Scholars@Duke
Mitchell Eric Horwitz

Mitchell Eric Horwitz

Professor of Medicine
Medicine, Hematologic Malignancies and Cellular Therapy
mitchell.horwitz@duke.edu
2400 Pratt St Suite 5000, DUMC Box 3961, Durham, NC 27710
2400 Pratt St Suite 5000, DUMC Box 3961, Durham, NC 27710

Selected Publications

Long-term survival after unrelated donor marrow transplantation for aplastic anaemia after optimized conditioning regimen: a retrospective multicentre cohort study.

Journal Article EClinicalMedicine · October 2024 BACKGROUND: Almost all acquired severe aplastic anaemia is immune mediated and characterised by hypocellular bone marrow and ≥2 affected haematopoietic lineages. The optimal preparartive regimen for unrelated donor transplantation remains to be established ... Full text Link to item Cite

Financial Toxicity and Quality of Life in Patients Undergoing Stem-Cell Transplant Evaluation: A Single-Center Analysis.

Journal Article JCO Oncol Pract · March 2024 PURPOSE: We investigated the prevalence of financial toxicity in a population undergoing hematopoietic cell transplantation (HCT) evaluation and measured its impact on post-transplant clinical and health-related quality-of-life outcomes. MATERIALS AND METH ... Full text Link to item Cite

Heightened TLR7 signaling primes BCR-activated B cells in chronic graft-versus-host disease for effector functions.

Journal Article Blood Adv · February 13, 2024 Chronic graft-versus-host disease (cGVHD) is a debilitating, autoimmune-like syndrome that can occur after allogeneic hematopoietic stem cell transplantation. Constitutively activated B cells contribute to ongoing alloreactivity and autoreactivity in patie ... Full text Open Access Link to item Cite

Targeting Hedgehog Signaling with Glasdegib in Patients with Refractory Sclerotic Chronic GVHD: A Report of Two Phase I/II Trials.

Journal Article Clin Cancer Res · October 13, 2023 PURPOSE: Sclerotic chronic GVHD (scGVHD) is characterized by progressive skin fibrosis and frequent refractoriness to available therapies. Aberrant activation of Hedgehog signaling in dermal fibroblasts has been implicated in scGVHD. Here, we report the re ... Full text Link to item Cite

Cell Therapy Informatics: Updates on the Integration of HCT/IEC Functionalities into an Electronic Medical Record System in the US to Promote Efficiency, Patient Safety, Research, and Data Interoperability.

Journal Article Transplant Cell Ther · September 2023 The use of electronic health/medical record (EMR) systems has streamlined medical practice and improved efficiency of clinical care in recent years. However, EMR systems are not generally well designed to support research and tracking of longitudinal outco ... Full text Link to item Cite

Immune Reconstitution Profiling Suggests Antiviral Protection after Transplantation with Omidubicel: A Phase 3 Substudy.

Journal Article Transplant Cell Ther · August 2023 Allogeneic hematopoietic cell transplantation (HCT) is a potentially curative treatment for hematologic malignancies and nonmalignant disorders. Rapid immune reconstitution (IR) following allogeneic HCT has been shown to be associated with improved clinica ... Full text Link to item Cite

Single-cell landscape analysis unravels molecular programming of the human B cell compartment in chronic GVHD.

Journal Article JCI insight · June 2023 Alloreactivity can drive autoimmune syndromes. After allogeneic hematopoietic stem cell transplantation (allo-HCT), chronic graft-versus-host disease (cGVHD), a B cell-associated autoimmune-like syndrome, commonly occurs. Because donor-derived B cells cont ... Full text Open Access Cite

A Phase I Trial of SYK Inhibition with Fostamatinib in the Prevention and Treatment of Chronic Graft-Versus-Host Disease.

Journal Article Transplant Cell Ther · March 2023 Despite the exciting advancement of novel therapies, chronic graft-versus-host disease (cGVHD) remains the most common cause of non-relapse mortality after allogeneic hematopoietic stem cell transplantation (HCT). Frontline treatment of cGVHD involves syst ... Full text Link to item Cite

NCCN Guidelines® Insights: Hematopoietic Cell Transplantation, Version 3.2022.

Journal Article J Natl Compr Canc Netw · February 2023 The NCCN Guidelines for Hematopoietic Cell Transplantation (HCT) provide an evidence- and consensus-based approach for the use of autologous and allogeneic HCT in the management of malignant diseases in adult patients. HCT is a potentially curative treatme ... Full text Link to item Cite

Health-Related Quality of Life Following Allogeneic Hematopoietic Cell Transplantation with Omidubicel versus Umbilical Cord Blood.

