Scholars@Duke
Vinod K. Prasad

Vinod K. Prasad

Medical Professor in the Department of Pediatrics
Pediatrics, Transplant and Cellular Therapy
vinod.prasad@duke.edu
2400 Pratt Street, Durham, NC 27705
2400 Pratt Street, Room 8044, Durham, NC 27705

Selected Publications

Umbilical Cord Blood Transplantation Provides an Alternative for Patients With Chronic Granulomatous Disease Lacking HLA-Matched Donors: A PIDTC Report.

Journal Article Transplant Cell Ther · October 2, 2025 BACKGROUND: Allogeneic hematopoietic cell transplantation corrects the phagocytic defect in patients with chronic granulomatous disease (CGD) and resolves infection risk and immune dysregulation. Umbilical cord blood transplantation (UCBT) is an option for ... Full text Link to item Cite

Newborn Screening for Hurler Syndrome Facilitates Early Transplant and Good Outcomes.

Journal Article Pediatr Neurol · February 2025 BACKGROUND: Hematopoietic cell transplantation (HCT) is the standard of care treatment for children with Hurler syndrome (HS). This study describes the impact of newborn screening (NBS) on HCT outcomes for these patients. METHODS: Retrospective study of HS ... Full text Link to item Cite

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

Journal Article Stem Cells Transl Med · January 17, 2025 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

Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy.

Journal Article N Engl J Med · October 10, 2024 BACKGROUND: Gene therapy with elivaldogene autotemcel (eli-cel) consisting of autologous CD34+ cells transduced with lentiviral vector containing ABCD1 complementary DNA (Lenti-D) has shown efficacy in clinical studies for the treatment of cerebral adrenol ... Full text Link to item Cite

Allogeneic hematopoietic cell transplantation is effective for p47phox chronic granulomatous disease: A  Primary Immune Deficiency Treatment Consortium study.

Journal Article J Allergy Clin Immunol · May 2024 BACKGROUND: P47phox (neutrophil cytosolic factor-1) deficiency is the most common cause of autosomal recessive chronic granulomatous disease (CGD) and is considered to be associated with a milder clinical phenotype. Allogeneic hematopoietic cell transplant ... Full text Link to item Cite

Genotype, oxidase status, and preceding infection or autoinflammation do not affect allogeneic HCT outcomes for CGD.

Journal Article Blood · December 14, 2023 Chronic granulomatous disease (CGD) is a primary immunodeficiency characterized by life-threatening infections and inflammatory conditions. Hematopoietic cell transplantation (HCT) is the definitive treatment for CGD, but questions remain regarding patient ... Full text Link to item Cite

Optical Coherence Tomography Findings in Cherry-Red Spot: Implications for Understanding Pathophysiology and Visual Prognosis.

Journal Article J Pediatr Ophthalmol Strabismus · 2023 PURPOSE: To report optical coherence tomography (OCT) findings of cherry-red spots from Tay-Sachs and Niemann-Pick disease. METHODS: Consecutive patients with Tay-Sachs and Niemann-Pick disease evaluated by the pediatric transplant and cellular therapy tea ... Full text Link to item Cite

Variables affecting outcomes after allogeneic hematopoietic stem cell transplant for cerebral adrenoleukodystrophy.

Journal Article Blood Adv · March 8, 2022 Allogeneic hematopoietic stem cell transplantation (allo-HSCT) in early cerebral adrenoleukodystrophy can stabilize neurologic function and improve survival but has associated risks including transplant-related mortality (TRM), graft failure, and graft-ver ... Full text Link to item Cite

Correction: Chronic Granulomatous Disease-Associated IBD Resolves and Does Not Adversely Impact Survival Following Allogeneic HCT.

Journal Article J Clin Immunol · November 2020 The original version of this article unfortunately contained the missing author, Caridad Martinez. The authors would like to correct the list. We apologize for any inconvenience that this may have caused. The correct author list is shown above. ... Full text Link to item Cite

Use of Letermovir for Salvage Therapy for Resistant Cytomegalovirus in a Pediatric Hematopoietic Stem Cell Transplant Recipient.

Journal Article J Pediatric Infect Dis Soc · September 17, 2020 We present here the first published use of letermovir for the treatment of resistant cytomegalovirus (CMV) in a pediatric patient. A 14-year-old girl underwent a double unrelated umbilical cord blood transplantation to treat her sickle cell disease (hemogl ... Full text Open Access Link to item Cite

Practice patterns and incidence of adenovirus infection in allogeneic hematopoietic cell transplant recipients: Multicenter survey of transplant centers in the United States.

