med · June 15, 2023 Hypoxic ischemic encephalopathy (HIE) in neonates causes increased mortality and long-term morbidity in surviving babies. Hypothermia (HT) has improved outcomes, however, mortality remains high with ~half of surviving babies developing neurological impairm ... Full text Link to item Cite

Feasibility Study of Cord Tissue Derived Mesenchymal Stromal Cells in COVID-19-Related Acute Respiratory Distress Syndrome.

Journal Article Stem Cells Transl Med · April 17, 2023 BACKGROUND: Treatment options for patients with COVID-19-related acute respiratory distress syndrome (ARDS) are desperately needed. Allogeneic human umbilical cord derived mesenchymal stromal cells (hCT-MSCs) have potential therapeutic benefits in these cr ... Full text Link to item Cite

Genetic and phenotypic spectrum in the NONO-associated syndromic disorder.

Journal Article Am J Med Genet A · February 2023 The non-POU domain-containing octamer-binding (NONO) protein is involved in multiple steps of gene regulation such as RNA metabolism and DNA repair. Hemizygous pathogenic variants in the NONO gene were confirmed to cause a rare X-linked syndromic disorder. ... Full text Link to item Cite

Unrelated Donor Cord Blood Transplantation in Children: Lessons Learned Over 3 Decades.

Journal Article Stem Cells Transl Med · January 30, 2023 Four decades ago, Broxmeyer et al. demonstrated that umbilical cord blood (CB) contained hematopoietic stem cells (HSC) and hypothesized that CB could be used as a source of donor HSC for rescue of myeloablated bone marrow. In 1988, Gluckman et al. reporte ... Full text Link to item Cite

The potential cost-effectiveness of novel cord blood therapies in children with autism spectrum disorder.

Journal Article PLoS One · 2023 OBJECTIVE: To model the long-term clinical and economic outcomes of potential cord blood therapy in autism spectrum disorder (ASD). STUDY DESIGN: Markov microsimulation of ASD over the lifespan was used to compare two strategies: 1) standard of care (SOC), ... Full text Link to item Cite

Improving thymus implantation for congenital athymia with interleukin-7.

Journal Article Clin Transl Immunology · 2023 OBJECTIVES: Thymus implantation is a recently FDA-approved therapy for congenital athymia. Patients receiving thymus implantation develop a functional but incomplete T cell compartment. Our objective was to develop a mouse model to study clinical thymus im ... Full text Open Access Link to item Cite

Motor function and safety after allogeneic cord blood and cord tissue-derived mesenchymal stromal cells in cerebral palsy: An open-label, randomized trial.

Journal Article Dev Med Child Neurol · December 2022 AIM: To evaluate safety and motor function after treatment with allogeneic umbilical cord blood (AlloCB) or umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC) in children with cerebral palsy (CP). METHOD: Ninety-one children (52 males, 39 fe ... Full text Open Access Link to item Cite

CT-314 Multicenter Long-Term Follow Up of Allogeneic Hematopoietic Stem Cell Transplantation With Omidubicel: A Pooled Analysis of Five Prospective Clinical Trials.

Journal Article Clin Lymphoma Myeloma Leuk · October 2022 CONTEXT: Allogeneic hematopoietic stem cell transplantation (allo-HCT) with umbilical cord blood (UCB) is limited by the low number of hematopoietic stem cells. Omidubicel is an ex-vivo expanded stem cell product derived from UCB. Prospective clinical tria ... Full text Link to item Cite

Abstract 12 Phase I Study of Cord Tissue Derived Mesenchymal Stromal Cells in COVID-19–Related Acute Respiratory Distress Syndrome

Conference Stem Cells Translational Medicine · September 6, 2022 AbstractIntroductionSARS-CoV-2 infection results in the COVID-19 disease that caused a global pandemic. In severe cases, COVID-19 lead ... Full text Cite

Abstract 24 Improving Cord Blood Unit Selection and Optimizing Manufacturing of a Unique Cord Blood-Derived Product (DUOC-01)

Conference Stem Cells Translational Medicine · September 6, 2022 AbstractIntroductionDUOC-01 are microglia-like cells derived from umbilical cord blood CD14-positive monocytes, which have been shown ... Full text Cite

Abstract 27 Limitations of Current Human Cell, Tissue, and Cellular-based Product Donor Eligibility Guidance: Analyzing the Impact on a Large Public Cord Blood Bank

Conference Stem Cells Translational Medicine · September 6, 2022 AbstractIntroductionCord blood (CB) is considered a human cell, tissue, and cellular-based product (HCT/P) by the U.S. Food and Drug A ... Full text Cite

Abstract 18 Developing a Potency Assay for Cord Tissue MSCs

Conference Stem Cells Translational Medicine · September 6, 2022 AbstractIntroductionThe potency of mesenchymal stromal cells (MSCs) can be assessed by their ability to suppress proliferation of thir ... Full text Cite

Abstract 5 Human Umbilical Cord Blood-Derived Cell Therapy Product, DUOC-01, Promotes Remyelination by Driving the Differentiation of Oligodendrocyte Progenitor Cells

Conference Stem Cells Translational Medicine · September 6, 2022 AbstractIntroductionDUOC-01 is a macrophage-like cell therapy product manufactured by culturing banked human umbilical cord blood cell ... Full text Cite

Abstract 4 Umbilical Cord-Derived Mesenchymal Stromal Cells Suppress Neuroinflammation and Promote Remyelination in the Spinal Cord

Conference Stem Cells Translational Medicine · September 6, 2022 AbstractIntroductionMesenchymal stromal cells (MSCs) are one of the most widely tested cell therapies due to their ability to suppress ... Full text Cite

Abstract 15 Safety of DUOC-01, Intrathecal Cord Blood-Derived Cellular Therapy, as an Adjunct to Allogeneic Cord Blood Transplantation in Children with Inherited Metabolic Diseases

Conference Stem Cells Translational Medicine · September 6, 2022 AbstractIntroductionUnrelated donor umbilical cord blood transplantation (CBT) improves function and extends life in children with cer ... Full text Cite

Abstract 13 A Randomized, Placebo-Controlled, Phase II Trial of Intravenous Allogeneic Umbilical Cord Blood Infusion for Adults with Ischemic Stroke

Conference Stem Cells Translational Medicine · September 6, 2022 AbstractIntroductionStroke is the fifth leading cause of death in the United States. Tissue plasminogen activator and mechanical throm ... Full text Cite

Maternal Fc-mediated non-neutralizing antibody responses correlate with protection against congenital human cytomegalovirus infection.

Journal Article J Clin Invest · August 15, 2022 Human cytomegalovirus (HCMV) is the most common congenital infection and a leading cause of stillbirth, neurodevelopmental impairment, and pediatric hearing loss worldwide. Development of a maternal vaccine or therapeutic to prevent congenital HCMV has bee ... Full text Open Access Link to item Cite

Efficacy and Safety of MSC Cell Therapies for Hospitalized Patients with COVID-19: A Systematic Review and Meta-Analysis.

Journal Article Stem Cells Transl Med · July 20, 2022 MSC (a.k.a. mesenchymal stem cell or medicinal signaling cell) cell therapies show promise in decreasing mortality in acute respiratory distress syndrome (ARDS) and suggest benefits in treatment of COVID-19-related ARDS. We performed a meta-analysis of pub ... Full text Open Access Link to item Cite

Characterizing human mesenchymal stromal cells' immune-modulatory potency using targeted lipidomic profiling of sphingolipids.

Journal Article Cytotherapy · June 2022 Cell therapies are expected to increase over the next decade owing to increasing demand for clinical applications. Mesenchymal stromal cells (MSCs) have been explored to treat a number of diseases, with some successes in early clinical trials. Despite earl ... Full text Open Access Link to item Cite

Benefits of newborn screening and hematopoietic cell transplant in infantile Krabbe disease.

Journal Article Blood Adv · May 10, 2022 Infantile Krabbe disease (IKD) can be treated with hematopoietic cell transplantation (HCT) if done during the first weeks of life before symptoms develop. To facilitate this, newborn screening (NBS) has been instituted in 8 US states. An application to ad ... Full text Open Access Link to item Cite

Congenital Human Cytomegalovirus Infection Is Associated With Decreased Transplacental IgG Transfer Efficiency Due to Maternal Hypergammaglobulinemia.

Journal Article Clin Infect Dis · April 9, 2022 BACKGROUND: Placentally transferred maternal immunoglobulin G (IgG) protects against pathogens in early life, yet vertically transmitted infections can interfere with transplacental IgG transfer. Although human cytomegalovirus (HCMV) is the most common pla ... Full text Open Access Link to item Cite

Changes in the geometry and robustness of diffusion tensor imaging networks: Secondary analysis from a randomized controlled trial of young autistic children receiving an umbilical cord blood infusion.

Journal Article Front Psychiatry · 2022 Diffusion tensor imaging (DTI) has been used as an outcome measure in clinical trials for several psychiatric disorders but has rarely been explored in autism clinical trials. This is despite a large body of research suggesting altered white matter structu ... Full text Link to item Cite

Guidelines for Pediatric Unrelated Cord Blood Transplantation-Unique Considerations.

Journal Article Transplant Cell Ther · December 2021 Cord blood (CB) is the stem cell source of choice for approximately 30% of pediatric patients undergoing hematopoietic cell transplantation. Cord blood is readily available and is a particularly appealing stem cell source for patients who lack appropriate ... Full text Open Access Link to item Cite

Review of Unrelated Donor Cord Blood Transplantation in Children over the Past 3 Decades

Conference Blood · November 5, 2021 AbstractFour decades ago, Hal Broxmeyer demonstrated that umbilical cord blood (CB) contained hematopoietic stem cells (HSC) and hypothesized that CB could be used as a source of donor HSC for rescue of myel ... Full text Cite

Single-cell RNA-seq of out-of-thaw mesenchymal stromal cells shows tissue-of-origin differences and inter-donor cell-cycle variations.

Journal Article Stem cell research & therapy · November 2021 BackgroundHuman Mesenchymal stromal cells (hMSCs) from various tissue sources are widely investigated in clinical trials. These MSCs are often administered to patients immediately after thawing the cryopreserved product (out-of-thaw), yet little i ... Full text Open Access Cite

Standardization, workforce development and advocacy in cell and gene therapies: a summary of the 2020 Regenerative Medicine InterCHANGE.

Journal Article Cytotherapy · October 2021 Cell and gene therapy is a promising and disruptive new field of medicine for diseases lacking effective treatments. Collaboration among stakeholders has become critically important as investigators, health care providers, manufacturers, couriers, data reg ... Full text Open Access Link to item Cite

Sibling umbilical cord blood infusion is safe in young children with cerebral palsy.

Journal Article Stem Cells Transl Med · September 2021 Preclinical and early phase clinical studies suggest that an appropriately dosed umbilical cord blood (CB) infusion has the potential to help improve motor function in young children with cerebral palsy (CP). As many children with CP do not have their own ... Full text Open Access Link to item Cite

Standardizing Definitions of Hematopoietic Recovery, Graft Rejection, Graft Failure, Poor Graft Function, and Donor Chimerism in Allogeneic Hematopoietic Cell Transplantation: A Report on Behalf of the American Society for Transplantation and Cellular Therapy.

Journal Article Transplant Cell Ther · August 2021 Allogeneic hematopoietic cell transplantation (allo-HCT) is potentially curative for certain hematologic malignancies and nonmalignant diseases. The field of allo-HCT has witnessed significant advances, including broadening indications for transplantation, ... Full text Open Access Link to item Cite

T cell-depleted cultured pediatric thymus tissue as a model for some aspects of human age-related thymus involution.

Journal Article Geroscience · June 2021 Human age-related thymus involution is characterized by loss of developing thymocytes and the thymic epithelial network that supports them, with replacement by adipose tissue. The mechanisms that drive these changes are difficult to study in vivo due to co ... Full text Open Access Link to item Cite

Consensus opinion on immune-mediated cytopenias after hematopoietic cell transplant for inherited metabolic disorders.

Journal Article Bone Marrow Transplant · June 2021 Hematopoietic stem cell transplantation (HCT) has been increasingly used for patients with inherited metabolic disorders (IMD). Immune mediated cytopenias (IMCs) after HCT, manifesting as hemolytic anemia, thrombocytopenia, and/or neutropenia, are recogniz ... Full text Open Access Link to item Cite

MRI surveillance of boys with X-linked adrenoleukodystrophy identified by newborn screening: Meta-analysis and consensus guidelines.

Journal Article J Inherit Metab Dis · May 2021 BACKGROUND: Among boys with X-Linked adrenoleukodystrophy, a subset will develop childhood cerebral adrenoleukodystrophy (CCALD). CCALD is typically lethal without hematopoietic stem cell transplant before or soon after symptom onset. We sought to establis ... Full text Open Access Link to item Cite

Stem cell therapies in cerebral palsy and autism spectrum disorder.

Journal Article Dev Med Child Neurol · May 2021 Across disciplines, there is great anticipation that evolving cell therapies may finally provide a therapeutic option for conditions in dire need. These conditions are typically complex and their pathophysiology incompletely understood, hindering the devel ... Full text Open Access Link to item Cite

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Journal Article J Pediatr · March 2021 Full text Link to item Cite

Allogeneic stem cell transplantation with omidubicel in sickle cell disease.

Journal Article Blood Adv · February 9, 2021 Many patients with sickle cell disease (SCD) do not have HLA-matched related donors for hematopoietic stem cell transplantation (HSCT). Unrelated cord blood (UCB) is an alternative graft option but is historically associated with high graft failure rates, ... Full text Open Access Link to item Cite

Anti-fibrotic effects of different sources of MSC in bleomycin-induced lung fibrosis in C57BL6 male mice.

Journal Article Respirology · February 2021 BACKGROUND AND OBJECTIVE: IPF is a fatal and debilitating lung disorder increasing in incidence worldwide. To date, two approved treatments only slow disease progression, have multiple side effects and do not provide a cure. MSC have promising therapeutic ... Full text Open Access Link to item Cite

Data and Code from: T Cell-Depleted Cultured Pediatric Thymus Tissue as a Model for Some Aspects of Human Age-Related Thymus Involution

Dataset · January 4, 2021 Human age-related thymus involution is characterized by loss of developing thymocytes and the thymic epithelial network that supports them, with replacement by adipose tissue. The mechanisms that drive these changes are difficult to study in vivo due to co ... Full text Cite

Mesenchymal stromal cells reprogram monocytes and macrophages with processing bodies.

Journal Article Stem Cells · January 2021 Mesenchymal stromal cells (MSCs) are widely used in clinical trials because of their ability to modulate inflammation. The success of MSCs has been variable over 25 years, most likely due to an incomplete understanding of their mechanism. After MSCs are in ... Full text Open Access Link to item Cite

Consensus recommendations for the classification and long-term follow up of infants who screen positive for Krabbe Disease.

Journal Article Mol Genet Metab · 2021 OBJECTIVE: To provide updated evidence and consensus-based recommendations for the classification of individuals who screen positive for Krabbe Disease (KD) and recommendations for long-term follow-up for those who are at risk for late onset Krabbe Disease ... Full text Open Access Link to item Cite

Maternal Fc-mediated non-neutralizing antibody responses correlate with protection against congenital human cytomegalovirus infection

Journal Article · 2021 Human cytomegalovirus (HCMV) is the most common congenital infection and a leading cause of stillbirth, neurodevelopmental impairment, and pediatric hearing loss worldwide. Development of a maternal vaccine or therapeutic to prevent congenital infection ha ... Full text Open Access Cite

Umbilical Cord Blood or Mesenchymal Stromal Cells as Potential Therapies for Autism Spectrum Disorder

Chapter · January 1, 2021 Autism spectrum disorder (ASD) is a neurodevelopmental disorder that presents in childhood and is characterized by restricted, repetitive behaviors and impairments in social communication. The exact cause in unknown, but genetic and immunologic factors are ... Full text Cite

Infusion of human umbilical cord tissue mesenchymal stromal cells in children with autism spectrum disorder.

Journal Article Stem Cells Transl Med · October 2020 Ongoing neuroinflammation may contribute to symptoms of autism spectrum disorder (ASD) in at least a portion of affected individuals. Mesenchymal stromal cells (MSCs) have demonstrated the capacity to modulate neuroinflammation, but safety and feasibility ... Full text Open Access Link to item Cite

Guidelines for Cord Blood Unit Thaw and Infusion.

Journal Article Biol Blood Marrow Transplant · October 2020 Full text Open Access Link to item Cite

Cell-based therapy to reduce mortality from COVID-19: Systematic review and meta-analysis of human studies on acute respiratory distress syndrome.

Journal Article Stem Cells Transl Med · September 2020 Severe cases of COVID-19 infection, often leading to death, have been associated with variants of acute respiratory distress syndrome (ARDS). Cell therapy with mesenchymal stromal cells (MSCs) is a potential treatment for COVID-19 ARDS based on preclinical ... Full text Open Access Link to item Cite

Measuring robustness of brain networks in autism spectrum disorder with Ricci curvature.

Journal Article Sci Rep · July 2, 2020 Ollivier-Ricci curvature is a method for measuring the robustness of connections in a network. In this work, we use curvature to measure changes in robustness of brain networks in children with autism spectrum disorder (ASD). In an open label clinical tria ... Full text Open Access Link to item Cite

A Phase II Randomized Clinical Trial of the Safety and Efficacy of Intravenous Umbilical Cord Blood Infusion for Treatment of Children with Autism Spectrum Disorder.

Journal Article J Pediatr · July 2020 OBJECTIVE: To evaluate whether umbilical cord blood (CB) infusion is safe and associated with improved social and communication abilities in children with autism spectrum disorder (ASD). STUDY DESIGN: This prospective, randomized, placebo-controlled, doubl ... Full text Open Access Link to item Cite

Intrastromal Gene Therapy Prevents and Reverses Advanced Corneal Clouding in a Canine Model of Mucopolysaccharidosis I.

Journal Article Mol Ther · June 3, 2020 Mucopolysaccharidosis type I (MPS I) is an autosomal recessive lysosomal storage disease characterized by severe phenotypes, including corneal clouding. MPS I is caused by mutations in alpha-l-iduronidase (IDUA), a ubiquitous enzyme that catalyzes the hydr ... Full text Open Access Link to item Cite

The critical role of psychosine in screening, diagnosis, and monitoring of Krabbe disease.

Journal Article Genet Med · June 2020 PURPOSE: Newborn screening (NBS) for Krabbe disease (KD) is performed by measurement of galactocerebrosidase (GALC) activity as the primary test. This revealed that GALC activity has poor specificity for KD. Psychosine (PSY) was proposed as a disease marke ... Full text Open Access Link to item Cite

Leukocyte and Dried Blood Spot Arylsulfatase A Assay by Tandem Mass Spectrometry.

Journal Article Anal Chem · May 5, 2020 Liquid chromatography-tandem mass spectrometry (LC-MS/MS) assays were developed to measure arylsulfatase A (ARSA) activity in leukocytes and dried blood spots (DBS) using deuterated natural sulfatide substrate. These new assays were highly specific and sen ... Full text Open Access Link to item Cite

A Phase 3, Single-Arm, Prospective Study of Remestemcel-L, Ex Vivo Culture-Expanded Adult Human Mesenchymal Stromal Cells for the Treatment of Pediatric Patients Who Failed to Respond to Steroid Treatment for Acute Graft-versus-Host Disease.

Journal Article Biol Blood Marrow Transplant · May 2020 Steroid-refractory acute graft-versus-host disease (SR-aGVHD) following hematopoietic cell transplantation (HSCT) is associated with poor clinical outcomes. Currently, there are no safe and effective therapies approved for use in the pediatric population u ... Full text Open Access Link to item Cite

Study 275: Updated Expanded Access Program for Remestemcel-L in Steroid-Refractory Acute Graft-versus-Host Disease in Children.

Journal Article Biol Blood Marrow Transplant · May 2020 Clinical outcomes in children with steroid-refractory acute graft-versus-host disease (SR-aGVHD) are generally poor, with a high mortality rate and limited therapeutic options. Here we report our updated investigational experience with mesenchymal stromal ... Full text Open Access Link to item Cite

Unlicensed Umbilical Cord Blood Units Provide a Safe and Effective Graft Source for a Diverse Population: A Study of 2456 Umbilical Cord Blood Recipients.

Journal Article Biol Blood Marrow Transplant · April 2020 Umbilical cord blood (UCB) transplantation (UCBT) is a curative procedure for patients with hematologic malignancies and genetic disorders and expands access to transplantation for non-Caucasian patients unable to find a fully matched unrelated donor. In 2 ... Full text Open Access Link to item Cite

Noninvasive white blood cell quantification in umbilical cord blood collection bags with quantitative oblique back-illumination microscopy.

Journal Article Transfusion · March 2020 BACKGROUND: Umbilical cord blood has become an important source of hematopoietic stem and progenitor cells for therapeutic applications. However, cord blood banking (CBB) grapples with issues related to economic viability, partially due to high discard rat ... Full text Open Access Link to item Cite

Histopathologic assessment of cultured human thymus.

Journal Article PLoS One · 2020 The maintenance and propagation of complex mixtures of cells in vitro in the form of native organs or engineered organoids has contributed to understanding mechanisms of cell and organ development and function which can be translated into therapeutic benef ... Full text Open Access Link to item Cite

The Effects of Umbilical Cord Blood and Cord Tissue Cell Therapies in Animal and Human Models of Cerebral Palsy

Chapter · January 1, 2020 Biologic and cell-based therapies are increasingly being developed for a multitude of diseases. They are being explored in the treatment of neurologic conditions, as traditional pharmacologic agents typically cannot fully address the pathologic complexity ... Full text Cite

Hematopoietic Stem Cell Transplantation to Treat Leukodystrophies: Clinical Practice Guidelines from the Hunter's Hope Leukodystrophy Care Network.

Journal Article Biol Blood Marrow Transplant · December 2019 The leukodystrophies are a heterogeneous group of inherited diseases characterized by progressive demyelination of the central nervous system leading to devastating neurologic symptoms and premature death. Hematopoietic stem cell transplantation (HSCT) has ... Full text Open Access Link to item Cite

No Engraftment Advantage after Single or Double Umbilical Cord Blood Transplant (CBT) with the Addition of a Non-HLA Matched Off-the-Shelf Expanded Cord Blood Unit Compared to Conventional CBT: Results of a Randomized Trial

Conference Blood · November 13, 2019 Background: Based on pilot study data demonstrating safety and excellent survival [Blood 2014 124:46] in acute leukemia patients undergoing myeloablative cord blood transplant (CBT) plus infusion of an off-the-shelf non-HLA matched expanded CB unit ... Full text Cite

A Genocentric Approach to Discovery of Mendelian Disorders.

Journal Article Am J Hum Genet · November 7, 2019 The advent of inexpensive, clinical exome sequencing (ES) has led to the accumulation of genetic data from thousands of samples from individuals affected with a wide range of diseases, but for whom the underlying genetic and molecular etiology of their cli ... Full text Open Access Link to item Cite

Massive clonal expansion of medulloblastoma-specific T cells during adoptive cellular therapy.

Journal Article Sci Adv · November 2019 In both human and murine systems, we have developed an adoptive cellular therapy platform against medulloblastoma and glioblastoma that uses dendritic cells pulsed with a tumor RNA transcriptome to expand polyclonal tumor-reactive T cells against a plurali ... Full text Open Access Link to item Cite

Pulmonary Complications in Pediatric and Adolescent Patients Following Allogeneic Hematopoietic Cell Transplantation.

Journal Article Biol Blood Marrow Transplant · October 2019 Pulmonary complications after hematopoietic cell transplantation (HCT) can lead to significant morbidity and mortality. Limited evaluation of the true incidence of these complications in children and subsequent outcomes of these complications have not been ... Full text Open Access Link to item Cite

1.14 GRAPH CURVATURE AS A METHOD FOR DISCERNING ROBUSTNESS IN BRAIN NETWORKS IN ASD

Conference Journal of the American Academy of Child & Adolescent Psychiatry · October 2019 Full text Cite

Survey of quality of life, phenotypic expression, and response to treatment in Krabbe leukodystrophy.