Conference Transplant Cell Ther · January 2023 Omidubicel is an advanced cell therapy derived from umbilical cord blood (UCB) for use in allogeneic hematopoietic cell transplantation (HCT). A recent randomized phase 3 clinical trial demonstrated faster engraftment, shorter length of hospital stays, and ... Full text Link to item Cite

Optimizing Management of the Central Nervous System in Patients with Acute Lymphoblastic Leukemia Undergoing Allogeneic Stem Cell Transplantation.

Journal Article Adv Radiat Oncol · 2023 PURPOSE: To evaluate clinical outcomes and patterns of failure, specifically in regards to the central nervous system (CNS), in patients with acute lymphoblastic leukemia (ALL) undergoing allogeneic hematopoietic stem cell transplantation (HSCT) using tota ... Full text Link to item Cite

Leveraging Natural Killer Cell Innate Immunity against Hematologic Malignancies: From Stem Cell Transplant to Adoptive Transfer and Beyond.

Journal Article Int J Mol Sci · December 22, 2022 Numerous recent advancements in T-cell based immunotherapies have revolutionized the treatment of hematologic malignancies. In the race towards the first approved allogeneic cellular therapy product, there is growing interest in utilizing natural killer (N ... Full text Link to item Cite

CT-039 Health-Related Quality of Life Following Allogeneic Hematopoietic Stem Cell Transplantation With Omidubicel Versus Standard Umbilical Cord Blood.

Journal Article Clin Lymphoma Myeloma Leuk · October 2022 CONTEXT: Omidubicel is an ex vivo expanded stem cell product derived from umbilical cord blood (UCB) for allogeneic hematopoietic stem cell transplantation (allo-HSCT). A multicenter phase III trial demonstrated faster engraftment and fewer infections with ... Full text 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

Haploidentical bone marrow transplantation in patients with relapsed or refractory severe aplastic anaemia in the USA (BMT CTN 1502): a multicentre, single-arm, phase 2 trial.

Journal Article Lancet Haematol · September 2022 BACKGROUND: Relapsed severe aplastic anaemia is a marrow failure disorder with high morbidity and mortality. It is often treated with bone marrow transplantation at relapse post-immunosuppressive therapy, but under-represented minorities often cannot find ... Full text Link to item Cite

Geriatric Assessment Reveals Actionable Impairments in Hematopoietic Stem Cell Transplantation Candidates Age 18 to 80 Years.

Journal Article Transplant Cell Ther · August 2022 Allogeneic hematopoietic stem cell transplantation (HCT) is a potentially curative treatment for both malignant and nonmalignant hematologic diseases; however, reported rates of treatment-related mortality approach 30%. Outcomes are worse in patients who b ... Full text Link to item Cite

Impact of Center Experience with Donor Type on Outcomes: A Secondary Analysis, Blood and Marrow Transplant Clinical Trials Network 1101Open for Accrual June 2012Open for Accrual June 2012.

Journal Article Transplant Cell Ther · July 2022 We previously reported the results of Blood and Marrow Transplant Clinical Trials Network (BMT CTN) 1101, a randomized comparison of hematopoietic cell transplantation (HCT) performed with double umbilical cord blood units (dUCB) or with haploidentical bon ... Full text Link to item Cite

The Use of Allogeneic Hematopoietic Stem Cell Transplantation in Primary Myelofibrosis.

Journal Article J Pers Med · April 2, 2022 Primary myelofibrosis (PMF) is a BCR-ABL1 negative myeloproliferative neoplasm characterized by clonal proliferation of myeloid cells. This leads to reactive bone marrow fibrosis, ultimately resulting in progressive marrow failure, hepatosplenomegaly, and ... Full text Link to item Cite

Home-Based Hematopoietic Cell Transplantation in the United States.

Journal Article Transplant Cell Ther · April 2022 Patients undergoing allogeneic (allo) and autologous (auto) hematopoietic cell transplantation (HCT) require extensive hospitalizations or daily clinic visits for the duration of their transplantation. Home HCT, wherein patients live at home and providers ... Full text Link to item Cite

Cognitive impairment in candidates for allogeneic hematopoietic stem cell transplantation.