Journal Article Transpl Infect Dis · August 2020 BACKGROUND: Adenovirus (AdV) is increasingly recognized as a threat to successful outcomes after allogeneic hematopoietic cell transplantation (allo-HCT). Guidelines have been developed to inform AdV screening and treatment practices, but the extent to whi ... Full text 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

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

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

Chronic Granulomatous Disease-Associated IBD Resolves and Does Not Adversely Impact Survival Following Allogeneic HCT.

Journal Article J Clin Immunol · October 2019 INTRODUCTION: Inflammatory bowel disease (IBD) affects approximately 1/3 of patients with chronic granulomatous disease (CGD). Comprehensive investigation of the effect of allogeneic hematopoietic cell transplantation (HCT) on CGD IBD and the impact of IBD ... Full text Link to item Cite

Higher Risks of Toxicity and Incomplete Recovery in 13- to 17-Year-Old Females after Marrow Donation: RDSafe Peds Results.

Journal Article Biol Blood Marrow Transplant · May 2019 Although donation of bone marrow (BM) or peripheral blood stem cells (PBSCs) from children to family members undergoing allogeneic transplantation are well-established procedures, studies detailing levels of pain, symptoms, and long-term recovery are lacki ... Full text Link to item Cite

Related peripheral blood stem cell donors experience more severe symptoms and less complete recovery at one year compared to unrelated donors.

Journal Article Haematologica · April 2019 Unlike unrelated donor registries, transplant centers lack uniform approaches to related donor assessment and deferral. To test whether related donors are at increased risk for donation-related toxicities, we conducted a prospective observational trial of ... Full text Link to item Cite

Effect of Aging and Predonation Comorbidities on the Related Peripheral Blood Stem Cell Donor Experience: Report from the Related Donor Safety Study.

Journal Article Biol Blood Marrow Transplant · April 2019 The development of reduced-intensity approaches for allogeneic hematopoietic cell transplantation has resulted in growing numbers of older related donors (RDs) of peripheral blood stem cells (PBSCs). The effects of age on donation efficacy, toxicity, and l ... Full text 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

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

Outcomes of Umbilical Cord Blood Transplantation in Children with Batten Disease

Conference BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION · 2019 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

Brincidofovir (CMX001) Toxicity Associated With Epithelial Apoptosis and Crypt Drop Out in a Hematopoietic Cell Transplant Patient: Challenges in Distinguishing Drug Toxicity From GVHD.

Journal Article J Pediatr Hematol Oncol · August 2018 Brincidofovir (CMX001) is an oral agent with activity against double-strand DNA viruses undergoing clinical trials in immunocompromised patients. We report a patient clinically diagnosed with brincidofovir-related gastrointestinal (GI) toxicity and his his ... Full text Link to item Cite

Disseminated Adenovirus Infection After Combined Liver-Kidney Transplantation.

Journal Article Front Cell Infect Microbiol · 2018 Human adenovirus (HAdV) infections are well-described after hematopoietic stem cell transplantation but less well understood in solid organ transplantation (SOT). We describe a case of disseminated HAdV type 21 infection 5 months after combined liver-kidne ... 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

First two bilateral hand transplantations in India (Part 1): From vision to reality

Journal Article Indian Journal of Plastic Surgery · May 1, 2017 Introduction: Vascularized composite tissue allotransplantation is a relatively new concept, which was unavailable in the Indian subcontinent till a bilateral hand transplant was carried out successfully in January 2015. Materials and Methods: The setting ... Full text 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

Extrapulmonary Aspergillus infection in patients with CARD9 deficiency.

Journal Article JCI Insight · October 20, 2016 Invasive pulmonary aspergillosis is a life-threatening mycosis that only affects patients with immunosuppression, chemotherapy-induced neutropenia, transplantation, or congenital immunodeficiency. We studied the clinical, genetic, histological, and immunol ... Full text 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

Bone Marrow Transplantation and Umbilical Cord Blood Transplantation for Inborn Errors of Metabolism

Journal Article BIRTH DEFECTS RESEARCH PART A-CLINICAL AND MOLECULAR TERATOLOGY · May 1, 2016 Link to item Cite

Survival after mesenchymal stromal cell therapy in steroid-refractory acute graft-versus-host disease: systematic review and meta-analysis.

Journal Article Lancet Haematol · January 2016 BACKGROUND: Graft-versus-host disease (GVHD) is the major limitation of allogeneic haemopoietic stem-cell transplantation (HSCT), for which no approved treatments are available. Use of mesenchymal stromal cells (MSCs) has become standard practice in some E ... Full text Link to item 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

Predictors of gastrostomy placement in children with inherited metabolic diseases treated by umbilical cord blood transplantation.