Journal Article JIMD Rep · May 2019 OBJECTIVES: To develop a quality of life (QOL) survey for Krabbe disease (KD), and to thereby improve understanding of its phenotypic expression and response to treatment. METHODS: The survey, the Leukodystrophy Quality of Life Assessment (LQLA) and the Vi ... Full text Open Access Link to item Cite

CD34+ cell content of 126 341 cord blood units in the US inventory: implications for transplantation and banking.

Journal Article Blood Adv · April 23, 2019 CD34+ cell dose is critical for cord blood (CB) engraftment. However, the CD34+ content of the CB inventory in the United States is unknown. We examined the CD34+ cell content of 126 341 red blood cell-depleted US units banked from January 2007 to Septembe ... Full text Open Access Link to item Cite

The influence of stem cell source on transplant outcomes for pediatric patients with acute myeloid leukemia.

Journal Article Blood Adv · April 9, 2019 When hematopoietic stem cell transplant (HSCT) is necessary for children with acute myeloid leukemia (AML), there remains debate about the best stem cell source. Post-HSCT relapse is a common cause of mortality, and complications such as chronic graft vers ... Full text Open Access Link to item Cite

Reduction in Mortality after Umbilical Cord Blood Transplantation in Children Over a 20-Year Period (1995-2014).

Journal Article Biol Blood Marrow Transplant · April 2019 Infections and graft-versus-host disease (GVHD) have historically resulted in high mortality among children undergoing umbilical cord blood transplantation (UCBT). However, recent advances in clinical practice have likely improved outcomes of these patient ... Full text Open Access Link to item Cite

Ethical considerations of using a single minor donor for three bone marrow harvests for three HLA-matched siblings with primary immunodeficiency.

Journal Article Pediatr Blood Cancer · April 2019 Allogeneic hematopoietic stem cell transplantation is curative for primary immunodeficiencies. Bone marrow from an unaffected human leukocyte antigen (HLA)-identical sibling donor is the ideal graft source. For minor donors, meaningful consent or assent ma ... Full text Open Access Link to item Cite

Extending Cord Blood to Regenerative Therapies for the Brain

Conference CELL TRANSPLANTATION · April 1, 2019 Link to item Cite

Survival and Functional Outcomes in Boys with Cerebral Adrenoleukodystrophy with and without Hematopoietic Stem Cell Transplantation.

Journal Article Biol Blood Marrow Transplant · March 2019 Cerebral adrenoleukodystrophy (CALD) is a rapidly progressing, often fatal neurodegenerative disease caused by mutations in the ABCD1 gene, resulting in deficiency of ALD protein. Clinical benefit has been reported following allogeneic hematopoietic stem c ... Full text Open Access Link to item Cite

The Hematopoietic Cell Transplant Comorbidity Index predicts survival after allogeneic transplant for nonmalignant diseases.

Journal Article Blood · February 14, 2019 Despite improvements, mortality after allogeneic hematopoietic cell transplantation (HCT) for nonmalignant diseases remains a significant problem. We evaluated whether pre-HCT conditions defined by the HCT Comorbidity Index (HCT-CI) predict probability of ... Full text Open Access Link to item Cite

Phase I/II Study of Stem-Cell Transplantation Using a Single Cord Blood Unit Expanded Ex Vivo With Nicotinamide.

Journal Article J Clin Oncol · February 10, 2019 PURPOSE: Increasing the number of hematopoietic stem and progenitor cells within an umbilical cord blood (UCB) graft shortens the time to hematopoietic recovery after UCB transplantation. In this study, we assessed the safety and efficacy of a UCB graft th ... Full text Open Access Link to item Cite

White Matter Tract Changes Associated with Clinical Improvement in an Open-Label Trial Assessing Autologous Umbilical Cord Blood for Treatment of Young Children with Autism.

Journal Article Stem Cells Transl Med · February 2019 Autism spectrum disorder (ASD) is a heterogeneous neurodevelopmental disorder characterized by social communication deficits and the presence of restricted interests and repetitive behaviors. We have previously reported significant improvements in behavior ... Full text Open Access Link to item Cite

Mutations in NCAPG2 Cause a Severe Neurodevelopmental Syndrome that Expands the Phenotypic Spectrum of Condensinopathies.

Journal Article Am J Hum Genet · January 3, 2019 The use of whole-exome and whole-genome sequencing has been a catalyst for a genotype-first approach to diagnostics. Under this paradigm, we have implemented systematic sequencing of neonates and young children with a suspected genetic disorder. Here, we r ... Full text Open Access Link to item Cite

Neutropenia in glycogen storage disease Ib: outcomes for patients treated with granulocyte colony-stimulating factor.

Journal Article Curr Opin Hematol · January 2019 PURPOSE OF REVIEW: Glycogen storage disease Ib (GSD Ib) is characterized by hepatomegaly, hypoglycemia, neutropenia, enterocolitis and recurrent bacterial infections. It is attributable to mutations in G6PT1, the gene for the glucose-6-phosphate transporte ... Full text Open Access Link to item Cite

Mesenchymal Stem Cell Therapy and Chronic Non-Tuberculous Mycobacterium Infection

Conference AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE · January 1, 2019 Link to item Cite

Human umbilical cord blood monocytes, but not adult blood monocytes, rescue brain cells from hypoxic-ischemic injury: Mechanistic and therapeutic implications.

Journal Article PLoS One · 2019 Cord blood (CB) mononuclear cells (MNC) are being tested in clinical trials to treat hypoxic-ischemic (HI) brain injuries. Although early results are encouraging, mechanisms underlying potential clinical benefits are not well understood. To explore these m ... Full text Open Access Link to item Cite

Outcomes of Umbilical Cord Blood Transplantation in Children with Batten Disease

Conference BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION · 2019 Cite

Automated thawing increases recovery of colony-forming units from banked cord blood unit grafts.

Conference Transfusion · December 2018 BACKGROUND: The cell dose infused for cord blood transplantation strongly correlates with outcomes following transplantation. Post thaw recoveries can be improved by washing cord blood units (CBUs) in dextran/albumin. Early methods used a labor-intensive m ... Full text Link to item Cite

Long-Term Functional Outcomes after Hematopoietic Stem Cell Transplant for Early Infantile Krabbe Disease.

Journal Article Biol Blood Marrow Transplant · November 2018 Allogeneic hematopoietic stem cell transplantation (HSCT) can retard the progression of early infantile Krabbe disease (EIKD). Superior outcomes are achieved if HSCT is performed before the onset of symptoms; however, little information is available about ... Full text Open Access Link to item Cite

Electrophysiological Biomarkers Predict Clinical Improvement in an Open-Label Trial Assessing Efficacy of Autologous Umbilical Cord Blood for Treatment of Autism.

Journal Article Stem Cells Transl Med · November 2018 This study was a phase I, single-center, and open-label trial of a single intravenous infusion of autologous umbilical cord blood in young children with autism spectrum disorder (ASD). Twenty-five children between the ages of 2 and 6 with a confirmed diagn ... Full text Open Access Link to item Cite

Allogeneic Umbilical Cord Blood Infusion for Adults with Ischemic Stroke: Clinical Outcomes from a Phase I Safety Study.

Journal Article Stem Cells Transl Med · July 2018 Stroke is a major cause of death and long-term disability, affecting one in six people worldwide. The only currently available approved pharmacological treatment for ischemic stroke is tissue plasminogen activator; however, relatively few patients are elig ... Full text Open Access Link to item Cite

Dual-wavelength oblique back-illumination microscopy for the non-invasive imaging and quantification of blood in collection and storage bags.

Journal Article Biomed Opt Express · June 1, 2018 There is currently no low-cost method to quantitatively assess the contents of a blood bag without breaching the bag and potentially damaging the sample. Towards this end, we adapt oblique back-illumination microscopy (OBM) to rapidly, inexpensively, and n ... Full text Open Access Link to item Cite

Consensus guidelines for newborn screening, diagnosis and treatment of infantile Krabbe disease.

Journal Article Orphanet J Rare Dis · February 1, 2018 BACKGROUND: Krabbe disease is a rare neurodegenerative genetic disorder caused by deficiency of galactocerebrosidase. Patients with the infantile form of Krabbe disease can be treated at a presymptomatic stage with human stem cell transplantation which imp ... Full text Open Access Link to item Cite

Early and late outcomes after cord blood transplantation for pediatric patients with inherited leukodystrophies.

Journal Article Blood Adv · January 9, 2018 Leukodystrophies (LD) are devastating inherited disorders leading to rapid neurological deterioration and premature death. Hematopoietic stem cell transplantation (HSCT) can halt disease progression for selected LD. Cord blood is a common donor source for ... Full text Open Access Link to item Cite

Cell therapy for diverse central nervous system disorders: inherited metabolic diseases and autism.

Journal Article Pediatr Res · January 2018 The concept of utilizing human cells for the treatment of medical conditions is not new. In its simplest form, blood product transfusion as treatment of severe hemorrhage has been practiced since the 1800s. The advent of hematopoietic stem cell transplanta ... Full text Open Access Link to item Cite

Cord Blood Stem Cells

Chapter · January 1, 2018 Cord blood (CB) has been established as a viable source of donor cells for hematopoietic reconstitution in allogeneic transplantation for all of the clinical indications treated with adult hematopoietic stem cells. CB is the only hematopoietic stem cell so ... Full text Cite

Effect of Autologous Cord Blood Infusion on Motor Function and Brain Connectivity in Young Children with Cerebral Palsy: A Randomized, Placebo-Controlled Trial.

Journal Article Stem Cells Transl Med · December 2017 Cerebral palsy (CP) is a condition affecting young children that causes lifelong disabilities. Umbilical cord blood cells improve motor function in experimental systems via paracrine signaling. After demonstrating safety, we conducted a phase II trial of a ... Full text Open Access Link to item Cite

Durable Chimerism and Long-Term Survival after Unrelated Umbilical Cord Blood Transplantation for Pediatric Hemophagocytic Lymphohistiocytosis: A Single-Center Experience.

Journal Article Biol Blood Marrow Transplant · October 2017 Hemophagocytic lymphohistiocytosis (HLH) is a life-threatening disorder of immune dysregulation characterized by fever, hepatosplenomegaly, cytopenias, central nervous system disease, increased inflammatory markers, and hemophagocytosis. Currently, allogen ... Full text Open Access Link to item Cite

Umbilical Cord Blood Transplantation in Children with Acute Leukemia: Impact of Conditioning on Transplantation Outcomes.

Journal Article Biol Blood Marrow Transplant · October 2017 The Blood and Marrow Transplant Clinical Trials Network (BMT CTN 0501) randomized children with hematologic malignancies to transplantation with 1 or 2 cord blood units (UCB) between 2006 and 2012. While the trial concluded that survival was similar regard ... Full text Open Access Link to item Cite

Factors Associated with Long-Term Risk of Relapse after Unrelated Cord Blood Transplantation in Children with Acute Lymphoblastic Leukemia in Remission.

Journal Article Biol Blood Marrow Transplant · August 2017 For pediatric patients with acute lymphoblastic leukemia (ALL), relapse is an important cause of treatment failure after unrelated cord blood transplant (UCBT). Compared with other donor sources, relapse is similar or even reduced after UCBT despite less g ... Full text Open Access Link to item Cite

Lymphocyte Galactocerebrosidase Activity by LC-MS/MS for Post-Newborn Screening Evaluation of Krabbe Disease.

Journal Article Clin Chem · August 2017 BACKGROUND: Deficiency of the lysosomal enzyme galactosylcerebrosidase (GALC) causes Krabbe disease. Newborn screening for Krabbe disease is ongoing, but improved methods for follow-up analysis of screen-positive babies are needed to better advise families ... Full text Open Access Link to item Cite

Allele-level HLA matching for umbilical cord blood transplantation for non-malignant diseases in children: a retrospective analysis.

Journal Article Lancet Haematol · July 2017 BACKGROUND: The standard for selecting unrelated umbilical cord blood units for transplantation for non-malignant diseases relies on antigen-level (lower resolution) HLA typing for HLA-A and HLA-B, and allele-level for HLA-DRB1. We aimed to study the effec ... Full text Open Access Link to item Cite

A risk factor analysis of outcomes after unrelated cord blood transplantation for children with Wiskott-Aldrich syndrome.

Journal Article Haematologica · June 2017 Wiskott-Aldrich syndrome is a severe X-linked recessive immune deficiency disorder. A scoring system of Wiskott-Aldrich syndrome severity (0.5-5) distinguishes two phenotypes: X-linked thrombocytopenia and classic Wiskott-Aldrich syndrome. Hematopoietic ce ... Full text Open Access Link to item Cite

Optimal Practices in Unrelated Donor Cord Blood Transplantation for Hematologic Malignancies.

Journal Article Biol Blood Marrow Transplant · June 2017 Unrelated donor cord blood transplantation (CBT) results in disease-free survival comparable to that of unrelated adult donor transplantation in patients with hematologic malignancies. Extension of allograft access to racial and ethnic minorities, rapid gr ... Full text Open Access Link to item Cite

Gene products promoting remyelination are up-regulated in a cell therapy product manufactured from banked human cord blood.

Journal Article Cytotherapy · June 2017 BACKGROUND AIMS: DUOC-01, a cell product being developed to treat demyelinating conditions, is composed of macrophages that arise from CD14+ monocytes in the mononuclear cell (MNC) population of banked cord blood (CB). This article demonstrates that expres ... Full text Open Access Link to item Cite

AAV Gene Therapy in a Canine Model of MPS1 Prevents and Reverses Corneal Blindness

Conference INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE · June 1, 2017 Link to item Cite

Autologous Cord Blood Infusions Are Safe and Feasible in Young Children with Autism Spectrum Disorder: Results of a Single-Center Phase I Open-Label Trial.

Journal Article Stem Cells Transl Med · May 2017 Despite advances in early diagnosis and behavioral therapies, more effective treatments for children with autism spectrum disorder (ASD) are needed. We hypothesized that umbilical cord blood-derived cell therapies may have potential in alleviating ASD symp ... Full text Open Access Link to item Cite

Announcing a New Journal Section: Cord Blood.

Journal Article Stem Cells Transl Med · May 2017 Full text Open Access Link to item Cite

Effect of maternal obesity on Maternal-Fetal transfer of preoperative cefazolin at cesarean section

Journal Article Journal of Pediatric Pharmacology and Therapeutics · May 1, 2017 OBJECTIVES American Congress of Obstetricians and Gynecologists recommends a single dose of antibiotic prophylaxis before all cesarean sections (C/S). This recommendation is based on pharmacokinetic studies that include only non-obese patients. We sought t ... Full text Open Access Cite

Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation.

Journal Article Blood · March 16, 2017 Despite advances in supportive therapy to prevent complications of sickle cell disease (SCD), access to care is not universal. Hematopoietic cell transplantation is, to date, the only curative therapy for SCD, but its application is limited by availability ... Full text Open Access Link to item Cite

Brincidofovir for Asymptomatic Adenovirus Viremia in Pediatric and Adult Allogeneic Hematopoietic Cell Transplant Recipients: A Randomized Placebo-Controlled Phase II Trial.

Journal Article Biol Blood Marrow Transplant · March 2017 Adenovirus infection in immunocompromised patients contributes to significant morbidity and mortality, especially after allogeneic hematopoietic cell transplantation (HCT). Brincidofovir (BCV, CMX001) is an orally bioavailable lipid conjugate of cidofovir ... Full text Open Access Link to item Cite

Gallbladder abnormalities in children with metachromatic leukodystrophy.

Journal Article J Surg Res · February 2017 BACKGROUND: Metachromatic leukodystrophy (MLD) is a lysosomal storage disease that leads to neurological deterioration and visceral involvement, including sulphatide deposition in the gallbladder wall. Using our institution's extensive experience in treati ... Full text Open Access Link to item Cite

Treatment of Childhood Apraxia with Autologous Cord Blood Infusions

Conference BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION · 2017 Cite

Allogeneic Umbilical Cord Blood Infusion for Adults with Ischemic Stroke (CoBIS): Clinical Outcomes from a Phase 1 Safety Study

Conference Blood · December 2, 2016 AbstractBackground: Over 750,000 patients/year in the US experience a debilitating stroke. Approximately 88% of adult strokes are ischemic in etiology, and occur when cerebral blood flow is blocked by a clot ... Full text Cite

Umbilical Cord Blood (UCB) Transplantation in Children with Acute Leukemia: Impact of Conditioning Regimen on Transplant Outcomes

Conference Blood · December 2, 2016 AbstractThe Blood and Marrow Transplant Clinical Trials Network (BMT CTN 0501; NCT00412360) randomized children with hematologic malignancy to one or two cord blood unit transplantation between December 2006 ... Full text Cite

Successful Engraftment of Donor Umbilical Cord Blood (UCB) Cells after Co-Transplantation of Nicord® (Ex Vivo Expanded UCB Progenitor Cells with Nicotinamide) and a Second Unmanipulated Cord Blood Unit after Myeloablative Chemotherapy in Pediatric Patients with Severe Sickle Cell Disease

Conference Blood · December 2, 2016 AbstractBackground: Patients with severe sickle cell disease (SCD) experience organ damage, poor quality of life, and are at high risk of premature mortality. To date, allogeneic hematopoietic stem cell tran ... Full text Cite

Clinical outcomes of children with abnormal newborn screening results for Krabbe disease in New York State.

Journal Article Genet Med · December 2016 BACKGROUND: Early infantile Krabbe disease is rapidly fatal, but hematopoietic stem cell transplantation (HSCT) may improve outcomes if performed soon after birth. New York State began screening all newborns for Krabbe disease in 2006. METHODS: Infants wit ... Full text Open Access Link to item Cite

Outcomes after Unrelated Umbilical Cord Blood Transplantation for Children with Osteopetrosis.

Journal Article Biol Blood Marrow Transplant · November 2016 Hematopoietic stem cell transplantation (HSCT) is the only curative treatment for most children with osteopetrosis (OP). Timing of HSCT is critical; therefore, umbilical cord blood transplantation (UCBT) is an attractive option. We analyzed outcomes after ... Full text Open Access Link to item Cite

How transplant centers deal with the dextran shortage: recommendations for comparing alternatives.

Journal Article Transfusion · November 2016 BACKGROUND: In the United States, dextran 40 in 0.9% NaCl is the preferred reagent for the thawing and preparation of cord blood units for hematopoietic stem cell transplantation. The recurring nationwide shortage of this reagent could have implications th ... Full text Open Access Link to item Cite

Late Effects after Umbilical Cord Blood Transplantation in Very Young Children after Busulfan-Based, Myeloablative Conditioning.

Journal Article Biol Blood Marrow Transplant · September 2016 Infants and young children who undergo allogeneic cord blood transplantation (CBT) are at increased risk for late effects because of exposure of developing organs to chemotherapy and radiation therapy typically used in transplant conditioning regimens. Bus ... Full text Open Access Link to item Cite

A cord blood monocyte-derived cell therapy product accelerates brain remyelination.

Journal Article JCI Insight · August 18, 2016 Microglia and monocytes play important roles in regulating brain remyelination. We developed DUOC-01, a cell therapy product intended for treatment of demyelinating diseases, from banked human umbilical cord blood (CB) mononuclear cells. Immunodepletion an ... Full text Open Access Link to item Cite

Voriconazole pharmacokinetics following HSCT: results from the BMT CTN 0101 trial.

Journal Article J Antimicrob Chemother · August 2016 BACKGROUND: Voriconazole is a first-line agent for the prevention and treatment of a number of invasive fungal diseases. Relatively little is known about the relationship between drug exposure and the prevention of invasive fungal infections. PATIENTS AND ... Full text Open Access Link to item Cite

Chagas Disease Screening in Maternal Donors of Publicly Banked Umbilical Cord Blood, United States.

Journal Article Emerg Infect Dis · August 2016 To assess patterns of Chagas disease, we reviewed results of screening umbilical cord blood from a US public cord blood bank during 2007-2014. Nineteen maternal donors tested positive for Trypanosoma cruzi parasites (0.04%). Because perinatal transmission ... Full text Open Access Link to item Cite

Development and validation of a rapid, aldehyde dehydrogenase bright-based cord blood potency assay.

Journal Article Blood · May 12, 2016 Banked, unrelated umbilical cord blood provides access to hematopoietic stem cell transplantation for patients lacking matched bone marrow donors, yet 10% to 15% of patients experience graft failure or delayed engraftment. This may be due, at least in part ... Full text Open Access Link to item Cite

Newborn screening for Krabbe disease in New York State: the first eight years' experience.

Journal Article Genet Med · March 2016 PURPOSE: Krabbe disease (KD) results from galactocerebrosidase (GALC) deficiency. Infantile KD symptoms include irritability, progressive stiffness, developmental delay, and death. The only potential treatment is hematopoietic stem cell transplantation. Ne ... Full text Open Access Link to item Cite

Allogeneic Umbilical Cord Blood Infusion for Adults with Ischemic Stroke

Conference Biology of Blood and Marrow Transplantation · March 2016 Full text Cite

Use of Umbilical Cord Blood to Treat Non-Fatal Submersion Events

Conference Biology of Blood and Marrow Transplantation · March 2016 Full text Cite

AAV Gene Therapy for MPS1-associated Corneal Blindness.

Journal Article Sci Rep · February 22, 2016 Although cord blood transplantation has significantly extended the lifespan of mucopolysaccharidosis type 1 (MPS1) patients, over 95% manifest cornea clouding with about 50% progressing to blindness. As corneal transplants are met with high rejection rates ... Full text Open Access Link to item Cite

Accelerating stem cell trials for Alzheimer's disease.

Journal Article Lancet Neurol · February 2016 At present, no effective cure or prophylaxis exists for Alzheimer's disease. Symptomatic treatments are modestly effective and offer only temporary benefit. Advances in induced pluripotent stem cell (iPSC) technology have the potential to enable developmen ... Full text Open Access Link to item Cite

Current perspectives on the use of ancillary materials for the manufacture of cellular therapies.

Journal Article Cytotherapy · January 2016 Continued growth in the cell therapy industry and commercialization of cell therapies that successfully advance through clinical trials has led to increased awareness around the need for specialized and complex materials utilized in their manufacture. Anci ... Full text Open Access Link to item Cite

530: Preterm umbilical cord blood is enriched for progenitor cells

Conference American Journal of Obstetrics and Gynecology · January 2016 Full text Cite

Cord Blood Transplantation in Hematological and Metabolic Diseases

Chapter · January 1, 2016 Umbilical cord blood (CB), as a source of hematopoietic stem and progenitor cells for hematopoietic reconstitution, has been used for over two decades in allogeneic transplantation to treat patients with hematological malignancies, bone marrow failure, imm ... Full text Cite

Repeated autologous umbilical cord blood infusions are feasible and had no acute safety issues in young babies with congenital hydrocephalus.

Journal Article Pediatr Res · December 2015 BACKGROUND: Babies with congenital hydrocephalus often experience developmental disabilities due to brain injury associated with prolonged increased pressure on the developing brain parenchyma. Umbilical cord blood (CB) infusion has favorable effects in an ... Full text Open Access Link to item Cite

Hydroxyethyl starch as a substitute for dextran 40 for thawing peripheral blood progenitor cell products.

Journal Article Cytotherapy · December 2015 BACKGROUND AIMS: Removing DMSO post-thaw results in: reduced infusion reactions, improved recovery and stability of viable CD34+ cells. Validated methods use 5%-8.3% Dextran 40 with 2.5%-4.2% HSA for this purpose. Recent shortages of clinical grade Dextran ... Full text Open Access Link to item Cite

27: Chagas disease screening in umbilical cord blood: an opportunity for prevention

Conference American Journal of Obstetrics and Gynecology · December 2015 Full text Cite

Durable engraftment and correction of hematological abnormalities in children with congenital amegakaryocytic thrombocytopenia following myeloablative umbilical cord blood transplantation.

Journal Article Pediatr Transplant · November 2015 The use of HSCT is the only potentially curative treatment for CAMT, but access is limited by the availability of suitable donors. We report five consecutive patients with CAMT who received MAC and partially HLA-mismatched, UCBT (unrelated, n = 4). Median ... Full text Open Access Link to item Cite

Umbilical cord blood donation: public or private?

Journal Article Bone Marrow Transplant · October 2015 Umbilical cord blood (UCB) is a graft source for patients with malignant or genetic diseases who can be cured by allogeneic hematopoietic cell transplantation (HCT), but who do not have an appropriately HLA-matched family or volunteer unrelated adult donor ... Full text Open Access Link to item Cite

Reprint of: Preclinical characterization of DUOC-01, a cell therapy product derived from banked umbilical cord blood for use as an adjuvant to umbilical cord blood transplantation for treatment of inherited metabolic diseases.

Journal Article Cytotherapy · September 2015 BACKGROUND AIMS: Cord blood (CB) transplantation slows neurodegeneration during certain inherited metabolic diseases. However, the number of donor cells in the brain of patients does not appear to be sufficient to provide benefit until several months after ... Full text Open Access Link to item Cite

Identification of cis-suppression of human disease mutations by comparative genomics.

Journal Article Nature · August 13, 2015 Patterns of amino acid conservation have served as a tool for understanding protein evolution. The same principles have also found broad application in human genomics, driven by the need to interpret the pathogenic potential of variants in patients. Here w ... Full text Open Access Link to item Cite

Rapid and Efficient Generation of Transgene-Free iPSC from a Small Volume of Cryopreserved Blood.

Journal Article Stem Cell Rev Rep · August 2015 Human peripheral blood and umbilical cord blood represent attractive sources of cells for reprogramming to induced pluripotent stem cells (iPSCs). However, to date, most of the blood-derived iPSCs were generated using either integrating methods or starting ... Full text Open Access Link to item Cite

Preclinical characterization of DUOC-01, a cell therapy product derived from banked umbilical cord blood for use as an adjuvant to umbilical cord blood transplantation for treatment of inherited metabolic diseases.

Journal Article Cytotherapy · June 2015 BACKGROUND AIMS: Cord blood (CB) transplantation slows neurodegeneration during certain inherited metabolic diseases. However, the number of donor cells in the brain of patients does not appear to be sufficient to provide benefit until several months after ... Full text Open Access Link to item Cite

Cord blood for brain injury.

Journal Article Cytotherapy · June 2015 Recovery from neurological injuries is typically incomplete and often results in significant and permanent disabilities. Currently, most available therapies are limited to supportive or palliative measures, aimed at managing the symptoms of the condition. ... Full text Open Access Link to item Cite

Cord blood is the optimal graft source for the treatment of pediatric patients with lysosomal storage diseases: clinical outcomes and future directions.

Journal Article Cytotherapy · June 2015 Initially used as an alternative hematopoietic stem cell source for patients without a human leukocyte antigen-matched bone marrow or peripheral blood stem cell donor, unrelated cord blood (UCB) is now the preferred donor source when hematopoietic stem cel ... Full text Open Access Link to item Cite

Newborns of obese parents have altered DNA methylation patterns at imprinted genes

Journal Article International Journal of Obesity · April 9, 2015 Background:Several epidemiologic studies have demonstrated associations between periconceptional environmental exposures and health status of the offspring in later life. Although these environmentally related effects have been attributed to epigenetic cha ... Full text Open Access Cite

Effect of cord blood processing on transplantation outcomes after single myeloablative umbilical cord blood transplantation.

Journal Article Biol Blood Marrow Transplant · April 2015 Variations in cord blood manufacturing and administration are common, and the optimal practice is not known. We compared processing and banking practices at 16 public cord blood banks (CBB) in the United States and assessed transplantation outcomes on 530 ... Full text Open Access Link to item Cite

Newborns of obese parents have altered DNA methylation patterns at imprinted genes.

Journal Article Int J Obes (Lond) · April 2015 BACKGROUND: Several epidemiologic studies have demonstrated associations between periconceptional environmental exposures and health status of the offspring in later life. Although these environmentally related effects have been attributed to epigenetic ch ... Full text Open Access Link to item Cite

Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study.

Journal Article Blood · March 26, 2015 Mucopolysaccharidosis type I-Hurler syndrome (MPS-IH) is a lysosomal storage disease characterized by multisystem morbidity and death in early childhood. Although hematopoietic cell transplantation (HCT) has been performed in these patients for more than 3 ... Full text Open Access Link to item Cite

Early HSCT corrects the skeleton in MPS.

Journal Article Blood · March 5, 2015 In this issue of Blood, Pievani et al have identified a potential solution to the remaining barrier to the success of hematopoietic stem cell transplantation (HSCT) in children with severe phenotype Hurler syndrome (mucopolysaccharidosis type I [MPS I]). ... Full text Open Access Link to item Cite

Sequential myeloablative autologous stem cell transplantation and reduced intensity allogeneic hematopoietic cell transplantation is safe and feasible in children, adolescents and young adults with poor-risk refractory or recurrent Hodgkin and non-Hodgkin lymphoma.

Conference Leukemia · February 2015 The outcome of children, adolescents and young adults (CAYA) with poor-risk recurrent/refractory lymphoma is dismal (⩽30%). To overcome this poor prognosis, we designed an approach to maximize an allogeneic graft vs lymphoma effect in the setting of low di ... Full text Link to item Cite

Brain structural connectivity increases concurrent with functional improvement: evidence from diffusion tensor MRI in children with cerebral palsy during therapy.

Journal Article Neuroimage Clin · 2015 Cerebral Palsy (CP) refers to a heterogeneous group of permanent but non-progressive movement disorders caused by injury to the developing fetal or infant brain (Bax et al., 2005). Because of its serious long-term consequences, effective interventions that ... Full text Open Access Link to item Cite

Progress toward curing HIV infection with hematopoietic cell transplantation.

Journal Article Stem Cells Cloning · 2015 HIV-1 infection afflicts more than 35 million people worldwide, according to 2014 estimates from the World Health Organization. For those individuals who have access to antiretroviral therapy, these drugs can effectively suppress, but not cure, HIV-1 infec ... Full text Open Access Link to item Cite

Human Umbilical Cord Blood Cells Ameliorate Motor Deficits in Rabbits in a Cerebral Palsy Model.

Journal Article Dev Neurosci · 2015 Cerebral palsy (CP) has a significant impact on both patients and society, but therapy is limited. Human umbilical cord blood cells (HUCBC), containing various stem and progenitor cells, have been used to treat various brain genetic conditions. In small an ... Full text Open Access Link to item Cite

Emerging uses of cord blood in regenerative medicine-neurological applications

Chapter · January 1, 2015 Regenerative medicine is dedicated to the study of repairing, replacing, or regenerating damaged human cells, tissues, or organs to restore or establish normal function, and it is predicted to develop into numerous applications to treat a wide variety of c ... Full text Cite

Rapid and efficient generation of transgene-free iPSC from a small volume of cryopreserved blood

Journal Article Stem Cell Reviews and Reports · 2015 © The Author(s) 2015.Human peripheral blood and umbilical cord blood represent attractive sources of cells for reprogramming to induced pluripotent stem cells (iPSCs). However, to date, most of the blood-derived iPSCs were generated using either integratin ... Full text Open Access Cite

Banking or Bankrupting: Strategies for Sustaining the Economic Future of Public Cord Blood Banks.

Journal Article PLoS One · 2015 BACKGROUND: Cord blood is an important source of stem cells. However, nearly 90% of public cord blood banks have declared that they are struggling to maintain their financial sustainability and avoid bankruptcy. The objective of this study is to evaluate h ... Full text Open Access Link to item Cite

Long-term functional outcomes of children with hurler syndrome treated with unrelated umbilical cord blood transplantation.

Chapter · 2015 OBJECTIVES: Hurler syndrome is characterized by progressive multisystem deterioration leading to early death in childhood. This prospective study evaluated the long-term outcomes of patients with Hurler syndrome who underwent umbilical cord blood transplan ... Full text Link to item Cite

Myelodysplastic syndrome evolving from aplastic anemia treated with immunosuppressive therapy: efficacy of hematopoietic stem cell transplantation.

Journal Article Haematologica · December 2014 A proportion of patients with aplastic anemia who are treated with immunosuppressive therapy develop clonal hematologic disorders, including post-aplastic anemia myelodysplastic syndrome. Many will proceed to allogeneic hematopoietic stem cell transplantat ... Full text Open Access Link to item Cite

Multi-site evaluation of the BD Stem Cell Enumeration Kit for CD34(+) cell enumeration on the BD FACSCanto II and BD FACSCalibur flow cytometers.

Journal Article Cytotherapy · November 2014 BACKGROUND AIMS: Evaluation of the BD Stem Cell Enumeration Kit was conducted at four clinical sites with flow cytometry CD34(+) enumeration to assess agreement between two investigational methods: (i) the BD FACSCanto II and BD FACSCalibur systems and (ii ... Full text Open Access Link to item Cite

Outcomes after hematopoietic stem cell transplantation for children with I-cell disease.

Journal Article Biol Blood Marrow Transplant · November 2014 Mucolipidosis type II (MLII), or I-cell disease, is a rare but severe disorder affecting localization of enzymes to the lysosome, generally resulting in death before the 10th birthday. Although hematopoietic stem cell transplantation (HSCT) has been used t ... Full text Open Access Link to item Cite

Umbilical cord blood transplantation to treat Pelizaeus-Merzbacher Disease in 2 young boys.

Journal Article Pediatrics · November 2014 Pelizaeus-Merzbacher Disease (PMD) is a rare X-linked recessive leukodystrophy caused by mutations in the proteolipid protein 1 gene on the Xq22 chromosome. PMD is a dysmyelinating disorder characterized by variable clinical presentation and course. Sympto ... Full text Open Access Link to item Cite

Abstract B22: Altered methylation profiles of imprinted genes in response to prenatal exposure to cigarette smoke in the Newborn Epigenetic STudy (NEST) cohort

Journal Article Cancer Epidemiology, Biomarkers & Prevention · November 1, 2014 AbstractBackground: Early life environmental exposures have adverse consequences on health and development but the mechanisms underlying these effects are unclear. Epigenetic deregulation resulting from ciga ... Full text Cite

One-unit versus two-unit cord-blood transplantation for hematologic cancers.

Journal Article N Engl J Med · October 30, 2014 BACKGROUND: Umbilical-cord blood has been used as the source of hematopoietic stem cells in an estimated 30,000 transplants. The limited number of hematopoietic cells in a single cord-blood unit prevents its use in recipients with larger body mass and resu ... Full text Open Access Link to item Cite

Results of cord blood transplantation in children with nonmalignant hematologic conditions

Chapter · August 12, 2014 This comprehensive volume discusses the current scope of umbilical cord blood transplantation (UCBT), including recent controversies and future developments for improving clinical outcomes. ... Cite

Erythrocyte folate concentrations, CpG methylation at genomically imprinted domains, and birth weight in a multiethnic newborn cohort.

Journal Article Epigenetics · August 2014 Epigenetic mechanisms are proposed to link maternal concentrations of methyl group donor nutrients with the risk of low birth weight. However, empirical data are lacking. We have examined the association between maternal folate and birth weight and assesse ... Full text Open Access Link to item Cite

Dose intensification of methotrexate and cytarabine during intensified continuation chemotherapy for high-risk B-precursor acute lymphoblastic leukemia: POG 9406: a report from the Children's Oncology Group.

Journal Article J Pediatr Hematol Oncol · July 2014 PURPOSE: To determine the efficacy and toxicity of higher dose versus standard dose intravenous methotrexate (MTX) and pulses of high-dose cytosine arabinoside with asparaginase versus standard dose cytosine arabinoside and teniposide during intensified co ... Full text Open Access Link to item Cite

Umbilical cord blood expansion with nicotinamide provides long-term multilineage engraftment.

Journal Article J Clin Invest · July 2014 BACKGROUND: Delayed hematopoietic recovery is a major drawback of umbilical cord blood (UCB) transplantation. Transplantation of ex vivo-expanded UCB shortens time to hematopoietic recovery, but long-term, robust engraftment by the expanded unit has yet to ... Full text Open Access Link to item Cite

Outcome of transplantation for acute lymphoblastic leukemia in children with Down syndrome.

Journal Article Pediatr Blood Cancer · June 2014 We report on 27 patients with Down syndrome (DS) and acute lymphoblastic leukemia (ALL) who received allogeneic hematopoietic cell transplantation (HCT) between 2000 and 2009. Seventy-eight percent of patients received myeloablative conditioning and 52% un ... Full text Open Access Link to item Cite

Feasibility of autologous cord blood cells for infants with hypoxic-ischemic encephalopathy.

Journal Article J Pediatr · May 2014 OBJECTIVE: To assess feasibility and safety of providing autologous umbilical cord blood (UCB) cells to neonates with hypoxic-ischemic encephalopathy (HIE). STUDY DESIGN: We enrolled infants in the intensive care nursery who were cooled for HIE and had ava ... Full text Open Access Link to item Cite

A novel reduced-intensity conditioning regimen for unrelated umbilical cord blood transplantation in children with nonmalignant diseases.

Journal Article Biol Blood Marrow Transplant · March 2014 Reduced-intensity conditioning (RIC) regimens have the potential to decrease transplantation-related morbidity and mortality. However, engraftment failure has been prohibitively high after RIC unrelated umbilical cord blood transplantation (UCBT) in chemot ... Full text Open Access Link to item Cite

In reply.

Journal Article Transfusion · February 2014 Full text Open Access Link to item Cite

Unrelated Umbilical Cord Blood Transplant for Diamond-Blackfan Anemia

Conference Biology of Blood and Marrow Transplantation · February 2014 Full text Cite

Brincidofovir (CMX001) Is Well Tolerated in Highly Immunocompromised Pediatric Patients

Conference Biology of Blood and Marrow Transplantation · February 2014 Full text Cite

Optimizing donor selection for public cord blood banking: influence of maternal, infant, and collection characteristics on cord blood unit quality.

Journal Article Transfusion · February 2014 BACKGROUND: Banked unrelated donor umbilical cord blood (CB) has improved access to hematopoietic stem cell transplantation for patients without a suitably matched donor. In a resource-limited environment, ensuring that the public inventory is enriched wit ... Full text Open Access Link to item Cite

Allogeneic human mesenchymal stem cell therapy (remestemcel-L, Prochymal) as a rescue agent for severe refractory acute graft-versus-host disease in pediatric patients.

Journal Article Biol Blood Marrow Transplant · February 2014 Severe steroid-refractory acute graft-versus-host disease (aGVHD) is related to significant mortality and morbidity after allogeneic stem cell transplantation. Early clinical trials of therapy with human mesenchymal stem cells (hMSCs) in pediatric patients ... Full text Open Access Link to item Cite

Standardization of health care provider competencies for intrathecal access procedures.

Journal Article J Pediatr Oncol Nurs · 2014 INTRODUCTION: This quality improvement (QI) project addresses a method for experienced health care providers to maintain skill-based competence for intrathecal access procedures. METHODS: A prospective QI design using intrathecal access simulation to asses ... Full text Open Access Link to item Cite

Maternal stress, preterm birth, and DNA methylation at imprint regulatory sequences in humans.

Journal Article Genet Epigenet · 2014 In infants exposed to maternal stress in utero, phenotypic plasticity through epigenetic events may mechanistically explain increased risk of preterm birth (PTB), which confers increased risk for neurodevelopmental disorders, cardiovascular disease, and ca ... Full text Open Access Link to item Cite

Neutropenia In Glycogen Storage Disease 1b (GSD1b)

Conference Blood · November 15, 2013 AbstractHypoglycemia, neutropenia, enterocolitis and recurrent infections are common manifestations of glycogen storage disease 1b (GSD1b). GSD1b is a rare genetic disorder caused by mutations in G6PT1, the ... Full text Cite

Analysis of risk factors influencing outcomes after cord blood transplantation in children with juvenile myelomonocytic leukemia: a EUROCORD, EBMT, EWOG-MDS, CIBMTR study.

Journal Article Blood · September 19, 2013 We retrospectively analyzed 110 patients with juvenile myelomonocytic leukemia, given single-unit, unrelated donor umbilical cord blood transplantation. Median age at diagnosis and at transplantation was 1.4 years (age range, 0.1-6.4 years) and 2.2 years ( ... Full text Open Access Link to item Cite

Comparative cost-effectiveness analysis of voriconazole and fluconazole for prevention of invasive fungal infection in patients receiving allogeneic hematopoietic cell transplants.

Journal Article Am J Health Syst Pharm · September 1, 2013 PURPOSE: The cost-effectiveness of voriconazole versus fluconazole prophylaxis against fungal infections in hematopoietic cell transplant (HCT) recipients is investigated. METHODS: A decision-analytic model was developed to estimate the drug costs associat ... Full text Open Access Link to item Cite

Outcomes of allogeneic hematopoietic cell transplantation in patients with dyskeratosis congenita.

Journal Article Biol Blood Marrow Transplant · August 2013 We describe outcomes after allogeneic transplantation in 34 patients with dyskeratosis congenita who underwent transplantation between 1981 and 2009. The median age at transplantation was 13 years (range, 2 to 35). Approximately 50% of transplantations wer ... Full text Open Access Link to item Cite

Rescuing the neonatal brain from hypoxic injury with autologous cord blood.

Journal Article Bone Marrow Transplant · July 2013 Brain injury resulting from perinatal hypoxic-ischemic encephalopathy (HIE) is a major cause of acute mortality in infants and chronic neurologic disability in surviving children. Recent multicenter clinical trials demonstrated the effectiveness of hypothe ... Full text Open Access Link to item Cite

Pre-engraftment syndrome after myeloablative dual umbilical cord blood transplantation: risk factors and response to treatment.

Journal Article Bone Marrow Transplant · July 2013 High fevers and/or rashes prior to neutrophil engraftment are frequently observed after umbilical cord blood (UCB) transplantation, and the condition is referred to as pre-engraftment syndrome (PES). Few studies have evaluated the risk factors for and trea ... Full text Open Access Link to item Cite

Associations between antibiotic exposure during pregnancy, birth weight and aberrant methylation at imprinted genes among offspring.

Journal Article Int J Obes (Lond) · July 2013 OBJECTIVES: Low birth weight (LBW) has been associated with common adult-onset chronic diseases, including obesity, cardiovascular disease, type II diabetes and some cancers. The etiology of LBW is multi-factorial. However, recent evidence suggests exposur ... Full text Open Access Link to item Cite

Outcome of transplantation for acute myelogenous leukemia in children with Down syndrome.

Journal Article Biol Blood Marrow Transplant · June 2013 Data on outcomes of allogeneic transplantation in children with Down syndrome and acute myelogenous leukemia (DS-AML) are scarce and conflicting. Early reports stress treatment-related mortality as the main barrier; a recent case series points to posttrans ... Full text Open Access Link to item Cite

Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning.

Journal Article Blood · May 9, 2013 We report transplantation outcomes of 258 children with Hurler syndrome (HS) after a myeloablative conditioning regimen from 1995 to 2007. Median age at transplant was 16.7 months and median follow-up was 57 months. The cumulative incidence of neutrophil r ... Full text Open Access Link to item Cite

Neurodevelopmental outcomes of umbilical cord blood transplantation in metachromatic leukodystrophy.

Journal Article Biol Blood Marrow Transplant · April 2013 Metachromatic leukodystrophy (MLD) is an inherited demyelinating disease that causes progressive neurologic deterioration, leading to severe motor disability, developmental regression, seizures, blindness, deafness, and death. The disease presents as a lat ... Full text Open Access Link to item Cite

Hematopoietic cell transplantation with cord blood for cure of HIV infections.

Journal Article Biol Blood Marrow Transplant · March 2013 Hematopoietic cell transplantation (HCT) using CCR5-Δ32/Δ32 stem cells from an adult donor has resulted in the only known cure of human immunodeficiency virus (HIV) infection. However, it is not feasible to repeat this procedure except rarely because of th ... Full text Open Access Link to item Cite

Paternal obesity is associated with IGF2 hypomethylation in newborns: results from a Newborn Epigenetics Study (NEST) cohort.

Journal Article BMC Med · February 6, 2013 BACKGROUND: Data from epidemiological and animal model studies suggest that nutrition during pregnancy may affect the health status of subsequent generations. These transgenerational effects are now being explained by disruptions at the level of the epigen ... Full text Open Access Link to item Cite

Impact of selection of cord blood units from the United States and swiss registries on the cost of banking operations.

Journal Article Transfus Med Hemother · February 2013 BACKGROUND: Over the last 2 decades, cord blood (CB) has become an important source of blood stem cells. Clinical experience has shown that CB is a viable source for blood stem cells in the field of unrelated hematopoietic blood stem cell transplantation. ... Full text Open Access Link to item Cite

Why Do Umbilical Cord Blood Units Fail to Qualify for Public Banking?

Conference Biology of Blood and Marrow Transplantation · February 2013 Full text Cite

Associations between birth and one year anthropometric measurements and IGF2 and IGF2R genetic variants in African American and Caucasian American infants.

Journal Article J Pediatr Genet · 2013 Insulin-like growth factor 2 receptor (IGF2R) and insulin-like growth factor 2 (IGF2) genetic variants have been inconsistently associated with low birth weight and birth length in Caucasian and Asian infants, however few studies have included African Amer ... Full text Open Access Link to item Cite

Extensive haplotype diversity in African American mothers and their cord blood units.

Journal Article Tissue Antigens · January 2013 HLA-A, -B, -C, -DRB1, -DQB1 assignments were obtained for 374 pairs of African American mothers and their umbilical cord blood units (CBU) by DNA sequencing. An algorithm developed by the National Marrow Donor Program was used to assign 1122 haplotypes by ... Full text Open Access Link to item Cite

Diffuse reduction of white matter connectivity in cerebral palsy with specific vulnerability of long range fiber tracts.

Journal Article Neuroimage Clin · 2013 Cerebral palsy (CP) is a heterogeneous group of non-progressive motor disorders caused by injury to the developing fetal or infant brain. Although the defining feature of CP is motor impairment, numerous other neurodevelopmental disabilities are associated ... Full text Open Access Link to item Cite

Maternal BMI, IGF-I Levels, and Birth Weight in African American and White Infants.

Journal Article Int J Pediatr · 2013 At birth, elevated IGF-I levels have been linked to birth weight extremes; high birth weight and low birth weight are risk factors for adult-onset chronic diseases including obesity, cardiovascular disease, and type 2 diabetes. We examined associations bet ... Full text Open Access Link to item Cite

Associations between antibiotic exposure during pregnancy, birth weight and aberrant methylation at imprinted genes among offspring

Journal Article International Journal of Obesity · 2013 Objectives: Low birth weight (LBW) has been associated with common adult-onset chronic diseases, including obesity, cardiovascular disease, type II diabetes and some cancers. The etiology of LBW is multi-factorial. However, recent evidence suggests exposur ... Full text Open Access Cite

Effect of HLA-matching recipients to donor noninherited maternal antigens on outcomes after mismatched umbilical cord blood transplantation for hematologic malignancy.