Journal Article J Pediatr Surg · July 2015 BACKGROUND/PURPOSE: Children with inherited metabolic diseases (IMDs) undergoing umbilical cord blood transplantation (UCBT) who are at risk for post-transplant failure to thrive may benefit from pretransplant gastrostomy tube (GT) placement. Here we sough ... Full text 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

Nonpermissive HLA-DPB1 mismatch increases mortality after myeloablative unrelated allogeneic hematopoietic cell transplantation.

Journal Article Blood · October 16, 2014 We examined current outcomes of unrelated donor allogeneic hematopoietic cell transplantation (HCT) to determine the clinical implications of donor-recipient HLA matching. Adult and pediatric patients who had first undergone myeloablative-unrelated bone ma ... Full text 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

Umbilical Cord Blood Transplantation for Inherited Metabolic Diseases

Chapter · January 1, 2014 Inherited metabolic diseases (IMD) belonging to lysosomal storage disorders (Hurler syndrome, Krabbe disease, metachromatic leukodystrophy, and others) and peroxisomal disorders (adrenoleukodystrophy) cause progressive degeneration of the nervous system, n ... Full text Cite

Amino acid substitution at peptide-binding pockets of HLA class I molecules increases risk of severe acute GVHD and mortality.

Journal Article Blood · November 21, 2013 HLA disparity has a negative impact on the outcomes of hematopoietic cell transplantation (HCT). We studied the independent impact of amino acid substitution (AAS) at peptide-binding positions 9, 99, 116, and 156, and killer immunoglobulin-like receptor bi ... Full text Link to item Cite

Challenges and opportunities for international cooperative studies in pediatric hematopoeitic cell transplantation: priorities of the Westhafen Intercontinental Group.

Journal Article Biol Blood Marrow Transplant · September 2013 More than 20% of allogeneic hematopoietic cell transplantations (HCTs) are performed in children and adolescents at a large number of relatively small centers. Unlike adults, at least one-third of HCTs in children are performed for rare, nonmalignant indic ... Full text 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

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

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

Amino Acid Substitution At Peptide-Binding Pockets of HLA Class I Molecules Adversely Impacts Hematopoietic Cell Transplantation Outcomes

Conference Blood · November 16, 2012 AbstractAbstract 467Background:While donor-recipient disparity at HLA loci is associated with greater ... 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

Differential impact of inhibitory and activating Killer Ig-Like Receptors (KIR) on high-risk patients with myeloid and lymphoid malignancies undergoing reduced intensity transplantation from haploidentical related donors.

Journal Article Bone Marrow Transplant · June 2012 The impact of activating KIR (aKIR) and inhibitory KIR (iKIR) on OS, relapse-related mortality (RRM) and acute GVHD (aGVHD) was prospectively studied in 84 adults with high-risk hematologic malignancies receiving reduced intensity conditioning (RIC) T-cell ... Full text 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

Vitamin D Deficiency in Children Undergoing Hematopoietic Stem Cell Transplantation

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

Current international perspectives on hematopoietic stem cell transplantation for inherited metabolic disorders.

Journal Article Pediatr Clin North Am · February 2010 Inherited metabolic disorders (IMD) or inborn errors of metabolism are a diverse group of diseases arising from genetic defects in lysosomal enzymes or peroxisomal function. These diseases are characterized by devastating systemic processes affecting neuro ... 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

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

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 Featured Publication 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

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

Journal Article Biol Blood Marrow Transplant · May 2009 Featured Publication 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

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

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 Featured Publication 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 Featured Publication 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 Featured Publication 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

Pediatric hematopoietic stem cell transplantation and the role of imaging.

Journal Article Radiology · August 2008 Featured Publication The use of hematopoietic stem cell transplantation (HSCT) in the treatment of children afflicted with many potentially fatal malignant and nonmalignant diseases is well recognized. Although outcomes continue to improve and the utility of HSCT is increasing ... Full text Link to item Cite

Emerging trends in transplantation of inherited metabolic diseases.

Journal Article Bone Marrow Transplant · January 2008 Featured Publication 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

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

Journal Article Pediatr Blood Cancer · December 2007 Featured Publication 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

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

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

Journal Article Biol Blood Marrow Transplant · June 2007 Featured Publication 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

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

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

Cytomegalovirus ventriculoencephalitis in a bone marrow transplant recipient receiving antiviral maintenance: clinical and molecular evidence of drug resistance.

Journal Article Clin Infect Dis · November 1, 2001 Featured Publication We describe a case of CMV ventriculoencephalitis in a severely immunocompromised bone marrow transplant recipient who was receiving combination therapy with ganciclovir and foscarnet for treatment of viremia and retinitis. Analysis of sequential viral isol ... Full text Link to item Cite

Stem cell transplantation for the treatment of Fanconi anaemia using a fludarabine-based cytoreductive regimen and T-cell-depleted related HLA-mismatched peripheral blood stem cell grafts.