Journal Article Biol Blood Marrow Transplant · December 2012 Transplantation-related mortality (TRM) is high after HLA-mismatched umbilical cord blood (UCB) transplantation (UCBT). In utero, exposure to noninherited maternal antigen (NIMA) is recognized by the fetus, which induces T regulator cells to that haplotype ... Full text Open Access Link to item Cite

Long Term Outcomes of Hematopoietic Stem Cell Transplantation in Patients with Severe Phenotype Hurler Syndrome: an International Multi-Center Study

Conference Blood · November 16, 2012 AbstractAbstract 1958Background:Hurler syndrome (HS), the most severe phenotype in the spectrum of Mu ... Full text Cite

Outcome of Transplantation for Acute Leukemia in Down Syndrome

Conference Blood · November 16, 2012 AbstractAbstract 1991Children with Down syndrome (DS) have a 10-to 20-fold increased incidence of acute lymphoblastic (ALL) and acute myeloid leukemia (AML) compared to the o ... Full text Cite

103-P

Conference Human Immunology · October 2012 Full text Cite

Myeloablative transplantation using either cord blood or bone marrow leads to immune recovery, high long-term donor chimerism and excellent survival in chronic granulomatous disease.

Journal Article Biol Blood Marrow Transplant · September 2012 The curative potential of hematopoietic stem cell transplantation in patients with chronic granulomatous disease depends on availability of a suitable donor, successful donor engraftment, and maintenance of long-term donor chimerism. Twelve consecutive chi ... Full text Open Access Link to item Cite

Long-term survival and late deaths after hematopoietic cell transplantation for primary immunodeficiency diseases and inborn errors of metabolism.

Journal Article Biol Blood Marrow Transplant · September 2012 It is uncertain whether late mortality rates after hematopoietic cell transplantation for severe combined immunodeficiency (SCID), non-SCID primary immunodeficiency diseases (non-SCID PIDD), and inborn errors of metabolism (IEM) return to rates observed in ... Full text Open Access Link to item Cite

Insulin-like growth factor 2/H19 methylation at birth and risk of overweight and obesity in children.

Journal Article J Pediatr · July 2012 OBJECTIVE: To determine whether aberrant DNA methylation at differentially methylated regions (DMRs) regulating insulin-like growth factor 2 (IGF2) expression in umbilical cord blood is associated with overweight or obesity in a multiethnic cohort. STUDY D ... Full text Open Access Link to item Cite

Depression in pregnancy, infant birth weight and DNA methylation of imprint regulatory elements.

Journal Article Epigenetics · July 2012 Depressed mood in pregnancy has been linked to low birth weight (LBW, < 2,500 g), a risk factor for adult-onset chronic diseases in offspring. We examined maternal depressed mood in relation to birth weight and evaluated the role of DNA methylation at regu ... Full text Open Access Link to item Cite

Relationship of race/ethnicity and survival after single umbilical cord blood transplantation for adults and children with leukemia and myelodysplastic syndromes.

Journal Article Biol Blood Marrow Transplant · June 2012 The relationship of race/ethnicity with outcomes of umbilical cord blood transplantation (UCBT) is not well known. We analyzed the association between race/ethnicity and outcomes of unrelated single UCBT for leukemia and myelodysplastic syndromes. Our retr ... Full text Open Access Link to item Cite

Safety and efficacy of CMX001 as salvage therapy for severe adenovirus infections in immunocompromised patients.

Journal Article Biol Blood Marrow Transplant · May 2012 No therapeutic agent has yet been established as the definitive therapy for adenovirus infections. We describe the clinical experience of 13 immunocompromised patients who received CMX001 (hexadecyloxypropyl cidofovir), an orally bioavailable lipid conjuga ... Full text Open Access Link to item Cite

Association of cord blood methylation fractions at imprinted insulin-like growth factor 2 (IGF2), plasma IGF2, and birth weight.

Journal Article Cancer Causes Control · April 2012 PURPOSE: Altered methylation at Insulin-like Growth Factor 2 (IGF2) regulatory regions has previously been associated with obesity, and several malignancies including colon, esophageal, and prostate adenocarcinomas, presumably via changes in expression and ... Full text Open Access Link to item Cite

Allogeneic hematopoietic SCT for alpha-mannosidosis: an analysis of 17 patients.

Journal Article Bone Marrow Transplant · March 2012 Alpha-mannosidosis is a rare lysosomal storage disease. Hematopoietic SCT (HSCT) is usually recommended as a therapeutic option though reports are anecdotal to date. This retrospective multi institutional analysis describes 17 patients that were diagnosed ... Full text Open Access Link to item Cite

National Cancer Institute, National Heart, Lung and Blood Institute/Pediatric Blood and Marrow Transplantation Consortium First International Consensus Conference on late effects after pediatric hematopoietic cell transplantation: the need for pediatric-specific long-term follow-up guidelines.

Journal Article Biol Blood Marrow Transplant · March 2012 Existing standards for screening and management of late effects occurring in children who have undergone hematopoietic cell transplantation (HCT) include recommendations from pediatric cancer networks and consensus guidelines from adult-oriented transplant ... Full text Open Access Link to item Cite

Building a Legacy

Conference Biology of Blood and Marrow Transplantation · February 2012 Full text Cite

The Cord Blood Apgar: a novel scoring system to optimize selection of banked cord blood grafts for transplantation (CME).

Journal Article Transfusion · February 2012 BACKGROUND: Engraftment failure and delays, likely due to diminished cord blood unit (CBU) potency, remain major barriers to the overall success of unrelated umbilical cord blood transplantation (UCBT). To address this problem, we developed and retrospecti ... Full text Open Access Link to item Cite

Unrelated Cord Blood Transplantation (CBT) of 101 Hemoglobinopathy (HGB) Patients

Conference Biology of Blood and Marrow Transplantation · February 2012 Full text Cite

Cord Blood Transplantation for Long Term Management or Possible Cure of HIV Infection

Conference Biology of Blood and Marrow Transplantation · February 2012 Full text Cite

Polyethylene Glycol-conjugated L-asparaginase versus native L-asparaginase in combination with standard agents for children with acute lymphoblastic leukemia in second bone marrow relapse: a Children's Oncology Group Study (POG 8866).

Journal Article J Pediatr Hematol Oncol · December 2011 BACKGROUND: Administration of L-asparaginase is limited by hypersensitivity reactions mediated by anti-asparaginase antibodies. To overcome this problem, native Escherichia coli L-asparaginase was conjugated to polyethylene glycol (PEG) to formulate PEG-L- ... Full text Open Access Link to item Cite

Family-directed umbilical cord blood banking.

Journal Article Haematologica · November 2011 Umbilical cord blood transplantation from HLA-identical siblings provides good results in children. These results support targeted efforts to bank family cord blood units that can be used for a sibling diagnosed with a disease which can be cured by allogen ... Full text Open Access Link to item Cite

The effects of depression and use of antidepressive medicines during pregnancy on the methylation status of the IGF2 imprinted control regions in the offspring.

Journal Article Clin Epigenetics · October 26, 2011 In utero exposures to environmental factors may result in persistent epigenetic modifications affecting normal development and susceptibility to chronic diseases in later life. We explored the relationship between exposure of the growing fetus to maternal ... Full text Open Access Link to item Cite

Umbilical cord blood: a guide for primary care physicians.

Journal Article Am Fam Physician · September 15, 2011 Umbilical cord blood stem cell transplants are used to treat a variety of oncologic, genetic, hematologic, and immunodeficiency disorders. Physicians have an important role in educating, counseling, and offering umbilical cord blood donation and storage op ... Open Access Link to item Cite

Partial splenectomy but not total splenectomy preserves immunoglobulin M memory B cells in mice.

Journal Article J Pediatr Surg · September 2011 PURPOSE: The mechanism by which partial splenectomy preserves splenic immune function is unknown. Immunoglobulin (Ig) M memory B cells are critical for the immune response against encapsulated bacteria and are reduced in asplenic patients, although it is u ... Full text Open Access Link to item Cite

Total colony-forming units are a strong, independent predictor of neutrophil and platelet engraftment after unrelated umbilical cord blood transplantation: a single-center analysis of 435 cord blood transplants.

Journal Article Biol Blood Marrow Transplant · September 2011 Graft failure occurs in approximately 20% of patients after unrelated umbilical cord blood transplantation (UCBT). This could be because of inadequate potency of the cord blood unit (CBU). To this end, we investigated the impact of graft characteristics on ... Full text Open Access Link to item Cite

Umbilical cord blood transplantation for children with thalassemia and sickle cell disease.

Journal Article Biol Blood Marrow Transplant · September 2011 We examined the efficacy of unrelated cord blood (CB) transplantation in children with thalassemia (n = 35) and sickle cell disease (n = 16), using data reported to 3 registries. Donor-recipient pairs were matched at HLA-A and -B (antigen level) and DRB1 ( ... Full text Open Access Link to item Cite

Guidelines for the development and validation of new potency assays for the evaluation of umbilical cord blood.

Journal Article Cytotherapy · August 2011 The following commentary was developed by the National Marrow Donor Program Cord Blood Advisory Group and is intended to provide an overview of umbilical cord blood (UCB) processing, summarize the current state of potency assays used to characterize UCB, a ... Full text Open Access Link to item Cite

Methylation variation at IGF2 differentially methylated regions and maternal folic acid use before and during pregnancy.

Journal Article Epigenetics · July 2011 Folic acid (FA) supplementation before and during pregnancy has been associated with decreased risk of neural tube defects although recent reports suggest it may also increase the risk of other chronic diseases. We evaluated exposure to maternal FA supplem ... Full text Open Access Link to item Cite

Isolation and expansion of oligodendrocyte progenitor cells from cryopreserved human umbilical cord blood.

Journal Article Cytotherapy · July 2011 BACKGROUND AIMS: Oligodendrocyte precursor cells (OPC) hold promise as a cellular therapy for demyelinating diseases. The feasibility of using OPC-based therapies in humans depends upon a reliable, readily available source. We have previously described the ... Full text Open Access Link to item Cite

Allogeneic transplantation for patients with high-risk or refractory neuroblastoma.

Journal Article J Clin Oncol · May 20, 2011 9552 Background: While the survival of high-risk neuroblastoma patients has improved with myeloablative chemotherapy (MAC) and 13-cis retinoic acid, 60% of children fail this approach, thus other strategies are needed. As a potential platform for future im ... Link to item Cite

Efficacy and safety of ex vivo cultured adult human mesenchymal stem cells (Prochymal™) in pediatric patients with severe refractory acute graft-versus-host disease in a compassionate use study.

Journal Article Biol Blood Marrow Transplant · April 2011 Preliminary studies using directed-donor ex vivo expanded human mesenchymal stem cells (hMSCs) have shown promise in the treatment of acute graft-versus-host disease (aGVHD). However, their production is cumbersome and standardization is difficult. We desc ... Full text Open Access Link to item Cite

Quality of Life Project – An Oral Presentation

Conference Biology of Blood and Marrow Transplantation · February 2011 Full text Cite

Fast Facts Flip Chart – A Quick Accessible Reference Guide for Busy Nurses

Conference Biology of Blood and Marrow Transplantation · February 2011 Full text Cite

Vitamin D Deficiency in Children Undergoing Hematopoietic Stem Cell Transplantation

Conference Biology of Blood and Marrow Transplantation · February 2011 Full text Cite

MPS I: Age at transplant is associated with better long-term developmental outcomes

Conference Molecular Genetics and Metabolism · February 2011 Full text Cite

MPS II: developmental outcomes after hematopoietic stem cell transplantation

Conference Molecular Genetics and Metabolism · February 2011 Full text Cite

Folic acid supplementation before and during pregnancy in the Newborn Epigenetics STudy (NEST).

Journal Article BMC Public Health · January 21, 2011 BACKGROUND: Folic acid (FA) added to foods during fortification is 70-85% bioavailable compared to 50% of folate occurring naturally in foods. Thus, if FA supplements also are taken during pregnancy, both mother and fetus can be exposed to FA exceeding the ... Full text Open Access Link to item Cite

Influence of nucleated cell dose on overall survival of unrelated cord blood transplantation for patients with severe acquired aplastic anemia: a study by eurocord and the aplastic anemia working party of the European group for blood and marrow transplantation.

Journal Article Biol Blood Marrow Transplant · January 2011 Information is scarce on outcomes after unrelated cord blood transplantation (UCBT) for patients with severe aplastic anemia (SAA). We retrospectively analyzed 71 patients (median age, 13 years; 28 adults) with SAA (9 with paroxysmal nocturnal hemoglobinur ... Full text Open Access Link to item Cite

Randomized, double-blind trial of fluconazole versus voriconazole for prevention of invasive fungal infection after allogeneic hematopoietic cell transplantation.

Journal Article Blood · December 9, 2010 Invasive fungal infection (IFI) is a serious threat after allogeneic hematopoietic cell transplant (HCT). This multicenter, randomized, double-blind trial compared fluconazole (N = 295) versus voriconazole (N = 305) for the prevention of IFI in the context ... Full text Open Access Link to item Cite

Clinical outcome of cerebrospinal fluid shunting for communicating hydrocephalus in mucopolysaccharidoses I, II, and III: a retrospective analysis of 13 patients.

Journal Article Neurosurgery · December 2010 BACKGROUND: Intracranial pathology is a well-documented feature of mucopolysaccharidoses (MPSs), including communicating hydrocephalus (CH). Neither the success nor the complications of cerebrospinal fluid shunting in MPS patients have been well documented ... Full text Open Access Link to item Cite

Hematopoietic reconstitution in a patient with Fanconi's anemia by means of umbilical-cord blood from an HLA-identical sibling

Journal Article Cellular Therapy and Transplantation · December 1, 2010 The clinical manifestations of Fanconi's anemia, an autosomal recessive disorder, include progressive pancyto-penia, a predisposition to neoplasia, and nonhematopoietic developmental anomalies [1-3]. Hypersensitivity to the clastogenic effect of DNA-cross- ... Full text Open Access Cite

Differences in quality between privately and publicly banked umbilical cord blood units: a pilot study of autologous cord blood infusion in children with acquired neurologic disorders.

Journal Article Transfusion · September 2010 BACKGROUND: A pilot study was conducted to determine the safety and feasibility of intravenous administration of autologous umbilical cord blood (CB) in young children with acquired neurologic disorders. Most CB units (CBUs) were electively stored in priva ... Full text Open Access Link to item Cite

Donor cell leukemia in umbilical cord blood transplant patients: a case study and literature review highlighting the importance of molecular engraftment analysis.

Journal Article J Mol Diagn · July 2010 Donor cell neoplasms are rare complications of treatment regimens that involve stem cell transplantation for hematological malignancies, myelodysplastic processes, or certain genetic or metabolic disorders. We report a case of donor cell leukemia in a pedi ... Full text Open Access Link to item Cite

Defibrotide for the treatment of severe hepatic veno-occlusive disease and multiorgan failure after stem cell transplantation: a multicenter, randomized, dose-finding trial.

Journal Article Biol Blood Marrow Transplant · July 2010 Therapeutic options for severe hepatic veno-occlusive disease (VOD) are limited and outcomes are dismal, but early phase I/II studies have suggested promising activity and acceptable toxicity using the novel polydisperse oligonucleotide defibrotide. This r ... Full text Open Access Link to item Cite

Inefficient dystrophin expression after cord blood transplantation in Duchenne muscular dystrophy.

Journal Article Muscle Nerve · June 2010 We report a boy who received two allogeneic stem cell transplantations from umbilical cord donors to treat chronic granulomatous disease (CGD). The CGD was cured after the second transplantation, but 2.5 years later he was diagnosed with Duchenne muscular ... Full text Open Access Link to item Cite

Splenectomy and partial splenectomy improve hematopoietic stem cell engraftment in hypersplenic mice.

Journal Article J Pediatr Surg · June 2010 BACKGROUND: Hematopoietic stem cell (HSC) engraftment is delayed after transplantation in children with hypersplenism, increasing the morbidity and costs of care. Preliminary clinical data suggest that splenectomy before HSC transplantation may improve HSC ... Full text Open Access Link to item Cite

OUTCOMES OF UNRELATED CORD BLOOD TRANSPLANT IN PATIENTS WITH HEMOGLOBINOPATHIES

Conference HAEMATOLOGICA-THE HEMATOLOGY JOURNAL · June 1, 2010 Link to item Cite

Stem cell source and outcome after hematopoietic stem cell transplantation (HSCT) in children and adolescents with acute leukemia.

Journal Article Pediatr Clin North Am · February 2010 Allogeneic hematopoietic stem cell transplantation from siblings, unrelated donors or HLA mismatched family members has become an important procedure to offer a chance of cure to children and adolescents with acute leukemia at high risk of relapse and thos ... Full text Link to item Cite

Cord blood and bone marrow transplantation in inherited metabolic diseases: scientific basis, current status and future directions.

Journal Article Br J Haematol · February 2010 Progressive degeneration of the central nervous system leading to the loss of neuromotor, neurophysiological and cognitive abilities is the fundamental clinical problem in patients with many inherited metabolic diseases (IMD). Worldwide experience shows th ... Full text Link to item Cite

Umbilical cord blood transplantation

Chapter · January 1, 2010 Overview. Over the past several decades, hematopoietic stem cell transplantation has emerged as an effective approach to curative therapy for both pediatric and adult patients with aggressive or recurrent malignancies, congenital immunodeficiency diseases, ... Full text Cite

Transplant outcomes in mucopolysaccharidoses.

Journal Article Semin Hematol · January 2010 The mucopolysaccharidoses (MPSs) are inherited metabolic disorders (IMDs) caused by single-gene defects leading to progressive cellular accumulation of glycosaminoglycans (GAGs) and damage to multiple organs, including the central nervous, musculoskeletal, ... Full text Link to item Cite

70: Epigenetic orchestration of circulating insulin-like growth factor-2 levels in cord blood

Conference American Journal of Obstetrics and Gynecology · December 2009 Full text Cite

Umbilical cord blood transplantation for non-malignant diseases.

Journal Article Bone Marrow Transplant · November 2009 Many factors, including lower risk of GVHD, rapid availability of 4/6-6/6 matched cord blood (CB) units and incremental gains in the outcomes, have led to an increasing use of CB transplantation (CBT) to treat many patients who lack fully matched adult BM ... Full text Link to item Cite

Unrelated donor umbilical cord blood transplantation in pediatric myelodysplastic syndrome: a single-center experience.

Journal Article Biol Blood Marrow Transplant · August 2009 Myelodysplastic syndromes (MDS) respond poorly to chemotherapy. Between 1995 and 2006, 23 pediatric patients with MDS were transplanted with unrelated donor umbilical cord blood (UUCB) at our center. The median age was 11.1 years (range: 1.1-19.7), median ... Full text Link to item Cite

Umbilical cord stem cell transplantation.

Journal Article Semin Oncol Nurs · May 2009 OBJECTIVES: To discuss the history, current state, and future directions of umbilical cord blood (UCB) transplantation as it relates to the emerging field of cellular therapies. DATA SOURCES: Research studies, articles, and personal experiences. CONCLUSION ... Full text Link to item Cite

Risk factor analysis of outcomes after unrelated cord blood transplantation in patients with hurler syndrome.

Journal Article Biol Blood Marrow Transplant · May 2009 Allogeneic stem cell transplantation (SCT) is considered effective in preventing disease progression in patients with Hurler syndrome (HS). Unrelated umbilical cord blood (UCB) grafts are suggested as an alternative to bone marrow (BM) or peripheral blood ... Full text Link to item Cite

Diffusion tensor imaging detects abnormalities in the corticospinal tracts of neonates with infantile Krabbe disease.

Journal Article AJNR Am J Neuroradiol · May 2009 BACKGROUND AND PURPOSE: It is not possible to determine if neonates diagnosed with Krabbe disease through statewide neonate screening programs will develop the disease as infants, juveniles, or adults. The only available treatment for this fatal neurodegen ... Full text Link to item Cite

Unintended changes in cognition, mood, and behavior arising from cell-based interventions for neurological conditions: ethical challenges.

Journal Article Am J Bioeth · May 2009 The prospect of using cell-based interventions (CBIs) to treat neurological conditions raises several important ethical and policy questions. In this target article, we focus on issues related to the unique constellation of traits that characterize CBIs ta ... Full text Link to item Cite

Newborn screening for Krabbe disease: the New York State model.

Journal Article Pediatr Neurol · April 2009 Krabbe disease is a rare inherited neurologic disorder affecting the central and peripheral nervous systems. The disease has four phenotypes: early infantile, later onset, adolescent, and adult. The only known treatment is hematopoietic stem cell transplan ... Full text Link to item Cite

Isolation and Expansion of Oligodendrocytes from Thawed, Cryopreserved Human Umblilical Cord Blood

Journal Article Biology of Blood and Marrow Transplantation · February 2009 Full text Cite

Update on umbilical cord blood transplantation.

Journal Article Curr Opin Pediatr · February 2009 PURPOSE OF REVIEW: 2008 marks the 20th anniversary of the first use of umbilical cord blood (UCB) as a source of donor cells for hematopoietic stem cell transplantation. In those early days, there was great doubt and skepticism about the utility of UCB as ... Full text Link to item Cite

Unrelated Umbilical Cord Blood Transplantation is an Effective Therapy for Wiskott-Aldrich Syndrome

Journal Article Biology of Blood and Marrow Transplantation · February 2009 Full text Cite

The role of animal models in evaluating reasonable safety and efficacy for human trials of cell-based interventions for neurologic conditions.

Journal Article J Cereb Blood Flow Metab · January 2009 Progress in regenerative medicine seems likely to produce new treatments for neurologic conditions that use human cells as therapeutic agents; at least one trial for such an intervention is already under way. The development of cell-based interventions for ... Full text Link to item Cite

Correlation of neurodevelopmental features and MRI findings in infantile Krabbe's disease.

Journal Article AJR Am J Roentgenol · January 2009 OBJECTIVE: The purpose of our study was to compare MRI findings with neurobehavioral development in infants with Krabbe's disease. MATERIALS AND METHODS: Nine infants with Krabbe's disease underwent a total of 19 MR studies during the first year of life as ... Full text Link to item Cite

Results of the Cord Blood Transplantation Study (COBLT): clinical outcomes of unrelated donor umbilical cord blood transplantation in pediatric patients with hematologic malignancies.

Journal Article Blood · November 15, 2008 Outcomes of unrelated donor cord blood transplantation in 191 hematologic malignancy children (median age, 7.7 years; median weight, 25.9 kg) enrolled between 1999 and 2003 were studied (median follow-up, 27.4 months) in a prospective phase 2 multicenter t ... Full text Link to item Cite

Unrelated donor umbilical cord blood transplantation for inherited metabolic disorders in 159 pediatric patients from a single center: influence of cellular composition of the graft on transplantation outcomes.

Journal Article Blood · October 1, 2008 Outcomes of 159 young patients with inherited metabolic disorders (IMDs) undergoing transplantation with partially HLA-mismatched unrelated donor umbilical cord blood were studied to investigate the impact of graft and patient characteristics on engraftmen ... Full text Link to item Cite

Posttransplant autoimmune hemolytic anemia and other autoimmune cytopenias are increased in very young infants undergoing unrelated donor umbilical cord blood transplantation.

Journal Article Biol Blood Marrow Transplant · October 2008 Autoimmune cytopenias are a recognized complication of hematopoietic stem cell transplant (HSCT), and are considered to be a feature of chronic graft-versus-host disease (cGVHD). We report on a cohort of very young infants (< or =3 months of age) receiving ... Full text Link to item Cite

Efficacy of high-dose chemotherapy or standard salvage therapy in patients with recurrent medulloblastoma.

Journal Article Neuro Oncol · October 2008 The efficacy of high-dose chemotherapy (HDC) or standard salvage therapy was evaluated in patients with recurrent medulloblastoma (MBL) using retrospective chart review of all patients with recurrent MBL treated at Duke University Medical Center between 19 ... Full text Link to item Cite

Cell-based interventions for neurologic conditions: ethical challenges for early human trials.

Journal Article Neurology · July 22, 2008 BACKGROUND: Attempts to translate basic stem cell research into treatments for neurologic diseases and injury are well under way. With a clinical trial for one such treatment approved and in progress in the United States, and additional proposals under rev ... Full text Link to item Cite

Isolation of oligodendrocyte-like cells from human umbilical cord blood.

Journal Article Cytotherapy · June 2008 Background As human umbilical cord blood (UCB) is known to be a rich source of progenitor cells, the prospect of isolating a subset of these cells that could differentiate into cells of non-hematopoietic lineages suggests a therapeutic use for patients wit ... Cite

A prospective longitudinal multicenter study of coagulation in pediatric patients undergoing allogeneic stem cell transplantation.

Journal Article Pediatr Blood Cancer · June 2008 BACKGROUND: Thrombotic complications occur in adult patients undergoing stem cell transplantation (SCT), especially following high dose chemo-radiotherapy. There is little published information in children on the impact of SCT on coagulation, as well as po ... Full text Link to item Cite

Prospective analysis of TEL gene rearrangements in childhood acute lymphoblastic leukemia: a Children's Oncology Group study.

Journal Article J Clin Oncol · May 1, 2008 PURPOSE: To prospectively determine the prognostic significance of the TEL-AML1 fusion in children with acute lymphoblastic leukemia (ALL). PATIENTS AND METHODS: TEL gene status was determined for 926 patients with B-precursor ALL enrolled on the Pediatric ... Full text Link to item Cite

Indications and donor selections for allogeneic stem cell transplantation in children with hematologic malignancies.

Journal Article Pediatr Clin North Am · February 2008 Allogeneic stem cell transplantation (SCT) is the only curative approach for many patients with advanced or high-risk leukemia. Advances in supportive care and management of graft-versus-host disease have resulted in improvements in outcomes of related and ... Full text Link to item Cite

439: Pass it on

Conference Biology of Blood and Marrow Transplantation · February 2008 Full text Cite

UNRELATED DONOR UMBILICAL CORD BLOOD TRANSPLANT FOR HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS (HLH)

Conference BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION · February 1, 2008 Link to item Cite

Emerging trends in transplantation of inherited metabolic diseases.

Journal Article Bone Marrow Transplant · January 2008 Allogeneic hematopoietic stem cell transplantation (HSCT) can prolong life and improve its quality in patients with inherited metabolic diseases. HSCT offers a permanent source of enzyme replacement therapy and also might mediate nonhematopoietic cell rege ... Full text Link to item Cite

Isolation of oligodendrocyte-like cells from human umbilical cord blood.

Journal Article Cytotherapy · 2008 BACKGROUND: As human umbilical cord blood (UCB) is known to be a rich source of progenitor cells, the prospect of isolating a subset of these cells that could differentiate into cells of non-hematopoietic lineages suggests a therapeutic use for patients wi ... Full text Link to item Cite

Correction of chronic granulomatous disease after second unrelated-donor umbilical cord blood transplantation.

Journal Article Pediatr Blood Cancer · December 2007 Allogeneic hematopoietic stem cell transplantation (HSCT) is curative for chronic granulomatous disease (CGD), but many patients lack a suitably matched related donor. We report successful outcomes after mismatched, unrelated-donor umbilical cord blood tra ... Full text Link to item Cite

Analysis of the Cellular Components of the Graft and Clinical Characteristics of 159 Children with Lysosomal and Peroxisomal Disorders (LSD) Undergoing Unrelated Umbilical Cord Blood Transplantation at a Single Center.

Conference Blood · November 16, 2007 AbstractBackground: Allogeneic bone marrow transplantation from related and unrelated donors leads to improvement in longevity and quality of life due to replacement of missing enzyme and engraftment of dono ... Full text Cite

Risk Factor Analysis of Outcomes after Unrelated Cord Blood Transplantation for Children with Hurler’s Syndrome. An Eurocord-Duke University Collaborative Study.

Conference Blood · November 16, 2007 AbstractHurler’s syndrome (HS), the most severe form of mucopolysaccharidosis type-I causes progressive deterioration of the central nervous system and death in childhood. Allogeneic-stem cell transplantatio ... Full text Cite

Post Transplant Autoimmune Hemolytic Anemia and Other Cytopenias Are Increased in Very Young Babies after Unrelated Donor Umbilical Cord Blood Transplantation.

Conference Blood · November 16, 2007 AbstractBackground: Unrelated donor umbilical cord blood transplantation (UCBT) is beneficial in the treatment of very young babies with early infantile, lysosomal storage diseases and hemoglobinopathies, di ... Full text Cite

Use of Mesenchymal Stem Cells To Treat Pediatric Patients with Severe (Grade III–IV) Acute Graft Versus Host Disease Refractory to Steroid and Other Agents on a Compassionate Use Basis.

Conference Blood · November 16, 2007 AbstractBackground: Severe acute Graft versus Host Disease (aGvHD) refractory to steroids and other immunosuppressive agents carries a high mortality and very poor prognosis. Preliminary studies have shown t ... Full text Cite

Post Thaw Colony Forming Units (CFU) Is a Strong Independent Predictor of Engraftment after Unrelated Donor Umbilical Cord Blood Transplantation (UCBT).

Conference Blood · November 16, 2007 AbstractBackground: Unrelated donor umbilical cord blood is an acceptable graft source for patients lacking related donors. However, a non-engraftment rate of approximately 20% despite adequate total nucleat ... Full text Cite

Ethical and policy issues relating to progenitor-cell-based strategies for prevention of atherosclerosis.

Journal Article J Med Ethics · November 2007 OBJECTIVE: To examine important ethical and societal issues relating to the use of progenitor-cell-based strategies for disease prevention, particularly atherosclerosis. BACKGROUND: Several nascent lines of evidence suggest the feasibility of using progeni ... Full text Link to item Cite

Unrelated cord blood transplantation in children with sickle cell disease: review of four-center experience.

Journal Article Pediatr Transplant · September 2007 UCBT was performed in seven children with SCD and stroke (HLA match 4/6 n=5; 5/6 n=2). Four received myeloablative regimens (BU, CY, ATG plus FLU in one patient). One had primary graft failure, three had sustained engraftment, two with grade III-IV GVHD (o ... Full text Link to item Cite

Results of unrelated cord blood transplant in fanconi anemia patients: risk factor analysis for engraftment and survival.

Journal Article Biol Blood Marrow Transplant · September 2007 We retrospectively analyzed results of unrelated cord blood transplantation (UCBT) in 93 Fanconi anemia (FA) patients. Median age at transplantation was 8.6 years (1-45). The units transplanted were HLA-A, -B, or -DRB1 identical in 12 cases, 1 HLA mismatch ... Full text Link to item Cite

Allergic reactions to E. coli L-asparaginase do not affect outcome in childhood B-precursor acute lymphoblastic leukemia: a Children's Oncology Group Study.

Journal Article J Pediatr Hematol Oncol · September 2007 We describe the outcome of children with B-precursor acute lymphoblastic leukemia registered on Pediatric Oncology Group 8602 who switched to Erwinia asparaginase (ASP) due to an allergy to the Escherichia coli product. Between February 1986 and January 19 ... Full text Link to item Cite

Antimetabolite therapy for lesser-risk B-lineage acute lymphoblastic leukemia of childhood: a report from Children's Oncology Group Study P9201.

Journal Article Blood · August 15, 2007 Pediatric Oncology Group (POG) protocol 9201 enrolled children with lesser-risk B-lineage acute lymphoblastic leukemia (ALL) defined by age (1-9), white blood cell count (WBC) less than 50 x 10(9)/L (50,000/microL), DNA findings of trisomies 4 and 10 (or D ... Full text Link to item Cite

Outcomes of transplantation of unrelated donor umbilical cord blood and bone marrow in children with acute leukaemia: a comparison study.

Journal Article Lancet · June 9, 2007 BACKGROUND: Although umbilical cord blood is an accepted alternative to bone marrow for transplantation, allele-matched bone marrow is generally regarded as the preferred graft source. Our aim was to assess leukaemia-free survival after transplantations of ... Full text Link to item Cite

Intensified PEG-L-asparaginase and antimetabolite-based therapy for treatment of higher risk precursor-B acute lymphoblastic leukemia: a report from the Children's Oncology Group.

Journal Article J Pediatr Hematol Oncol · June 2007 The Pediatric Oncology Group 9203 pilot protocol was designed to determine the feasibility of delivering 29 biweekly doses of polyethylene glycol (PEG)-L-asparaginase, on a backbone of intensive multiagent antimetabolite-based consolidation and maintenance ... Full text Link to item Cite

Outcomes of unrelated umbilical cord blood transplantation for X-linked adrenoleukodystrophy.

Journal Article Biol Blood Marrow Transplant · June 2007 Adrenoleukodystrophy (ALD) is an X-linked disorder caused by a defect in the metabolism of long chain fatty acids leading to demyelination, neurodegeneration, and death. The disease typically presents in young boys and adolescent boys. Allogeneic bone marr ... Full text Link to item Cite

Comparison of the Digene Hybrid Capture System Cytomegalovirus (CMV) DNA (version 2.0), Roche CMV UL54 analyte-specific reagent, and QIAGEN RealArt CMV LightCycler PCR reagent tests using AcroMetrix OptiQuant CMV DNA quantification panels and specimens from allogeneic-stem-cell transplant recipients.

Journal Article J Clin Microbiol · June 2007 The Digene Hybrid Capture system cytomegalovirus (CMV) DNA (version 2.0), Roche CMV UL54 analyte-specific reagent, and QIAGEN RealArt CMV LightCycler PCR reagent tests were compared using whole-virus standards and plasma specimens collected from allogeneic ... Full text Link to item Cite

Effective asparagine depletion with pegylated asparaginase results in improved outcomes in adult acute lymphoblastic leukemia: Cancer and Leukemia Group B Study 9511.

Journal Article Blood · May 15, 2007 CALGB 9511 used pegaspargase (PEG-ASP) in lieu of the native enzyme. The aim was to compare differences in overall survival (OS) and disease-free survival (DFS) between patients who did and did not achieve asparagine depletion, defined by enzyme levels gre ... Full text Link to item Cite

Hematopoietic cell transplantation for Chediak-Higashi syndrome.

Journal Article Bone Marrow Transplant · April 2007 We reviewed outcomes after allogeneic hematopoietic cell transplantation (HCT) in 35 children with Chediak-Higashi syndrome (CHS). Twenty-two patients had a history of the life-threatening accelerated phase of CHS before HCT and 11 were in accelerated phas ... Full text Link to item Cite

379: Fly me to the moon

Conference Biology of Blood and Marrow Transplantation · February 2007 Full text Cite

414: Engraftment syndrome

Conference Biology of Blood and Marrow Transplantation · February 2007 Full text Cite

413: The Duke pediatric blood and marrow transplant nursing graduate integration program

Conference Biology of Blood and Marrow Transplantation · February 2007 Full text Cite

Isolation of early hematopoietic cells, including megakaryocyte progenitors, in the ALDH-bright cell population of cryopreserved, banked UC blood.

Journal Article Cytotherapy · 2007 BACKGROUND: ALDH-bright (ALDH(br)) cell populations sorted from freshly collected umbilical cord blood (UCB) on the basis of their high aldehyde dehydrogenase (ALDH) activity are highly enriched for HPC. HPC with low ALDH activity (ALDH(dim)) are primarily ... Full text Link to item Cite

Modification of a commercial cell sorter to support efficient and reliable preparation of ALDH-bright cells for clinical use.

Journal Article Cytotherapy · 2007 BACKGROUND: Cell populations manufactured by conventional commercial cell sorters have been safely infused into patients, but reliably sterilizing these instruments remains challenging. We are developing clinical protocols involving use of ALDH bright cell ... Full text Link to item Cite

Cord blood banking for potential future transplantation.

Journal Article Pediatrics · January 2007 In recent years, umbilical cord blood, which contains a rich source of hematopoietic stem and progenitor cells, has been used successfully as an alternative allogeneic donor source to treat a variety of pediatric genetic, hematologic, immunologic, and onco ... Full text Link to item Cite

Antigen-specific T-lymphocyte function after cord blood transplantation.

Journal Article Biol Blood Marrow Transplant · December 2006 It has not been possible to determine the singular contribution of naive T lymphocytes to antigen-specific immunity after hematopoietic stem cell transplantation (HSCT), because of the confounding effects of donor-derived antigen-specific T lymphocytes pre ... Full text Link to item Cite

Outcomes of 122 diverse adult and pediatric cord blood transplant recipients from a large cord blood bank.

Journal Article Transfusion · December 2006 BACKGROUND: Umbilical cord blood is a useful stem cell source for some patients. The American Red Cross Cord Blood Program was established as a national network of cord blood banks. Nine thousand cord blood units were cryopreserved for transplant use. STUD ... Full text Link to item Cite

Transplantation of a child with sickle cell anemia with an unrelated cord blood unit after reduced intensity conditioning.

Journal Article J Pediatr Hematol Oncol · December 2006 Sickle cell disease can be corrected by hematopoietic cell transplantation but success is limited by low availability of matched related/unrelated donors and comorbidities leading to the increased transplant-related morbidity/mortality. There is a need for ... Full text Link to item Cite

Preliminary Results of a Pilot Trial of Unrelated Umbilical Cord Blood Transplantation (UCBT) Augmented with Cytokine-Primed Aldehyde Dehydrogenase-Bright (ALDHbr) Cells.

Conference Blood · November 16, 2006 AbstractUmbilical cord blood (UCB) transplantation results in delayed myeloid and platelet engraftment, likely due to lower cell dosing with cord blood. One strategy to overcome this problem is to augment th ... Full text Cite

Prospective Analysis of TEL and MLL Gene Rearrangements in Childhood Acute Lymphoblastic Leukemia: A Children’s Oncology Group Study.

Conference Blood · November 16, 2006 AbstractIn a retrospective study, we previously demonstrated that TEL gene rearrangements, representing the TEL-AML1 fusion created by the t(12;21), conferred a favorable prognosis among children with newly ... Full text Cite

Testicular Relapse in Lesser, Standard, and High Risk Patients Treated with Frontline Therapy for Childhood ALL. Pediatric Oncology Group Protocols 9201, 9405, 9605, and 9406.

Conference Blood · November 16, 2006 AbstractFrom 1984–94, testicular relapse (TR) was reported in 10–20% of boys with ALL. Addition of intermediate and higher dose methotrexate (MTX) in subsequent trials may have reduced this risk. Pediatric O ... Full text Cite

Dosing of Post-Thaw Colony Forming Units (CFU) and CD34 Cells Predict Engraftment and Survival in Recipients of Banked Unrelated Donor Umbilical Cord Blood (UCB) for Allogeneic Transplantation.

Conference Blood · November 16, 2006 AbstractA single umbilical cord blood unit (CBU) often doses patients at the threshhold for successful hematopoietic cell transplantation. While both cryopreserved and infused total nucleated cell (TNC) cont ... Full text Cite

Acute Graft Versus Host Disease (GVHD) Is Increased in Patients Receiving Cyclosporine/Cellcept as Frontline Prophylaxis Against GVHD in Pediatric Patients Undergoing Allogeneic Stem Cell Transplantation.

Conference Blood · November 16, 2006 AbstractTraditionally, pediatric patients are prophylaxed against GvHD with cyclosporine (CSA) plus either methotrexate (Mtx) or methylprednisolone (Pred). Mtx worsens the severity of mucositis and renal ins ... Full text Cite

Melphalan Containing Cytoreduction Results in a Good Overall Survival (OS) in Pediatric Patients with Relapse or Refractory AML Undergoing Unrelated Umbilical Cord Blood Transplantation (UCBT).

Conference Blood · November 16, 2006 AbstractChildren with AML unable to achieve remission after initial diagnosis or at relapse will not be cured by conventional therapy but may be salvaged by allogeneic hematopoietic stem cell transplantation ... Full text Cite

The Addition of Fludarabine to a Conventional Busulfan/Melphalan/Anti-Thymocyte Globulin (Flu/Bu/Mel/ATG) Preparative Regimen Increased Engraftment without Additional Toxicity.

Journal Article Blood · November 16, 2006 AbstractBusulfan/Melphalan/ATG (Bu/Mel/ATG) have been used as a preparative regimen in children undergoing hematopoetic stem cell transplantation who are too young for or who cannot tolerate total body irrad ... Full text Cite

Multivariate Analysis of Patient and Graft Specific Factors among 330 Recipients of Unrelated Cord Blood Transplant (UCBT) To Predict Risk of Death from Opportunistic Infections in the First 6 Months after UCBT.

Conference Blood · November 16, 2006 AbstractUnrelated umbilical cord blood transplantation (UCBT) is a suitable option for those who lack HLA-matched sibling donors. However, opportunistic infections (OI), with the majority detected in the fir ... Full text Cite

In vitro priming and expansion of cytomegalovirus-specific Th1 and Tc1 T cells from naive cord blood lymphocytes.

Journal Article Blood · September 1, 2006 Adoptive transfer of CMV-specific cytotoxic T cells (CTLs) expanded in vitro from memory donor T cells can reduce the incidence of CMV disease in allogeneic transplant recipients. However, this approach has been unavailable in the cord blood (CB) transplan ... Full text Link to item Cite

Successful immune reconstitution decreases leukemic relapse and improves survival in recipients of unrelated cord blood transplantation.

Journal Article Biol Blood Marrow Transplant · September 2006 Allogeneic hematopoietic stem cell transplantation (HSCT) is established therapy for selected patients with acute leukemia. After transplantation, antileukemic immune responses are believed to eliminate residual leukemia cells and decrease the likelihood o ... Full text Link to item Cite

Neurophysiologic assessment of mucopolysaccharidosis III.

Journal Article Clin Neurophysiol · September 2006 OBJECTIVE: To describe finding of various neurophysiologic tests in patients with mucopolysaccharidosis III (MPS III) early in the disease course. METHODS: Patients were evaluated with flash visual evoked potentials (VEP), brainstem auditory evoked potenti ... Full text Link to item Cite

A staging system for infantile Krabbe disease to predict outcome after unrelated umbilical cord blood transplantation.

Journal Article Pediatrics · September 2006 OBJECTIVE: Infantile Krabbe disease, a rare neurodegenerative disorder that leads to rapid demyelination, dysmyelination, and death in the first 2 years of life, is responsive to treatment with umbilical cord blood transplantation provided that the patient ... Full text Link to item Cite

Characterization of cord blood natural killer and lymphokine activated killer lymphocytes following ex vivo cellular engineering.

Journal Article Biol Blood Marrow Transplant · June 2006 Cord blood (CB) natural killer (NK) and lymphokine-activated killer (LAK) cytotoxic cells are poorly characterized but might be used to treat minimal residual and/or recurrent malignant disease. Currently, there is no mechanism to use CB for adoptive cance ... Full text Link to item Cite

Ectopic expression of the proto-oncogene Mer in pediatric T-cell acute lymphoblastic leukemia.

Journal Article Clin Cancer Res · May 1, 2006 PURPOSE: The Mer receptor tyrosine kinase, cloned from a B-lymphoblastoid library, is the mammalian orthologue of the chicken retroviral oncogene v-eyk and sends antiapoptotic and transforming signals when activated. To determine if Mer expression is ectop ... Full text Link to item Cite

Compassion fatigue: Care for the caring

Conference Biology of Blood and Marrow Transplantation · February 2006 Full text Cite

Expansion of umbilical cord blood derived oligodendrocytes

Conference Biology of Blood and Marrow Transplantation · February 2006 Full text Cite

Nursing care of the granulocyte donor for pediatric stem cell transplant patients

Conference Biology of Blood and Marrow Transplantation · February 2006 Full text Cite

Results of the cord blood transplantation study (COBLT): outcomes of unrelated donor umbilical cord blood transplantation in pediatric patients with lysosomal and peroxisomal storage diseases.

Journal Article Biol Blood Marrow Transplant · February 2006 The Cord Blood Transplantation Study (COBLT), sponsored by the National Heart, Lung, and Blood Institute, is a phase II multicenter study designed to evaluate the use of cord blood in allogeneic transplantation. In this report, we evaluated the outcomes of ... Full text Link to item Cite

Graves’ disease following unrelated umbilical cord blood transplantation in pediatric patients

Conference Biology of Blood and Marrow Transplantation · February 2006 Full text Cite

Patient controlled analgesia by proxy

Conference Biology of Blood and Marrow Transplantation · February 2006 Full text Cite

Comparable long-term survival after unrelated and HLA-matched sibling donor hematopoietic stem cell transplantations for acute leukemia in children younger than 18 months.

Journal Article J Clin Oncol · January 1, 2006 PURPOSE: To describe outcomes after unrelated donor stem cell transplantation (HCT) in children (< 18 months at diagnosis) with acute leukemia and compare these with outcomes after human leukocyte antigen (HLA)-matched sibling donor HCT. PATIENTS AND METHO ... Full text Link to item Cite

Quantitative analysis of diffusion tensor imaging data in serial assessment of Krabbe disease.

Journal Article Ann N Y Acad Sci · December 2005 Krabbe disease is a rare autosomal recessive pediatric white matter (WM) disorder that is due to deficiency of a specific enzyme, beta-galactocerebrosidase. This report reviews our experience with use of diffusion tensor imaging (DTI) in serial assessment ... Full text Link to item Cite

Unique Reversible but Serious Adverse Events in Very Young Infants Undergoing Unrelated Donor Umbilical Cord Blood Transplantation for Non-Malignant Conditions: Increased Autoimmune Cytopenias and Hypertrophic Cardiomyopathy.

Conference Blood · November 16, 2005 AbstractBetween September 1998 and June 2004, 15 young infants, <3 months of age, 60% males, 14 Caucasian, 1 African American, underwent unrelated donor umbilical cord blood transplantation for treatment ... Full text Cite

Post-Thaw Colony Forming Unit (CFU) Counts and Yield Are the Most Important Predictors of Engraftment and Survival Following Unrelated Donor Cord Blood Transplantation (CBT): A COBLT Study Report.

Conference Blood · November 16, 2005 AbstractTotal nucleated cell count (TNC), CD34 and CFU content of cryopreserved cord blood units (CBUs) are measurements of hematopoietic potential previously demonstrated to be predictive of engraftment and ... Full text Cite

Transplant Outcomes and Stabilization of Neurological Function in Young Children with MPS III (Sanfilippo Syndrome) after Unrelated Donor Umbilical Cord Blood Transplantation.

Conference Blood · November 16, 2005 AbstractBetween September 1998 and June 2004, 15 children, ages 10 – 55 months, 60% males, 14 Caucasian, 1 mixed racial background, underwent unrelated donor umbilical cord blood transplantation for treatmen ... Full text Cite

Comparison of cytomegalovirus polymerase chain reaction and serology for screening umbilical cord blood components.

Journal Article Transfusion · November 2005 BACKGROUND: Recipients of umbilical cord blood (UCB) transplants are susceptible to opportunistic infections, including cytomegalovirus (CMV). To prevent CMV transmission from UCB donors, most laboratories perform serology on corresponding maternal samples ... Full text Link to item Cite

Untying the Gordian knot: policies, practices, and ethical issues related to banking of umbilical cord blood.

Journal Article J Clin Invest · October 2005 Since the first successful transplantation of umbilical cord blood in 1988, cord blood has become an important source of hematopoietic stem and progenitor cells for the treatment of blood and genetic disorders. Significant progress has been accompanied by ... Full text Link to item Cite

Busulfan/melphalan/antithymocyte globulin followed by unrelated donor cord blood transplantation for treatment of infant leukemia and leukemia in young children: the Cord Blood Transplantation study (COBLT) experience.

Journal Article Biol Blood Marrow Transplant · August 2005 A non-total body irradiation-containing preparative regimen was studied in young children (<4 years old) undergoing unrelated donor cord blood transplantation as part of the Cord Blood Transplantation trial for the treatment of acute lymphoblastic leukemia ... Full text Link to item Cite

Krabbe disease treated with hematopoietic stem cell transplantation: serial assessment of anisotropy measurements--initial experience.