Journal Article Br J Haematol · December 2000 Featured Publication We have employed a new cytoreductive regimen to transplant two patients with Fanconi anaemia (FA), using T cell-depleted two HLA-allele disparate related peripheral blood stem cell transplants (PBSCTs). Patient 1, a 5-year-old male with FA and aplastic ana ... Full text Link to item Cite

Rolandic encephalopathy and epilepsia partialis continua following bone marrow transplant.

Journal Article Bone Marrow Transplant · October 2000 Featured Publication Epilepsia partialis continua (EPC) is a condition defined by prolonged focal myoclonus. Often resistant to therapy, EPC in children is frequently present in Rasmussen encephalitis, a form of chronic encephalitis of uncertain etiology. We discuss a child wh ... Full text Link to item Cite

The probability of HLA-C matching between patient and unrelated donor at the molecular level: estimations based on the linkage disequilibrium between DNA typed HLA-B and HLA-C alleles.

Journal Article Transplantation · October 15, 1999 Featured Publication BACKGROUND: Recent evidence suggests a more significant role of HLA-C as a target of alloreactions after bone marrow transplantation than previously suspected. Although linkage disequilibrium (LD) between HLA-B and -C serogroups is well documented, the lev ... Full text Link to item Cite

DNA typing for HLA-A and HLA-B identifies disparities between patients and unrelated donors matched by HLA-A and HLA-B serology and HLA-DRB1.

Journal Article Blood · January 1, 1999 Featured Publication High incidences of graft failure and graft-versus-host disease in the recipients of bone marrow transplantations (BMT) from unrelated donors (URD) may reflect the existence of allelic disparities between the patient and the URD despite apparent HLA identit ... Link to item Cite

HLA-C disparity between patients and unrelated donors matched for HLA-A, -B, and -DRB1 alleles: impact of serological vs. DNA typing for HLA-A and -B loci.

Journal Article Biol Blood Marrow Transplant · 1999 Featured Publication High incidences of graft failure, graft-vs.-host disease (GVHD), and serious infections following unrelated donor (URD) marrow transplantation, despite apparent human leukocyte antigen (HLA) identity, may reflect the presence of molecular disparities, incl ... Full text Link to item Cite

Hl.a-c disparity between patients and unrelated donors (urd) matched for hla-a, -b, and -drb1 alleles by dna typing

Journal Article Experimental Hematology · December 1, 1998 High incidences of complications including graft failure, GVHO. and serious infections following URD marrow transplantation despite apparent HLA identity at HLA-A, -B. and DRB1 loci may be due lo genetic disparities at HLA-C locus which currently has 42 al ... Cite

TTP following ITP in an HIV-positive boy.

Journal Article J Pediatr Hematol Oncol · November 1996 Featured Publication PURPOSE: To report the previously undescribed development of thrombotic thrombocytopenic purpura (TTP) in a human immunodeficiency virus (HIV)-positive child and discuss the differential diagnosis. PATIENT AND METHODS: Our patient was a 9-year-old boy with ... Full text Link to item Cite

Progressive glomerular toxicity of ifosfamide in children.

Journal Article Med Pediatr Oncol · September 1996 Featured Publication Glomerular toxicity following ifosfamide (IFO) is not as well recognized as renal tubular damage. Following a case of ifosfamide-induced renal failure with histological evidence of glomerular changes, we undertook a retrospective study of all IFO-treated c ... Full text Link to item Cite

Allele assignment for HLA-A, -B, and -C genes to the Tenth International Histocompatibility Workshop cell lines.

Journal Article Tissue Antigens · June 1996 Featured Publication Development of DNA typing for Class I HLA alleles has lagged behind that of class II for a variety of technical reasons. Following the recognition of locus specific sequences in the first and the third intron, and acquiring the ability to amplify genomic D ... Full text Link to item Cite

Treatment of hyperkalaemia using intravenous and nebulised salbutamol.

Journal Article Arch Dis Child · February 1994 Featured Publication In 11 children (aged 5-18 years) with end stage chronic renal failure, the effect on plasma potassium of two doses of salbutamol (separated by two hours) given intravenously (4 micrograms/kg) and on a separate date, of salbutamol administered by nebuliser ... Full text Link to item Cite

Ifosfamide enantiomers: pharmacokinetics in children.

Journal Article Cancer Chemother Pharmacol · 1994 Featured Publication Ifosfamide, like other oxazaphosphorine drugs, is chiral and there is some evidence, mainly from animal studies, of stereo-selective differences in metabolism, excretion and cytotoxic activity between the two enantiomers. The pharmacokinetics of racemic if ... Full text Link to item Cite

Tetanus neonatorum: clinico-epidemiological profile.

Journal Article Indian Pediatr · November 1988 Featured Publication Link to item Cite