Journal Article Radiology · July 2005 PURPOSE: To prospectively compare diffusion-tensor magnetic resonance (MR) imaging anisotropy measurements of white matter (WM) regions in early and late treatment groups of Krabbe disease patients treated with stem cell transplantation. MATERIALS AND METH ... Full text Link to item Cite

Results of the cord blood transplantation (COBLT) study unrelated donor banking program.

Journal Article Transfusion · June 2005 BACKGROUND: The goals of the Cord Blood Transplantation (COBLT) Study banking program initiated in 1996 were to develop standard operating procedures (SOPs) for cord blood (CB) donor recruitment and banking and to build an ethnically diverse unrelated CB b ... Full text Link to item Cite

Characterization of banked umbilical cord blood hematopoietic progenitor cells and lymphocyte subsets and correlation with ethnicity, birth weight, sex, and type of delivery: a Cord Blood Transplantation (COBLT) Study report.

Journal Article Transfusion · June 2005 BACKGROUND: The Cord Blood Transplantation (COBLT) Study banking program was initiated in 1996. The study goals were to develop standard operating procedures for cord blood (CB) donor recruitment and banking and to build an ethnically diverse unrelated CB ... Full text Link to item Cite

Phase II study of nelarabine (compound 506U78) in children and young adults with refractory T-cell malignancies: a report from the Children's Oncology Group.

Journal Article J Clin Oncol · May 20, 2005 PURPOSE: Nelarabine (compound 506U78), a water soluble prodrug of 9-b-d-arabinofuranosylguanine, is converted to ara-GTP in T lymphoblasts. We sought to define the response rate of nelarabine in children and young adults with refractory or recurrent T-cell ... Full text Link to item Cite

Phase I study of 506U78 administered on a consecutive 5-day schedule in children and adults with refractory hematologic malignancies.

Journal Article J Clin Oncol · May 20, 2005 PURPOSE: A phase I study was conducted to determine the maximum-tolerated dose (MTD), toxicity profile, and pharmacokinetics of a novel purine nucleoside, nelarabine, a soluble prodrug of 9-beta-D-arabinosylguanine (araG; Nelarabine), in pediatric and adul ... Full text Link to item Cite

Transplantation of umbilical-cord blood in babies with infantile Krabbe's disease.

Journal Article N Engl J Med · May 19, 2005 BACKGROUND: Infantile Krabbe's disease produces progressive neurologic deterioration and death in early childhood. We hypothesized that transplantation of umbilical-cord blood from unrelated donors before the development of symptoms would favorably alter t ... Full text Link to item Cite

Establishment of a National Cord Blood Banking Network through the National Marrow Donor Program

Conference Biology of Blood and Marrow Transplantation · February 2005 Full text Cite

Globoid cell leukodystrophy (Krabbe disease): normal umbilical cord blood galactocerebrosidase activity and polymorphic mutations.

Journal Article J Inherit Metab Dis · 2005 Globoid cell leukodystrophy is an inherited metabolic disorder of the central nervous system caused by deficiency of the lysosomal enzyme galactocerebrosidase. Haematopoietic stem cell transplantation is the only available effective treatment. The engraftm ... Full text Link to item Cite

Partial splenectomy before a hematopoietic stem cell transplantation in children.

Journal Article J Pediatr Surg · January 2005 UNLABELLED: Hematopoietic stem cell (HSC) engraftment is delayed in children with hypersplenism, and splenectomy may improve HSC engraftment. However, the use of total splenectomy in children is limited because of concerns for postsplenectomy sepsis. In th ... Full text Link to item Cite

Cord Blood Transplantation for Lysosomal Storage Diseases Demonstrates the Potential of Cord Blood Cells for Future Cellular Therapies.

Conference Blood · November 16, 2004 Cord blood stem and progenitor cells can rescue the bone marrow and immune system of pediatric and selected adult patients undergoing myeloablative chemotherapy. Cord blood cells exhibit immunologic tolerance allowing transplantation across partial ... Full text Cite

Immunophenotype and Analysis of Allo-Reactivity of Umbilical Cord Blood (UCB)-Derived T-Cells Following Ex-Vivo Expansion.

Conference Blood · November 16, 2004 AbstractIntroduction: Unrelated umbilical cord blood (UCB) is an effective source of allogeneic hematopoietic stem cells for transplantation therapy in patients lacking matched bone marrow donors. The major ... Full text Cite

High Expression of Granzymes and Perforin along with Increased T cell Cycling In Vivo Predicts the Development of Opportunistic Infections (OI) Following Unrelated Cord Blood Transplantation (UCBT).

Conference Blood · November 16, 2004 AbstractIntroduction: Unrelated umbilical cord blood transplantation (UUCBT) is a viable option for those who lack HLA-matched sibling donors. However, opportunistic infections (OI) occurring in the first 10 ... Full text Cite

Establishment of the NMDP Cord Blood Banking Network.

Conference Blood · November 16, 2004 AbstractBone marrow transplantation can successfully treat patients with advanced malignancies, bone marrow failure syndromes, congenital immunodeficiency syndromes, hemoglobinopathies and inborn errors of m ... Full text Cite

Defibrotide (DF) for the Treatment of Severe Veno-Occlusive Disease (VOD) and Multi-System Organ Failure (MOF) Post SCT: Final Results of a Phase II, Multicenter, Randomized Study and Preliminary Analyses of Surrogate Markers and Ultrasound Findings.

Conference Blood · November 16, 2004 AbstractIntroduction: DF, a polydisperse oligonucleotide, has anti-thrombotic, anti-ischemic and thrombolytic properties, especially on microvasculature. Studies have suggested that DF modulates endothelial ... Full text Cite

Dendritic and T Cell Subsets Correlate with the Development and Site of Acute GVHD Following Unrelated Cord Blood Transplantation. A Multivariate Analysis of Host, Graft, and Day+50 Immune Profile.

Conference Blood · November 16, 2004 AbstractHLA-mismatched unrelated umbilical cord blood transplantation (uUCBT) is a suitable option for those who lack HLA-matched sibling donors. Although UCB T cells mount significantly less cytotoxicity an ... Full text Cite

Purine nucleoside phosphorylase deficiency (PNP-def) presenting with lymphopenia and developmental delay: successful correction with umbilical cord blood transplantation.

Journal Article J Pediatr · November 2004 Purine nucleoside phosphorylase deficiency is a primary immunodeficiency syndrome characterized by the triad of recurrent infection, neurologic dysfunction, and autoimmunity. This patient presented atypically with few infections and normal T-cell function. ... Full text Link to item Cite

Cord blood transplantation in genetic disorders

Conference Biology of Blood and Marrow Transplantation · October 2004 Full text Cite

The incidence of testicular recurrence in boys with acute leukemia treated with total body and testicular irradiation and stem cell transplantation.

Journal Article Cancer · August 15, 2004 BACKGROUND: The incidence of testicular recurrence of childhood acute leukemia after total body irradiation (TBI) in conjunction with stem cell transplantation (SCT) has been reported to be as high as 24%. The authors studied the incidence of testicular fa ... Full text Link to item Cite

Utility of end consolidation bone marrow aspirates in childhood acute lymphoblastic leukemia [ALL]: A Pediatric Oncology Group study [POG].

Journal Article J Clin Oncol · July 15, 2004 8564 Background: There is little information on the value of bone marrow aspirates (BMA) as a guide to treatment in pediatric ALL. Although BMA morphology provides useful information at diagnosis, end of induction, and end of therapy, its value when perfor ... Link to item Cite

Unrelated umbilical cord blood transplantation for an infant with beta-thalassemia major.

Journal Article J Pediatr Hematol Oncol · June 2004 BACKGROUND: beta-thalassemia major, one of the most prevalent hemoglobinopathies throughout the world, can be cured by allogeneic stem cell transplantation therapy. Many patients, however, lack a suitably matched related donor. Unrelated umbilical cord blo ... Full text Link to item Cite

Cord-blood transplants from unrelated donors in patients with Hurler's syndrome.

Journal Article N Engl J Med · May 6, 2004 BACKGROUND: Hurler's syndrome (the most severe form of mucopolysaccharidosis type I) causes progressive deterioration of the central nervous system and death in childhood. Allogeneic bone marrow transplantation before the age of two years halts disease pro ... Full text Link to item Cite

Racial diversity with high nucleated cell counts and CD34 counts achieved in a national network of cord blood banks.

Journal Article Biol Blood Marrow Transplant · April 2004 Banked, unrelated, partially HLA-matched, umbilical cord blood is an alternative stem cell source for patients in need of transplantation therapy who lack traditionally matched donors. A presumed advantage of cord blood is the ability to increase recruitme ... Full text Link to item Cite

Ex vivo expansion of immature and mature T cells derived from umbilical cord blood (UCB)

Conference Biology of Blood and Marrow Transplantation · February 2004 Full text Cite

Umbilical cord blood cells engraft and differentiate in cardiac tissues after human tranplantation

Conference Biology of Blood and Marrow Transplantation · February 2004 Full text Cite

Isolation of oligodendrocyte precursors from umbilical cord blood

Conference Biology of Blood and Marrow Transplantation · February 2004 Full text Cite

Results of the cord blood transplantation study (COBLT) unrelated donor banking program

Conference Biology of Blood and Marrow Transplantation · February 2004 Full text Cite

Unrelated umbilical cord blood transplantation for familial erythrophagocytic lymphohistiocytosis

Conference Biology of Blood and Marrow Transplantation · February 2004 Full text Cite

Unrelated umbilical cord blood transplantation in adult patients.

Journal Article Biol Blood Marrow Transplant · December 2003 Since January 1996, we have administered myeloablative therapy followed by infusion of unrelated umbilical cord blood cells in 57 adult patients with high-risk disease. The median age was 31 years (range, 18-58 years), and the median weight was 70 kg (rang ... Full text Link to item Cite

Coexistent naïve phenotype and higher cycling rate of cord blood T cells as compared to adult peripheral blood.

Journal Article Exp Hematol · August 2003 OBJECTIVE: Umbilical cord blood (UCB) T cells are predominantly CD45RA(+), secrete less cytokines, and have diminished cytotoxicity compared to adult peripheral blood (PB). We hypothesized that the functional impairment of bulk UCB cells results from the r ... Full text Link to item Cite

Cerebral phaeohyphomycosis in an immunodeficient child treated medically with combination antifungal therapy.

Journal Article Med Mycol · August 2003 Cerebral phaeohyphomycosis is a rare fungal infection with a poor prognosis when using conventional antifungal therapy in the absence of neurosurgical intervention. We present a case of a pediatric patient with inoperable Cladophialophora bantiana cerebral ... Full text Link to item Cite

A Phase I-II trial of polyethylene glycol-conjugated L-asparaginase in patients with multiple myeloma.

Journal Article Cancer · July 1, 2003 BACKGROUND: Multiple myeloma remains an incurable disease. New agents are needed to improve therapy for patients with this disease. Previous investigators evaluated in vitro sensitivity of myeloma cells to polyethylene glycol-conjugated L-asparaginase (PEG ... Full text Link to item Cite

Augmentation of umbilical cord blood (UCB) transplantation with ex vivo-expanded UCB cells: results of a phase 1 trial using the AastromReplicell System.

Journal Article Blood · June 15, 2003 Allogeneic stem cell transplantation with umbilical cord blood (UCB) cells is limited by the cell dose a single unit provides recipients. Ex vivo expansion is one strategy to increase the number of cells available for transplantation. Aastrom Biosciences d ... Full text Link to item Cite

High-dose chemotherapy with autologous stem-cell rescue in children and adults with newly diagnosed pineoblastomas.

Journal Article J Clin Oncol · June 1, 2003 PURPOSE: We evaluated the usefulness of a treatment regimen that included high-dose chemotherapy (HDC) with autologous stem-cell rescue (ASCR) in patients with newly diagnosed pineoblastoma (PBL). PATIENTS AND METHODS: Twelve patients with PBL were initial ... Full text Link to item Cite

Marrow cell transplantation for infantile hypophosphatasia.

Journal Article J Bone Miner Res · April 2003 An 8-month-old girl who seemed certain to die from the infantile form of hypophosphatasia, an inborn error of metabolism characterized by deficient activity of the tissue-nonspecific isoenzyme of alkaline phosphatase (TNSALP), underwent the first trial of ... Full text Link to item Cite

Vincristine-induced neuropathy as the initial presentation of charcot-marie-tooth disease in acute lymphoblastic leukemia: a Pediatric Oncology Group study.

Journal Article J Pediatr Hematol Oncol · April 2003 PURPOSE: After profound peripheral neurotoxicity during induction chemotherapy for acute lymphoblastic leukemia (ALL) in the index patient with Charcot-Marie-Tooth hereditary neuropathy (CMT), study coordinators of the Pediatric Oncology Group (POG) front- ... Full text Link to item Cite

Related umbilical cord blood transplantation in patients with thalassemia and sickle cell disease.

Journal Article Blood · March 15, 2003 Allogeneic bone marrow transplantation (BMT) from HLA-identical siblings is an accepted treatment for both thalassemia and sickle cell disease (SCD). However, it is associated with decided risk of both transplant-related mortality (TRM) and chronic graft-v ... Full text Link to item Cite

A patient with extensive stem cell factor-induced hyperpigmentation.

Journal Article Cutis · February 2003 Stem cell factor (SCF) is a cytokine that stimulates development of erythroid precursors and, consequently, may have potential importance in the treatment of certain anemias. We report a case of a young woman with Diamond-Blackfan anemia who received SCF t ... Link to item Cite

Absolute values of dendritic cell subsets in bone marrow, cord blood, and peripheral blood enumerated by a novel method.

Journal Article Stem Cells · 2003 Dendritic cells (DCs) are pivotal in inducing immunity or alternatively downregulating immune reactivity. In humans, the opposing phenotypic subsets of CD11c(+)/CD123(-) "myeloid" DCs and CD123(+)/CD11c(-) "lymphoid" DCs have been proposed to orchestrate t ... Full text Link to item Cite

Multi-institutional use of defibrotide in 88 patients after stem cell transplantation with severe veno-occlusive disease and multisystem organ failure: response without significant toxicity in a high-risk population and factors predictive of outcome.

Journal Article Blood · December 15, 2002 Veno-occlusive disease (VOD) is the most common regimen-related toxicity accompanying stem cell transplantation (SCT). Severe VOD complicated by multisystem organ failure (MOF) remains almost uniformly fatal. Preliminary experience with defibrotide (DF), a ... Full text Link to item Cite

Optimization of informed consent for umbilical cord blood banking.

Journal Article Am J Obstet Gynecol · December 2002 OBJECTIVE: The purpose of this project was to evaluate the informed consent process for donation to a public umbilical cord blood bank. STUDY DESIGN: Telephone interviews were conducted with 170 women who had given consent to donate their newborn infants' ... Full text Link to item Cite

Placental/umbilical cord blood for unrelated-donor bone marrow reconstitution: relevance of nucleated red blood cells.

Journal Article Blood · October 1, 2002 Placental/umbilical cord blood (PCB) is a source of hematopoietic stem cells for bone marrow reconstitution. Engraftment speed and survival are related to the total nucleated cell (TNC) dose of the graft. This study explored the possible influence on engra ... Full text Link to item Cite

Combination treatment of bullous pemphigoid with anti-CD20 and anti-CD25 antibodies in a patient with chronic graft-versus-host disease.

Journal Article Bone Marrow Transplant · September 2002 In this case report we describe a novel treatment with two chimeric monoclonal antibodies (MoAb) targeting the autoimmune B cell clone responsible for bullous pemphigoid (BP) as a manifestation of steroid refractory chronic graft-versus-host disease (GVHD) ... Full text Link to item Cite

Ex vivo expansion, maturation, and activation of umbilical cord blood-derived T lymphocytes with IL-2, IL-12, anti-CD3, and IL-7. Potential for adoptive cellular immunotherapy post-umbilical cord blood transplantation.

Journal Article Exp Hematol · March 2002 OBJECTIVES: We investigated whether umbilical cord blood (UCB) T cells could be ex vivo expanded and activated in short-term culture for potential utilization as adoptive cellular immunotherapy post-umbilical cord blood transplantation (UCBT). METHODS: Fre ... Full text Link to item Cite

Human herpesvirus 6 limbic encephalitis after stem cell transplantation.

Journal Article Ann Neurol · November 2001 Central nervous system complications are common in stem cell transplant recipients, but selective involvement of the medial temporal area is unusual. The 5 patients reported here presented after stem cell transplantation with increased hippocampal T2 signa ... Full text Link to item Cite

Hematopoietic engraftment and survival in adult recipients of umbilical-cord blood from unrelated donors.

Journal Article N Engl J Med · June 14, 2001 Featured Publication BACKGROUND: Umbilical-cord blood from unrelated donors who are not HLA-identical with the recipients can restore hematopoiesis after myeloablative therapy in children. We studied the use of transplantation of umbilical-cord blood to restore hematopoiesis i ... Full text Link to item Cite

Evaluation of white matter anisotropy in Krabbe disease with diffusion tensor MR imaging: initial experience.

Journal Article Radiology · March 2001 PURPOSE: To compare diffusion tensor magnetic resonance imaging with conventional T2-weighted imaging for evaluation of white matter changes in patients with Krabbe disease. MATERIALS AND METHODS: In eight patients with Krabbe disease and eight age-matched ... Full text Link to item Cite

Recombinant human granulocyte colony-stimulating factor therapy for patients with neutropenia and/or neutrophil dysfunction secondary to glycogen storage disease type 1b.

Journal Article Blood · January 15, 2001 The purpose of this study was to evaluate the efficacy and toxicity of recombinant human granulocyte colony-stimulating factor (rhG-CSF) therapy in patients with neutropenia and/or neutrophil dysfunction secondary to glycogen storage disease (GSD) type 1b. ... Full text Link to item Cite

Management of cholelithiasis in pediatric patients who undergo bone marrow transplantation.

Journal Article J Pediatr Surg · January 2001 PURPOSE: The aim of this study was to determine the incidence, risk factors, and proper management for asymptomatic cholelithiasis in children undergoing bone marrow transplantation (BMT). METHODS: The authors reviewed retrospectively the records of 575 ch ... Full text Link to item Cite

Stable mixed hematopoietic chimerism after bone marrow transplantation for sickle cell anemia.

Journal Article Biol Blood Marrow Transplant · 2001 A multicenter investigation of allogeneic bone marrow transplantation for children with sickle cell disease was conducted that included 27 European and North American transplant centers. Fifty-nine patients who ranged in age from 3.3 to 15.9 years (median, ... Full text Link to item Cite

Low incidence of Epstein-Barr virus-associated posttransplantation lymphoproliferative disorders in 272 unrelated-donor umbilical cord blood transplant recipients.

Journal Article Biol Blood Marrow Transplant · 2001 Umbilical cord blood (UCB) is being increasingly used for transplantation, but the ability of neonatal T cells to regulate Epstein-Barr virus (EBV)-associated lymphoproliferation is unknown. Because UCB transplantation (UCBT) is associated with a relativel ... Full text Link to item Cite

T-Cell recovery in adults and children following umbilical cord blood transplantation.

Journal Article Biol Blood Marrow Transplant · 2001 T-cell reconstitution following allogeneic stem cell transplantation may involve thymic education of donor-derived precursors or peripheral expansion of mature T cells transferred in the graft. T cell-receptor excision circles (sjTRECs) are generated withi ... Full text Link to item Cite

Graft-versus-leukemia-induced complete remission following unrelated umbilical cord blood transplantation for acute leukemia.

Journal Article Bone Marrow Transplant · December 2000 A 15-year-old female received an unrelated three of six HLA antigen matched umbilical cord blood (UCB) transplant for refractory, relapsed T-cell ALL. Conditioning consisted of TBI, melphalan, and anti-thymocyte globulin (ATG), with cyclosporin A (CsA) and ... Full text Link to item Cite

Assay method, cord blood bank, nucleated cell count, and neonatal factors influence the CD34+ cell count in cord blood: The coblt experience

Journal Article Blood · December 1, 2000 CD34+ cells in umbilical cord blood are important in determining the hematopoietic stem cell dose and its potential influence on neutrophil recovery following cord blood transplantation. In the NHLBI sponsored Cord Blood Transplantation (COBLT) Study, stan ... Cite

Preliminary characterization of the surface staining of placental derived adherent cells: a potential new source of stroma for umbilical cord blood (ucb) expansion

Journal Article Blood · December 1, 2000 Stromal-based ex-vivo expansion of UCB provides a potential method to overcome the limited graft cell dose for adult recipients. The benefit of stromal-based expansion compared to cytokine-based expansion is maintenance of primitive stem and progenitor cel ... Cite

High dose chemotherapy followed by transplantation of unrelated, banked, umbilical cord blood provides curative therapy for young pediatric patients with familial erythrophagocytic lymphohistiocytosis fed

Journal Article Blood · December 1, 2000 Familial erythrophagocytic lymphohistiocytosis (PEL) is an inherited disease that is uniformly fatal without myeloablation and transplantation therapy. Frequently babies and young children with this condition need to proceed to transplant rapidly before di ... Cite

Combination treatment of bullous skin gvhd with ANTI-CD20 and ANTI-CD25 antibodies

Journal Article Blood · December 1, 2000 We report on a novel treatment for bullous skin GVHD prompted by immunologie monitoring. A 9 year old boy with ALL in CR3 received a 4/6 HLA-matched unrelated placental graft. Grade 3 aGVHD of the gut 5 weeks after transplant was controlled with steroids, ... Cite

Outcomes of unrelated umbilical cord blood transplantation in 158 pediatric patients consecutively transplanted at a single institution using cord blood units from a single bank

Journal Article Blood · December 1, 2000 Over the past decade, umbilical cord blood (UCB) has been shown to provide adequate stem cells for bone marrow reconstitution after myeloablative therapy. Reports to date include 2 from registries and a few small pilot studies, but there have been no repor ... Cite

Impact of bone marrow transplantation for symptomatic sickle cell disease: an interim report. Multicenter investigation of bone marrow transplantation for sickle cell disease.

Journal Article Blood · March 15, 2000 Fifty children who had symptomatic sickle cell disease received matched sibling marrow allografts between September 1991 and March 1999, with Kaplan-Meier probabilities of survival and event-free survival of 94% and 84%, respectively. Twenty-six patients ( ... Link to item Cite

Impact of bone marrow transplantation for symptomatic sickle cell disease: An interim report

Journal Article Blood · March 15, 2000 Fifty children who had symptomatic sickle cell disease received matched sibling marrow allografts between September 1991 and March 1999, with Kaplan- Meier probabilities of survival and event-free survival of 94% and 84%, respectively. Twenty-six patients ... Cite

Effects of cranial radiation in children with high risk T cell acute lymphoblastic leukemia: a Pediatric Oncology Group report.

Journal Article Leukemia · March 2000 Contemporary chemotherapy has significantly improved event-free survival among patients with T cell-lineage acute lymphoblastic leukemia (T-ALL). Unlike B-precursor ALL, most investigators are still using cranial radiation (CRT) and are hesitant to rely so ... Full text Link to item Cite

Measurements from normal umbilical cord blood of four lysosomal enzymatic activities: alpha-L-iduronidase (Hurler), galactocerebrosidase (globoid cell leukodystrophy), arylsulfatase A (metachromatic leukodystrophy), arylsulfatase B (Maroteaux-Lamy).

Journal Article Bone Marrow Transplant · March 2000 Umbilical cord blood (UCB) has received increasing attention as a source of unrelated hematopoietic stem cells for transplantation. Lysosomal diseases have been effectively treated and normal enzymatic activity has occurred subsequent to engraftment using ... Full text Link to item Cite

Pharmacokinetics of nelarabine and 9-beta-D-arabinofuranosyl guanine in pediatric and adult patients during a phase I study of nelarabine for the treatment of refractory hematologic malignancies.

Journal Article J Clin Oncol · March 2000 PURPOSE: To characterize the pharmacokinetics of nelarabine (506U78), the water-soluble prodrug of 9-beta-D-arabinofuranosyl guanine (ara-G), and ara-G in pediatric and adult patients with refractory hematologic malignancies. Ara-G is phosphorylated within ... Full text Link to item Cite

Evaluation of pre-radiotherapy cyclophosphamide in patients with newly diagnosed glioblastoma multiforme. Writing Committee for The Brain Tumor Center at Duke.

Journal Article J Neurooncol · 2000 Cyclophosphamide is an alkylating agent that has shown activity in the treatment of pediatric brain tumors, including high-grade gliomas. This study was designed to evaluate the response of patients with newly diagnosed glioblastoma multiforme to pre-radio ... Full text Link to item Cite

Umbilical cord blood collection, storage and transplantation: Issues and recommendations for expectant parents and patients

Journal Article Cancer Research, Therapy and Control · December 1, 1999 Clinical investigators have recently explored the suitability of umbilical cord blood as an alternative source of hematopoietic stem cells for children and adults in need of transplantation who do not have suitably matched bone marrow donors. Despite the g ... Cite

Association of reticular dysgenesis (thymic alymphoplasia and congenital aleukocytosis) with bilateral sensorineural deafness.

Journal Article J Pediatr · September 1999 Reticular dysgenesis is a rare congenital disorder characterized by severe combined immunodeficiency and profound neutropenia, curable to date, only by bone marrow transplantation. This report describes the association of bilateral sensorineural deafness w ... Full text Link to item Cite

Present realities of stem cell transplantation

Journal Article Cancer Research Therapy and Control · July 27, 1999 Cite

Cord blood banking for potential future transplantation: subject review. American Academy of Pediatrics. Work Group on Cord Blood Banking.

Journal Article Pediatrics · July 1999 In recent years, umbilical cord blood, which contains a large number of hematopoietic stem cells, has been used successfully for allogeneic transplantation to treat a variety of pediatric genetic, hematologic, and oncologic disorders. It is a potential alt ... Full text Link to item Cite

Cord-blood transplants - Reply

Journal Article NEW ENGLAND JOURNAL OF MEDICINE · April 22, 1999 Link to item Cite

Cord-blood transplants.

Journal Article N Engl J Med · April 22, 1999 Full text Link to item Cite

Swainsonine protects both murine and human haematopoietic systems from chemotherapeutic toxicity.

Journal Article Br J Cancer · April 1999 The haematopoietic system is sensitive to cytotoxic damage and is often the site of dose-limiting toxicity. We previously reported that swainsonine, an inhibitor of protein glycosylation, reduced the bone marrow toxicity resulting from a single dose of ant ... Full text Link to item Cite

Intensive high-dose asparaginase consolidation improves survival for pediatric patients with T cell acute lymphoblastic leukemia and advanced stage lymphoblastic lymphoma: a Pediatric Oncology Group study.

Journal Article Leukemia · March 1999 This study was designed to test the hypothesis that high-dose asparaginase consolidation therapy improves survival in pediatric patients with T cell acute lymphoblastic leukemia and advanced stage lymphoblastic lymphoma. Five hundred and fifty-two patients ... Full text Link to item Cite

Complete hematologic and cytogenetic response to 2-amino-9-beta-D-arabinosyl-6-methoxy-9H-guanine in a patient with chronic myelogenous leukemia in T-cell blastic phase: a case report and review of the literature.

Journal Article Cancer · January 1, 1999 BACKGROUND: T-cell lymphoid blastic phase (BP) transformation is rare in chronic myelogenous leukemia (CML). 2-amino-9-beta-D-arabinosyl-6-methoxy-9H-guanine (GW506U78), a prodrug of arabinosylguanine (ara-G), is effective in T-cell leukemias. METHODS: The ... Link to item Cite

Retroviral vector-mediated gene transfer into umbilical cord blood-derived megakaryocyte and platelet progenitors.

Journal Article Biol Blood Marrow Transplant · 1999 Optimizing platelet engraftment following hematopoietic stem cell transplantation is essential for minimizing transplant-related morbidity, particularly following umbilical cord blood transplantation (UCBT), where platelet engraftment frequently takes >60 ... Full text Link to item Cite

Central nervous system atypical teratoid tumor/rhabdoid tumor: response to intensive therapy and review of the literature.

Journal Article J Neurooncol · December 1998 Central nervous system atypical teratoid/rhabdoid tumor (ATT/RT) of infancy and childhood is a unique histologic entity with an extremely aggressive natural history. Standard therapy for infant and childhood medulloblastoma, for which this entity is often ... Full text Link to item Cite

Cord Blood Transplantation Study (COBLT): cord blood bank standard operating procedures.

Journal Article J Hematother · December 1998 In 1995, the National Heart Lung and Blood Institute (NHLBI) solicited requests for a proposal (RFP) entitled "Transplant Centers for Clinical Research on Transplantation of Umbilical Cord Stem and Progenitor Cells." Three banks, six transplant centers, an ... Full text Link to item Cite

Outcomes among 562 recipients of placental-blood transplants from unrelated donors.

Journal Article N Engl J Med · November 26, 1998 BACKGROUND: A program for banking, characterizing, and distributing placental blood, also called umbilical-cord blood, for transplantation provided grafts for 562 patients between August 24, 1992, and January 30, 1998. We evaluated this experience. METHODS ... Full text Link to item Cite

Compound GW506U78 in refractory hematologic malignancies: relationship between cellular pharmacokinetics and clinical response.

Journal Article J Clin Oncol · November 1998 PURPOSE: In vitro investigations with arabinosylguanine (ara-G) demonstrated potent cytotoxicity to T-lymphoblastoid cell lines. The goals of the present study were to evaluate GW506U78, a prodrug of ara-G, against human hematologic malignancies and to det ... Full text Link to item Cite

Toxicity, pharmacology and feasibility of administration of PEG-L-asparaginase as consolidation therapy in patients undergoing bone marrow transplantation for acute lymphoblastic leukemia.

Journal Article Bone Marrow Transplant · May 1998 We attempted to administer PEG-L-asparaginase (PEG-L-A) following hematologic recovery to 38 patients undergoing autologous or allogeneic marrow transplantation for acute lymphoblastic leukemia (ALL). Twenty-four patients (12 of 22 receiving allogeneic and ... Full text Link to item Cite

Hematopoietic stem-cell transplantation in globoid-cell leukodystrophy.

Journal Article N Engl J Med · April 16, 1998 BACKGROUND: Globoid-cell leukodystrophy is caused by a deficiency of galactocerebrosidase, which results in progressive central nervous system deterioration. We investigated whether allogeneic hematopoietic stem-cell transplantation can provide a source of ... Full text Link to item Cite

Hurler syndrome: II. Outcome of HLA-genotypically identical sibling and HLA-haploidentical related donor bone marrow transplantation in fifty-four children. The Storage Disease Collaborative Study Group.

Journal Article Blood · April 1, 1998 Untreated patients with Hurler syndrome (MPSIH) experience progressive neurologic deterioration and early death. Allogeneic bone marrow transplantation (BMT) ameliorates or halts this course. The Storage Disease Collaborative Study Group was formed to eval ... Link to item Cite

Multiparameter-fluorescence activated cell sorting analysis of retroviral vector gene transfer into primitive umbilical cord blood cells.

Journal Article Exp Hematol · April 1998 Retroviral vector gene transfer strategies are currently being developed to treat a variety of hematopoietic disorders. To date, genetic modification of human pluripotent hematopoietic stem cells has been inefficient. In the present study we developed reag ... Link to item Cite

Hurler syndrome: II. Outcome of HLA-genotypically identical sibling and HLA-haploidentical related donor bone marrow transplantation in fifty-four children

Journal Article Blood · April 1, 1998 Untreated patients with Hurler syndrome (MPSIH) experience progressive neurologic deterioration and early death. Allogeneic bone marrow transplantation (BMT) ameliorates or halts this course. The Storage Disease Collaborative Study Group was formed to eval ... Full text Cite

Activity of high-dose cyclophosphamide in the treatment of childhood malignant gliomas.

Journal Article Med Pediatr Oncol · February 1998 Seventeen patients less than or equal to 20 years of age with newly diagnosed (n = 10) or recurrent (n = 7) malignant gliomas (anaplastic astrocytoma and glioblastoma multiforme) were treated with cyclophosphamide in association with hematopoietic cytokine ... Full text Link to item Cite

Cord blood banking.

Journal Article Bull World Health Organ · 1998 Link to item Cite

Collaborative multicenter investigation of marrow transplantation for sickle cell disease: current results and future directions.

Journal Article Biol Blood Marrow Transplant · December 1997 We present updated results of a multicenter collaborative investigation of bone marrow transplantation for sickle cell disease. Between September 1991 and April 1997, thirty-four children less than 16 years of age with severe sickle cell disease received m ... Link to item Cite

Cord blood stem cells.

Journal Article Curr Opin Hematol · November 1997 Use of bone marrow transplant therapy has been limited both by the availability of suitable stem cell donors as well as the various immunologic complications, namely graft-versus-host disease (GVHD) and profound immunosuppression resulting in opportunistic ... Full text Link to item Cite

Ethical issues in umbilical cord blood banking. Working Group on Ethical Issues in Umbilical Cord Blood Banking.

Journal Article JAMA · September 17, 1997 OBJECTIVE: Banking umbilical cord blood (UCB) to be used as a source of stem cells for transplantation is associated with a set of ethical issues. An examination of these issues is needed to inform public policy and to raise the awareness of prospective pa ... Link to item Cite

Umbilical cord blood stem cells: application for the treatment of patients with hemoglobinopathies.

Journal Article J Pediatr · May 1997 Even though there is recognized morbidity and death associated with bone marrow transplantation, this procedure has been performed successfully in a substantial number of patients with hemoglobinopathies. However, finding a suitable related donor is often ... Full text Link to item Cite

High-dose chemotherapy with autologous stem-cell rescue in patients with recurrent and high-risk pediatric brain tumors.

Journal Article J Clin Oncol · May 1997 PURPOSE: We treated 49 patients with recurrent or poor-prognosis CNS malignancies with high-dose chemotherapy regimens followed by autologous marrow rescue with or without peripheral-blood stem-cell augmentation to determine the toxicity of and event-free ... Full text Link to item Cite

A phase I trial of recombinant human interleukin-1 beta (OCT-43) following high-dose chemotherapy and autologous bone marrow transplantation.

Journal Article Bone Marrow Transplant · February 1997 We studied the effects of escalating doses of recombinant human IL-1 beta in patients receiving high-dose chemotherapy and ABMT for metastatic breast cancer or malignant melanoma. Sixteen patients received IL-1 beta, 4 to 32 ng/kg/day administered subcutan ... Full text Link to item Cite

Placental-blood transplantation - Reply

Journal Article NEW ENGLAND JOURNAL OF MEDICINE · January 2, 1997 Link to item Cite

Placental-blood transplantation.

Journal Article N Engl J Med · January 2, 1997 Full text Link to item Cite

Umbilical cord blood stem cells

Journal Article INTERNATIONAL JOURNAL OF PEDIATRIC HEMATOLOGY/ONCOLOGY · January 1, 1997 Link to item Cite

Umbilical cord blood stem cells. Application for the treatment of patients with thalassemia

Journal Article International Journal of Pediatric Hematology/Oncology · January 1, 1997 Cite

Variable loss of oxidative burst function in stored neutrophils obtained by leukopheresis.

Journal Article JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY · January 1, 1997 Link to item Cite

Phase I trial and pharmacokinetic (PK) and pharmacodynamics (PD) study of topotecan using a five-day course in children with refractory solid tumors: a pediatric oncology group study.

Journal Article J Pediatr Hematol Oncol · November 1996 PURPOSE: A phase I trial was conducted in children with refractory solid tumors to determine the maximum tolerated dose (MTD), dose-limiting toxicity (DLT), pharmacokinetics, and pharmacodynamics for topotecan administered by a 30-min infusion for 5 consec ... Full text Link to item Cite

Placental blood as a source of hematopoietic stem cells for transplantation into unrelated recipients.

Journal Article N Engl J Med · July 18, 1996 Featured Publication BACKGROUND: Transplantation of bone marrow from unrelated donors is limited by a lack of HLA-matched donors and the risk of graft-versus-host disease (GVHD). Placental blood from sibling donors can reconstitute hematopoiesis. We report preliminary results ... Full text Link to item Cite

State of the art in umbilical cord transplantation.

Journal Article Oncology (Williston Park) · July 1996 Link to item Cite

Successful treatment of childhood pilocytic astrocytomas metastatic to the leptomeninges with high-dose cyclophosphamide.

Journal Article Med Pediatr Oncol · July 1996 Leptomeningeal dissemination of childhood pilocytic astrocytoma (PA) is a rare event with little information available regarding therapy. We report here four children with disseminated PA whom we treated with high doses of cyclophosphamide with clinical be ... Full text Link to item Cite

Treatment of patients with pineoblastoma with high dose cyclophosphamide.

Journal Article Med Pediatr Oncol · June 1996 The outcome for patients with pineoblastoma has historically been very poor, with most patients dying of disseminated disease despite irradiation. Furthermore, the low incidence of this tumor has hindered progress toward defining better treatment strategie ... Full text Link to item Cite

Successful treatment of two children with Langerhans' cell histiocytosis with 2'-deoxycoformycin.

Journal Article J Pediatr Hematol Oncol · May 1996 PURPOSE: Langerhans' cell histiocytosis (LCH) is problematic illness with a subset of patients experiencing a relapsing or progressive course despite therapy with chemotherapy and/or immunosuppressive agents. Novel therapies are required. 2'-deoxycoformyci ... Full text Link to item Cite

Umbilical cord blood for transplantation.

Journal Article JAMA · March 27, 1996 Link to item Cite

Ethical aspects of banking placental blood for transplantation.

Journal Article JAMA · December 13, 1995 Transplantation of blood cells harvested from the umbilical cord immediately after birth has been effective in repopulating the bone marrow. These placental blood transplantations may be safer than conventional bone marrow transplantations and may suspend ... Link to item Cite

Effects of granulocyte-macrophage colony stimulating factor produced in Chinese hamster ovary cells (regramostim), Escherichia coli (molgramostim) and yeast (sargramostim) on priming peripheral blood progenitor cells for use with autologous bone marrow after high-dose chemotherapy.

Journal Article Eur J Haematol · November 1995 Peripheral blood progenitor cells (PBPCs) were collected without prior association with chemotherapy but after the administration of granulocyte-macrophage colony-stimulating factor (GM-CSF) produced in Chinese hamster ovary cells (CHO-GM, regramostim), Es ... Full text Link to item Cite

Human recombinant stem-cell factor induces melanocytic hyperplasia in susceptible patients.

Journal Article J Am Acad Dermatol · October 1995 BACKGROUND: Recombinant human stem-cell factor (SCF), a cytokine acting on hematopoietic progenitor cells, has potential for the treatment of several hematologic and oncologic disorders. In a hematology-oncology phase I trial of SCF, several patients had c ... Full text Link to item Cite

Cyclophosphamide in combination with sargramostim for treatment of recurrent medulloblastoma.

Journal Article Med Pediatr Oncol · September 1995 Thirteen patients with recurrent medulloblastoma were treated with cyclophosphamide in association with Sargramostim. Cyclophosphamide was given at doses ranging between 1.0-2.5 g/m2 daily for two doses. Sargramostim was given at a fixed dose of 250 microg ... Full text Link to item Cite

Transplantation of cord-blood cells.

Journal Article N Engl J Med · July 6, 1995 Full text Link to item Cite

Effects of granulocyte-macrophage colony stimulating factor produced in Chinese hamster ovary cells (regramostim), Escherichia coli (molgramostim) and yeast (sargramostim) on priming peripheral blood progenitor cells for use with autologous bone marrow after high-dose chemotherapy.

Journal Article Eur J Haematol · May 1995 Peripheral blood progenitor cells (PBPCs) were collected without prior association with chemotherapy but after the administration of granulocyte-macrophage colony-stimulating factor (GM-CSF) produced in Chinese hamster ovary cells (CHO-GM, regramostim), Es ... Full text Link to item Cite

Dose escalation trial of cyclophosphamide with Sargramostim in the treatment of central nervous system (CNS) neoplasms.

Journal Article Med Pediatr Oncol · April 1995 We conducted a dose escalation trial of cyclophosphamide plus Sargramostim in the therapy of patients with newly diagnosed or recurrent central nervous system tumors. Cyclophosphamide was administered at doses ranging between 1.0 and 2.5 g/m2 daily for two ... Full text Link to item Cite

An open-label, multicenter study of polyethylene glycol-L-asparaginase for the treatment of acute lymphoblastic leukemia.

Journal Article Cancer · March 1, 1995 BACKGROUND: L-asparaginase has been a mainstay of therapy along with vincristine and prednisone in the treatment of acute lymphoblastic leukemia (ALL) in children for almost 30 years. Because L-asparaginase is a foreign protein, the potential exists for se ... Full text Link to item Cite

Disseminated histoplasmosis: a cause of infection-associated hemophagocytic syndrome.

Journal Article Am J Pediatr Hematol Oncol · November 1994 PURPOSE: We report a case of infection-associated hemophagocytic syndrome in the setting of disseminated histoplasmosis. PATIENTS AND METHODS: A 6-year-old boy with chronic mucocutaneous candidiasis developed a fulminant hemophagocytic syndrome. Evaluation ... Link to item Cite

The c-kit proto-oncogene receptor is expressed on a subset of human CD3-CD4-CD8- (triple-negative) thymocytes.

Journal Article Exp Hematol · September 1994 The c-kit receptor is a tyrosine-kinase transmembrane receptor first identified as an oncogene in the HZ4-feline leukemia virus and later found to be important in hematopoiesis in mice. The ligand for this receptor (Steel factor) can stimulate hematopoiesi ... Link to item Cite

CORD-BLOOD TRANSPLANTATION - AN UPDATE

Journal Article EXPERIMENTAL HEMATOLOGY · August 1, 1994 Link to item Cite

EXPRESSION OF CD44 ISOFORMS DURING ERYTHROID-DIFFERENTIATION

Journal Article CLINICAL RESEARCH · April 1, 1994 Link to item Cite

Idiopathic autoimmune thrombocytopenic purpura.

Journal Article Adv Pediatr · 1994 Childhood ITP occurs in acute and chronic forms. In general, both diseases are mild and can be managed conservatively. Approaches to therapy are highly controversial and vary widely among pediatricians and academic centers. Approximately 5% of children wit ... Link to item Cite

The use of umbilical cord blood in mismatched related and unrelated hemopoietic stem cell transplantation.

Journal Article Blood Cells · 1994 Over the past 6 years, umbilical cord blood has emerged as an efficacious alternative source of hematopoietic stem cells in related bone marrow transplantation. These encouraging results led us to extend this technology to the mismatched related and unrela ... Link to item Cite

Hematopoietic recovery following high-dose combined alkylating-agent chemotherapy and autologous bone marrow support in patients in phase-I clinical trials of colony-stimulating factors: G-CSF, GM-CSF, IL-1, IL-2, M-CSF.

Journal Article Ann Hematol · December 1993 Hematopoietic recovery in 115 patients with metastatic breast cancer or metastatic melanoma, enrolled in phase-I studies of recombinant growth factors while undergoing treatment with high-dose chemotherapy with autologous bone marrow support, was examined ... Full text Link to item Cite

Efficacy and toxicity of 9-beta-D-arabinofuranosylguanine (araG) as an agent to purge malignant T cells from murine bone marrow: application to an in vivo T-leukemia model.

Journal Article Leukemia · August 1993 9-beta-D-Arabinofuranosylguanine (araG), an analog of deoxyguanosine which is not degraded by purine nucleoside phosphorylase, has been previously shown in in vitro studies by our laboratory to be effective in purging malignant T cells from human bone marr ... Link to item Cite

Management of leukemic hyperleukocytosis with hydration, urinary alkalinization, and allopurinol. Are cranial irradiation and invasive cytoreduction necessary?

Journal Article Am J Pediatr Hematol Oncol · August 1993 PURPOSE: Hyperleukocytosis secondary to acute leukemia is a medical emergency. Intracranial hemorrhage often leads to death in this setting. Early efforts to prevent this serious complication have included emergent cranial irradiation, with its associated ... Link to item Cite

Guanine arabinoside as a bone marrow-purging agent.

Journal Article Ann N Y Acad Sci · June 23, 1993 Arabinosylguanine (araG) is a nucleoside analogue that is rapidly converted by cells of the T lymphoid lineage to its corresponding arabinosylguanine nucleotide triphosphate (araGTP), resulting in inhibition of DNA synthesis and selective in vitro toxicity ... Full text Link to item Cite

Role of cytokines in autologous bone marrow transplantation.

Journal Article Hematol Oncol Clin North Am · June 1993 High-dose chemotherapy with autologous bone marrow support has been increasingly used in the treatment of malignant disease. The use of hematopoietic colony-stimulating factors (CSF), particularly when coupled with the use of CSF-primed peripheral blood pr ... Link to item Cite

High-dose chemotherapy and autologous bone marrow support as consolidation after standard-dose adjuvant therapy for high-risk primary breast cancer.

Journal Article J Clin Oncol · June 1993 PURPOSE: We studied high-dose cyclophosphamide, cisplatin, and carmustine (CPA/cDDP/BCNU) with autologous bone marrow support (ABMS) as consolidation after standard-dose adjuvant chemotherapy treatment of primary breast cancer involving 10 or more axillary ... Full text Link to item Cite

A randomized controlled phase III trial of recombinant human granulocyte colony-stimulating factor (filgrastim) for treatment of severe chronic neutropenia.

Journal Article Blood · May 15, 1993 Patients with idiopathic, cyclic, and congenital neutropenia have recurrent severe bacterial infections. One hundred twenty-three patients with recurrent infections and severe chronic neutropenia (absolute neutrophil count < 0.5 x 10(9)/L) due to these dis ... Link to item Cite

Comparative effects of granulocyte-macrophage colony-stimulating factor (GM-CSF) and granulocyte colony-stimulating factor (G-CSF) on priming peripheral blood progenitor cells for use with autologous bone marrow after high-dose chemotherapy.

Journal Article Blood · April 1, 1993 Two hematopoietic colony-stimulating factors, granulocyte colony-stimulating factor (G-CSF) and granulocyte-macrophage CSF (GM-CSF), have been shown to accelerate leukocyte and neutrophil recovery after high-dose chemotherapy and autologous bone marrow (BM ... Link to item Cite

Evaluation of synergy between carbovir and 3'-azido-2',3'-deoxythymidine for inhibition of human immunodeficiency virus type 1.

Journal Article Antimicrob Agents Chemother · January 1993 3'-Azido-2',3'-deoxythymidine and carbovir [racemic and (-) enantiomer] were evaluated individually and in combination for antiviral activity against human immunodeficiency virus type 1 replication and cytotoxicity in vitro. The combination of these drugs ... Full text Link to item Cite

Retroviral gene transduction of circulating progenitor cells in patients with metastatic breast cancer.

Journal Article Surg Oncol · 1993 The use of somatic gene therapy for the treatment of breast cancer has many potential applications. Because chemotherapeutic protocols for breast cancer are commonly limited by bone marrow toxicity, transduction of genes into pleuripotent stem cells may al ... Full text Link to item Cite

Development of a model of melphalan-induced gastrointestinal toxicity in mice.

Journal Article Cancer Chemother Pharmacol · 1993 The tolerated dose of melphalan is limited by bone marrow suppression; when this complication is ameliorated by bone marrow transplantation, the dose-limiting toxicity becomes gastrointestinal mucositis. No intervention to date has been successful in modul ... Full text Link to item Cite

Successful therapy for trilateral retinoblastoma.

Journal Article Am J Ophthalmol · July 15, 1992 Trilateral retinoblastoma, the intracranial malignancy associated with bilateral retinoblastoma, is an uncommon and clinically aggressive malignancy with a uniformly fatal outcome. Three children with newly diagnosed trilateral retinoblastoma were treated ... Full text Link to item Cite

Modulation of macrophage colony stimulating factor in cultured human retinal pigment epithelial cells.

Journal Article Exp Eye Res · April 1992 Steady-state mRNA expression and protein production of macrophage colony stimulating factor were measured in visually confluent monolayers of unstimulated cultured human retinal pigment epithelial cells and after cells were stimulated with recombinant cyto ... Full text Link to item Cite

Alloimmune thrombocytopenia may be associated with systemic disease.

Journal Article Am J Obstet Gynecol · January 1992 Adverse outcome was encountered in a case of neonatal alloimmune thrombocytopenia after in utero platelet transfusion. This may have resulted from generalized systemic vascular endothelial damage, because several cell types, including umbilical endothelial ... Full text Link to item Cite

Reversal of radiation-induced neutropenia by granulocyte colony-stimulating factor.

Journal Article Med Pediatr Oncol · 1992 Myelosuppression is a common sequelae of radiotherapy, occasionally delaying the completion of treatment. In this report, we describe successful reversal of radiation-induced neutropenia in a child receiving craniospinal irradiation by granulocyte colony-s ... Full text Link to item Cite

The incidence of zoster after immunization with live attenuated varicella vaccine. A study in children with leukemia. Varicella Vaccine Collaborative Study Group.

Journal Article N Engl J Med · November 28, 1991 BACKGROUND: The Oka strain of live attenuated varicella vaccine is immunogenic and highly protective, but there has been concern about the risk of zoster after immunization. METHODS: We examined the incidence of zoster, risk factors for it, and measures of ... Full text Link to item Cite

Pharmacologic purging of malignant T cells from human bone marrow using 9-beta-D-arabinofuranosylguanine.

Journal Article Transplantation · October 1991 Arabinosylguanine (araG) is a nucleoside analog that is rapidly converted by cells of the T lymphoid lineage to its corresponding arabinosylguanine nucleotide triphosphate, resulting in inhibition of DNA synthesis and selective in vitro toxicity to T lymph ... Full text Link to item Cite

Non-Hodgkin's lymphoma. Biologic classification and implication for therapy.

Journal Article Pediatr Clin North Am · April 1991 The non-Hodgkin's lymphomas are a group of diseases for which substantial progress has been made in understanding tumor biology and effectiveness of treatment during the last decade. These advances may provide insight into the development of neoplasms beca ... Full text Link to item Cite

Isolation and characterization of the genomic human CD7 gene: structural similarity with the murine Thy-1 gene.

Journal Article Proc Natl Acad Sci U S A · January 15, 1991 The human CD7 molecule is a 40-kDa member of the immunoglobulin gene superfamily that is expressed on T-lymphoid and myeloid precursors in fetal liver and bone marrow. CD7 is also expressed on T lymphocytes in multiple stages of T-cell development, includi ... Full text Link to item Cite

Umbilical cord blood hematopoietic stem and repopulating cells in human clinical transplantation.

Journal Article Blood Cells · 1991 This paper reviews our recent laboratory and clinical studies demonstrating the efficacious use of human umbilical cord blood for HLA-matched allogeneic sibling stem/progenitor cell transplantation in cases of Fanconi's anemia. Future implications and pote ... Link to item Cite

Vincristine therapy for severe platelet alloimmunization.

Journal Article Am J Pediatr Hematol Oncol · 1991 A 19-month-old girl with idiopathic severe aplastic anemia refractory to multi-agent immunosuppressive therapy developed severe platelet alloimmunization following several months of platelet transfusions. She became refractory to human leukocyte antigen (H ... Full text Link to item Cite

Differential toxicity of carbovir and AZT to human bone marrow hematopoietic progenitor cells in vitro.

Journal Article Exp Hematol · November 1990 We have studied the in vitro effects of carbovir as compared to 3'-azido-3'-deoxythymidine (AZT) on the growth of clonal bone marrow-derived hematopoietic progenitor cells. (-)Carbovir, the enantiomer with anti-human immunodeficiency virus (HIV) activity, ... Link to item Cite

Evidence for susceptibility of intrathymic T-cell precursors and their progeny carrying T-cell antigen receptor phenotypes TCR alpha beta + and TCR gamma delta + to human immunodeficiency virus infection: a mechanism for CD4+ (T4) lymphocyte depletion.

Journal Article Proc Natl Acad Sci U S A · October 1990 Individuals infected by the human immunodeficiency virus type 1 (HIV-1) demonstrate progressive depletion and qualitative dysfunction of the helper T4 (CD4+) cell population. Mechanisms proposed for attrition of CD4+ T cells include direct cytopathicity of ... Full text Link to item Cite

Paradoxical expression of adenosine deaminase in T cells cultured from a patient with adenosine deaminase deficiency and combine immunodeficiency.

Journal Article J Clin Invest · August 1990 T lymphocytes cultured from a patient (T.D.) with adenosine deaminase (ADA) deficiency expressed ADA activity in the normal range, inconsistent with her severe immunodeficiency, metabolic abnormalities, and with the absence of ADA activity in her B lymphoc ... Full text Link to item Cite

Elimination of malignant clonogenic T cells from human bone marrow using chemoimmunoseparation with 2'-deoxycoformycin, deoxyadenosine and an immunotoxin.

Journal Article Bone Marrow Transplant · June 1990 Autologous bone marrow transplantation may contribute to the treatment of several types of lymphoreticular malignancies. Recent studies have suggested that a combination of chemoseparation and immunoseparation may be more effective than either modality alo ... Link to item Cite

Human umbilical cord blood: a clinically useful source of transplantable hematopoietic stem/progenitor cells.

Journal Article Int J Cell Cloning · January 1990 This is a review and discussion of studies leading to the first use of human umbilical cord blood, material usually discarded, for the provision of stem/progenitor cells for clinical hematopoietic reconstitution. This prospect arose as a result of extensiv ... Full text Link to item Cite

Papillary adenocarcinoma of the renal pelvis in a child: case report and brief review of the literature.

Journal Article Med Pediatr Oncol · 1990 Tumors of the renal pelvis are not common in adults and are extremely rare in children. Forty-eight cases of adenocarcinoma of the renal pelvis have been reported, with only one patient under 32 years of age. We report the youngest patient (an 11 year old ... Full text Link to item Cite

Immature human thymocytes can be driven to differentiate into nonlymphoid lineages by cytokines from thymic epithelial cells.

Journal Article Proc Natl Acad Sci U S A · October 1989 Featured Publication The signals and cellular interactions required for hematopoietic stem-cell commitment to the T lineage are unknown, yet are central to understanding the early stages of normal T-cell development. To study the differentiative capacity of T-cell precursors, ... Full text Link to item Cite

Involvement of the TCL5 gene on human chromosome 1 in T-cell leukemia and melanoma.

Journal Article Proc Natl Acad Sci U S A · July 1989 We analyzed a t(1;14)(p32;q11) chromosomal translocation in a human lymphohemopoietic stem cell line derived from a patient with acute T-lymphoblastic leukemia. The chromosomal joining on the 1p+ chromosome occurred at the T-cell receptor delta diversity ( ... Full text Link to item Cite

Pulmonary septic emboli mimicking metastatic rhabdomyosarcoma.

Journal Article J Pediatr Surg · May 1989 Central venous catheters have proven to be an important aid for the care of pediatric patients with malignancies receiving chemotherapy. A rare complication of such catheters is pulmonary septic emboli. This report describes a 15-year-old white girl with r ... Full text Link to item Cite

Human postnatal CD4- CD8- CD3- thymic T cell precursors differentiate in vitro into T cell receptor delta-bearing cells.

Journal Article J Immunol · May 1, 1989 The signals required for activation and the differentiation of human triple negative postnatal thymocytes were studied in vitro. Highly purified populations of CD4-, CD8-, CD3- (triple negative) thymocytes were isolated by combined panning and preparative ... Link to item Cite

Ontogeny of T-cell precursors: a model for the initial stages of human T-cell development.

Journal Article Immunol Today · March 1989 Although most investigators agree that the CD4- CD8- CD3- thymocyte subset represents the most immature intrathymic T cell capable of repopulating the thymus in vivo, little is known of the earliest stages of human T-cell development. Using mAbs to hematop ... Full text Link to item Cite

Chromosomal translocation in a human leukemic stem-cell line disrupts the T-cell antigen receptor delta-chain diversity region and results in a previously unreported fusion transcript.

Journal Article Proc Natl Acad Sci U S A · March 1989 We have studied a leukemic stem-cell line, DU.528, that is able to differentiate into myeloid and lymphoid cells. The leukemic cells have a translocation between chromosomes 1 and 14, t(1;14)(p33;q11), which we have molecularly cloned and sequenced. Initia ... Full text Link to item Cite

CD7+, CD4-, CD8- acute leukemia: a syndrome of malignant pluripotent lymphohematopoietic cells.

Journal Article Blood · February 1989 Featured Publication Following our initial observation of in vivo conversion of CD7+, CD4-, CD8- acute lymphoblastic leukemia (ALL) cells from lymphoid to myeloid lineages (Proc Natl Acad Sci (USA) 81:253, 1984) we have studied eight additional cases of ALL with this leukemic ... Link to item Cite

Criteria for termination of phase II chemotherapy for patients with progressive or recurrent brain tumor.

Journal Article Neurology · January 1989 We report six patients with progressive primary tumors of the brain who had prolonged periods with stable contrast-enhancing CT lesions following initial responses to chemotherapy. Chemotherapy was discontinued after 21 to 36 months, despite the persistenc ... Full text Link to item Cite

Human postnatal CD4- CD8- CD3- thymic T cell precursors differentiate in vitro into T cell receptor δ-bearing cells

Journal Article Journal of Immunology · 1989 The signals required for activation and the differentiation of human triple negative postnatal thymocytes were studied in vitro. Highly purified populations of CD4-, CD8-, CD3- (triple negative) thymocytes were isolated by combined panning and preparative ... Cite

Melphalan transport, glutathione levels, and glutathione-S-transferase activity in human medulloblastoma.

Journal Article Cancer Res · October 1, 1988 Melphalan transport, glutathione levels, and glutathione-S-transferase activity were measured in two continuous human medulloblastoma cell lines and transplantable xenografts in athymic nude mice, TE-671 and Daoy. In vitro mean glutathione levels were 10.0 ... Link to item Cite

Early events in human T cell ontogeny. Phenotypic characterization and immunohistologic localization of T cell precursors in early human fetal tissues.

Journal Article J Exp Med · September 1, 1988 During early fetal development, T cell precursors home from fetal yolk sac and liver to the epithelial thymic rudiment. From cells that initially colonize the thymus arise mature T cells that populate T cell zones of the peripheral lymphoid system. Whereas ... Full text Link to item Cite

Human thymic epithelial cells produce granulocyte and macrophage colony-stimulating factors.

Journal Article J Immunol · August 15, 1988 The development of culture conditions for growing normal human thymic epithelial (TE) cells free from contamination with other stromal cells has allowed us to identify and characterize TE cell-derived cytokines. In this study, we report that cultured human ... Link to item Cite

Human thymic epithelial cells directly induce activation of autologous immature thymocytes.

Journal Article Proc Natl Acad Sci U S A · May 1988 To study the role that epithelial cells of the thymic microenvironment play in promoting activation of immature CD7+, CD2+, CD4-, CD8- (double-negative) human thymocytes, we have isolated thymocyte subsets from normal postnatal thymus and have cocultured a ... Full text Link to item Cite

Effect of recombinant human granulocyte-macrophage colony-stimulating factor on hematopoietic reconstitution after high-dose chemotherapy and autologous bone marrow transplantation.

Journal Article N Engl J Med · April 7, 1988 Recombinant human granulocyte-macrophage colony-stimulating factor (rHuGM-CSF) has been reported to increase the leukocyte count in subhuman primates subjected to total-body irradiation and in patients with the acquired immunodeficiency syndrome. We admini ... Full text Link to item Cite

HUMAN THYMIC EPITHELIAL-CELLS PRODUCE GRANULOCYTE AND MACROPHAGE COLONY-STIMULATING FACTORS

Journal Article JOURNAL OF INVESTIGATIVE DERMATOLOGY · April 1, 1988 Link to item Cite

Congenital Pelger-Huet anomaly in triplets.

Journal Article Am J Hematol · March 1988 Congenital Pelger-Huet anomaly is an autosomal dominant disorder characterized by incomplete nuclear segmentation of granulocytes. The morphology of these mature but hyposegmented granulocytes may be confused with that of immature granulocytes (bands), the ... Full text Link to item Cite

Phenotypic and genotypic analysis of a human medulloblastoma cell line and transplantable xenograft (D341 Med) demonstrating amplification of c-myc.

Journal Article Am J Pathol · March 1988 D341 Med is a new continuous cell line and transplantable xenograft derived from a cerebellar medulloblastoma. This line grew in vitro in suspension culture with spontaneous macroscopic spheroid formation and demonstrated 20-fold amplification of c-myc. Cu ... Link to item Cite

A PHASE-II STUDY OF POLYETHYLENE-GLYCOL (PEG) CONJUGATED L-ASPARAGINASE IN PATIENTS WITH REFRACTORY ACUTE LEUKEMIAS

Journal Article PROCEEDINGS OF THE AMERICAN ASSOCIATION FOR CANCER RESEARCH · March 1, 1988 Link to item Cite

The coenrichment of stem cells, prothymocytes, and stromal elements with ecotropic retrovirus-producing cells from the bone marrow of leukemia-prone AKR mice.

Journal Article Virology · February 1988 Ecotropic virus-producing cells in the bone marrow of the leukemia-prone AKR strain of mice were significantly enriched by fractionation on discontinuous density gradients of Percoll and were found in a low-density population of cells comprised predominant ... Full text Link to item Cite

Childhood hepatic mesenchymoma: successful treatment with surgery and multiple-agent chemotherapy.

Journal Article Med Pediatr Oncol · 1988 Malignant undifferentiated sarcoma (mesenchymoma) of the liver is a rare tumor of childhood, with fewer than 100 cases reported in the literature. The overall prognosis for patients with this malignancy is poor. In this report, two children with hepatic me ... Full text Link to item Cite

Yersinia enterocolitica: A clinical presentation mimicking neuroblastoma

Journal Article Journal of Pediatric Hematology/Oncology · January 1, 1988 We describe a girl with 4 weeks of abdominal pain, fever, and weight loss, in whom a 2-cm pararenal mass was found, consistent with neuroblastoma. Gastrointestinal work-up revealedYersinia enterocoliticain her stool, and she was treated with antibiotic the ... Full text Cite

A PHASE-II STUDY OF POLYETHYLENE-GLYCOL (PEG) CONJUGATED L-ASPARAGINASE IN PATIENTS WITH REFRACTORY ACUTE LEUKEMIAS

Journal Article BIOMATERIALS ARTIFICIAL CELLS AND ARTIFICIAL ORGANS · January 1, 1988 Link to item Cite

Elimination of clonogenic malignant human T cells using monoclonal antibodies in combination with 2'-deoxycoformycin.

Journal Article J Clin Oncol · December 1987 2'Deoxycoformycin (dCF) specifically inhibits adenosine deaminase (ADA) and causes selective cytotoxicity of normal and malignant T cells. In clinical trials, dCF caused rapid lysis of malignant T lymphoblasts. Although dCF has been associated with dose-li ... Full text Link to item Cite

Efficacy of intravenous gamma globulin in autoimmune-mediated pediatric blood dyscrasias.

Journal Article Am J Med · October 23, 1987 Sixteen pediatric patients diagnosed with a variety of autoimmune-mediated hematocytopenias were treated with one to 50 courses of intravenous gamma globulin (IVIG), pH 4.25, over the course of one to 30 months. Thirteen patients had immune thrombocytopeni ... Full text Link to item Cite

Treatment of platelet alloimmunization with intravenous immunoglobulin. Two case reports and review of the literature.

Journal Article Am J Med · October 23, 1987 Two pediatric patients with severe aplastic anemia, elevated antiplatelet antibody levels, refractoriness to human lymphocyte antigen-matched platelet transfusions, and sustained bleeding problems were treated with intravenous immunoglobulin (IVIG), pH 4.2 ... Full text Link to item Cite

Combined chemoseparation and immunoseparation of clonogenic T lymphoma cells from human bone marrow using 2'-deoxycoformycin, deoxyadenosine, 3A1 monoclonal antibody, and complement.

Journal Article Cancer Res · September 1, 1987 Chemoseparation and immunoseparation techniques have been combined to eliminate malignant clonogenic T lymphoma cells from human bone marrow. Incubation with 5 microM 2'-deoxycoformycin and 500 microM deoxyadenosine has eliminated 2 logs of HSB-2 T lymphom ... Link to item Cite

Studies of human bone marrow treated with soybean lectin and sheep erythrocytes: stepwise analysis of cell morphology, phenotype and function.

Journal Article Clin Exp Immunol · June 1987 Morphological, phenotypic and functional analyses were made of cells obtained at each step after successive treatments of 23 separate human bone marrow suspensions with soybean lectin and sheep erythrocytes (SRBC). The average total number of nucleated cel ... Link to item Cite

Treatment of adenosine deaminase deficiency with polyethylene glycol-modified adenosine deaminase.

Journal Article N Engl J Med · March 5, 1987 We treated two children who had adenosine deaminase deficiency and severe combined immunodeficiency disease by injecting bovine adenosine deaminase modified by conjugation with polyethylene glycol. The modified enzyme was rapidly absorbed after intramuscul ... Full text Link to item Cite

BONE-MARROW PURGING WITH ANTIBODY AND COMPLEMENT - AN AMERICAN REVIEW

Journal Article BONE MARROW TRANSPLANTATION · January 1, 1987 Link to item Cite

Treatment of lymphohistiocytic erythrophagocytosis with VP-16 and aziridinylbenzoquinone.

Journal Article Med Pediatr Oncol · 1987 Lymphohistiocytic erythrophagocytosis (LE) is a usually fatal disease characterized by fever, organomegaly, hyperlipidemia, central nervous system involvement, and cellular immunodeficiency. Treatment with corticosteroids, cytotoxic chemotherapy, and blood ... Full text Link to item Cite

Management of human immunodeficiency virus-associated thrombocytopenia with intravenous gamma globulin.

Journal Article Am J Pediatr Hematol Oncol · 1987 A 17-month-old boy in whom immune-mediated thrombocytopenia (ITP) was the presenting manifestation of infection with human immunodeficiency virus (HIV) is being successfully managed with intermittent high-dose intravenous gamma globulin (IVIG) allowing mai ... Full text Link to item Cite

BIOLOGY OF THE NORMAL AND LEUKEMIC CD-7+ STEM-CELL

Journal Article JOURNAL OF CELLULAR BIOCHEMISTRY · January 1, 1987 Link to item Cite

Treatment of trilateral retinoblastoma with vincristine and cyclophosphamide.

Journal Article Am J Ophthalmol · November 15, 1986 Current therapy for patients with trilateral retinoblastoma, consisting primarily of surgical intervention and radiotherapy, has resulted in no long-term survivors. The use of adjuvant chemotherapy has not improved this outcome. After observing a tumor res ... Full text Link to item Cite

Immunoglobulin and T-cell receptor gene rearrangement and expression in human lymphoid leukemia cells at different stages of maturation.

Journal Article Proc Natl Acad Sci U S A · November 1986 The use of probes to genes (IG and TCRB) encoding immunoglobulins (IG) and the beta chain of the T-cell antigen receptor (TCRB), respectively, have become a sensitive means to assess clonality and lineage in lymphoid malignancies. It has become apparent th ... Full text Link to item Cite

9p- in a girl with acute lymphocytic leukemia and sickle cell disease.

Journal Article Cancer Genet Cytogenet · April 1, 1986 A 4 year old girl with sickle cell disease developed acute lymphocytic leukemia null cell type. The bone marrow karyotype was 46,XX,del(9)(p13). This girl is among the few patients with acute lymphocytic leukemia and abnormalities of #9 to have an isolated ... Full text Link to item Cite

Severe Mycoplasma pneumonia in three sisters with sickle cell disease.

Journal Article Pediatr Hematol Oncol · 1986 Mycoplasma pneumoniae, which usually causes mild infections in normal children, has been shown to cause isolated cases of severe pneumonia in children with sickle cell disease. We recently observed an outbreak of Mycoplasma pneumonia in three sisters with ... Full text Link to item Cite

Establishment of the DU.528 human lymphohemopoietic stem cell line.

Journal Article J Exp Med · November 1, 1985 We have established the DU.528 cell line from the pretreatment leukemia cells of a patient who underwent a T lymphoblastic-to-promyelocytic phenotype conversion during treatment with the adenosine deaminase inhibitor, deoxycoformycin. The cell line and clo ... Full text Link to item Cite

Determinants of deoxyadenosine toxicity in hybrids between human T- and B- lymphoblasts as a model for the development of drug resistance in T-cell acute lymphoblastic leukemia.

Journal Article Cancer Res · April 1985 Cultured human T-lymphoblastoid cell lines are more sensitive than B-cell lines to 2'-deoxyadenosine in the presence of 2'-deoxycoformycin, a potent inhibitor of adenosine deaminase. This difference is related to the greater efficiency with which T-lymphob ... Link to item Cite

SELECTIVE REMOVAL OF MALIGNANT B-CELL AND T-CELL NEOPLASMS FROM HUMAN-BONE MARROW

Journal Article INTERNATIONAL JOURNAL OF CELL CLONING · January 1, 1985 Link to item Cite

Retinoblastoma metastatic to bone marrow. A case with florid spread.

Journal Article Clin Pediatr (Phila) · March 1984 A 28-month-old white male, 7 months status postenucleation for retinoblastoma, presented with lethargy, irritability, anemia, and thrombocytopenia. Bone marrow examination revealed almost total replacement of marrow elements with clumps of small round baso ... Full text Link to item Cite

Cisplatinum-induced renal salt wasting.

Journal Article Med Pediatr Oncol · 1984 We report two patients who developed high urinary excretion of sodium chloride after receiving cisplatinum. One patient became symptomatically hyponatremic during intravenous administration of hypotonic fluid. The second patient developed orthostatic hypot ... Full text Link to item Cite

Conversion of a stem cell leukemia from a T-lymphoid to a myeloid phenotype induced by the adenosine deaminase inhibitor 2'-deoxycoformycin.

Journal Article Proc Natl Acad Sci U S A · January 1984 Selective failure of lymphoid development occurs in genetic deficiency of adenosine deaminase (ADA). We examined the in vivo effects of a potent inhibitor of ADA, 2'-deoxycoformycin, which was used to treat a patient with refractory acute leukemia. Unexpec ... Full text Link to item Cite

THE ESTABLISHMENT OF A LYMPHOHEMOPOIETIC STEM-CELL LINE, A NEW MODEL FOR THE STUDY OF CELLULAR-DIFFERENTIATION

Conference PROCEEDINGS OF THE AMERICAN ASSOCIATION FOR CANCER RESEARCH · January 1, 1984 Link to item Cite

S-adenosylhomocysteine catabolism and basis for acquired resistance during treatment of T-cell acute lymphoblastic leukemia with 2'-deoxycoformycin alone and in combination with 9-beta-D-arabinofuranosyladenine.

Journal Article Cancer Res · July 1983 A patient with refractory T-cell acute lymphoblastic leukemia was treated with eight courses of the adenosine deaminase inhibitor, 2'-deoxycoformycin (dCF), over a 5-month period. After developing resistance to dCF, he responded to treatment with the combi ... Link to item Cite

CONVERSION OF T-CELL TO MYELOID-LEUKEMIA BY DEOXYCOFORMYCIN (DCF), A POTENT ADENOSINE-DEAMINASE (ADA) INHIBITOR

Journal Article PROCEEDINGS OF THE AMERICAN ASSOCIATION FOR CANCER RESEARCH · January 1, 1983 Link to item Cite