Conference Bone Marrow Transplant · January 2022 Hematopoietic cell transplant (HCT) is an increasingly common and curative treatment strategy to improve survival among individuals with malignant and nonmalignant diseases, with over one million HCTs having been performed worldwide. Neurocognitive dysfunc ... Full text Link to item Cite

Impact of Center Experience with Donor Type and Treatment Platform on Outcomes: A Secondary Analysis BMT CTN 1101

Conference Blood · November 5, 2021 AbstractBACKGROUND: Our group recently reported on the results of Blood and Marrow Transplant (BMT) Clinical Trials Network (CTN) 1101 a randomized comparison between double umbilical cord blood (dUCB) and h ... Full text Cite

Hematopoietic Stem Cell Transplantation (HSCT) with Omidubicel Is Associated with Robust Immune Reconstitution and Lower Rates of Severe Infection Compared to Standard Umbilical Cord Blood Transplantation

Conference Blood · November 5, 2021 AbstractIntroductionOmidubicel is an advanced cell therapy for allogeneic hematopoietic stem cell transplantation (HSCT), derived from appropriately HLA-matched umbilical cor ... Full text Cite

Lymphoid and myeloid immune cell reconstitution after nicotinamide-expanded cord blood transplantation.

Journal Article Bone Marrow Transplant · November 2021 Omidubicel (nicotinamide-expanded cord blood) is a potential alternative source for allogeneic hematopoietic cell transplantation (HCT) when an HLA-identical donor is lacking. A phase I/II trial with standalone omidubicel HCT showed rapid and robust neutro ... Full text Link to item Cite

Omidubicel vs standard myeloablative umbilical cord blood transplantation: results of a phase 3 randomized study.

Journal Article Blood · October 21, 2021 Omidubicel is an ex vivo expanded hematopoietic progenitor cell and nonexpanded myeloid and lymphoid cell product derived from a single umbilical cord blood unit. We report results of a phase 3 trial to evaluate the efficacy of omidubicel compared with sta ... Full text Link to item Cite

Autologous and allogeneic hematopoietic cell transplantation for diffuse large B-cell lymphoma-type Richter syndrome.

Journal Article Blood Adv · September 28, 2021 Richter syndrome (RS) represents a transformation from chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) to aggressive lymphoma, most commonly diffuse large B-cell lymphoma (DLBCL), which is associated with a dismal prognosis. Patients with ... Full text Link to item Cite

Female Sex Is Associated with Improved Long-Term Survival Following Allogeneic Hematopoietic Stem Cell Transplantation.

Journal Article Transplant Cell Ther · September 2021 Life expectancy for long-term survivors of allogeneic hematopoietic stem cell transplantation (alloHSCT), defined as those living ≥5 years post-transplantation, is significantly lower compared with that of the age-matched general population despite a relat ... Full text Link to item Cite

Decreased Mortality in 1-Year Survivors of Umbilical Cord Blood Transplant vs. Matched Related or Matched Unrelated Donor Transplant in Patients with Hematologic Malignancies.

Journal Article Transplant Cell Ther · August 2021 Allogeneic hematopoietic stem cell transplantation (HCT) has the potential to cure hematologic malignancies but is associated with significant morbidity and mortality. Although deaths during the first year after transplantation are often attributable to tr ... Full text Open Access Link to item Cite

Guidelines for the Prevention and Management of Graft-versus-Host Disease after Cord Blood Transplantation.

Journal Article Transplant Cell Ther · July 2021 The incidence of graft-versus-host disease (GVHD) after cord blood (CB) transplantation (CBT) is lower than expected given the marked degree of human leukocyte antigen (HLA)-mismatch of CB grafts. While the exact mechanism that underlies this biology remai ... Full text Link to item Cite

Myeloablative versus Reduced-Intensity Conditioning for Hematopoietic Cell Transplantation in Acute Myelogenous Leukemia and Myelodysplastic Syndromes-Long-Term Follow-Up of the BMT CTN 0901 Clinical Trial.

Journal Article Transplant Cell Ther · June 2021 Several prospective randomized trials comparing conditioning intensity before allogeneic hematopoietic cell transplantation (HCT) have been performed, with conflicting results. Although reduced-intensity conditioning (RIC) leads to lower treatment-related ... Full text Link to item Cite

Chlorhexidine Gluconate Bathing Reduces the Incidence of Bloodstream Infections in Adults Undergoing Inpatient Hematopoietic Cell Transplantation.

Journal Article Transplant Cell Ther · March 2021 Bloodstream infections (BSIs) occur in 20% to 45% of inpatient autologous and allogeneic hematopoietic cell transplant (HCT) patients. Daily bathing with the antiseptic chlorhexidine gluconate (CHG) has been shown to reduce the incidence of BSIs in critica ... Full text Link to item Cite

Assessing the Feasibility of a Novel mHealth App in Hematopoietic Stem Cell Transplant Patients.

Journal Article Transplant Cell Ther · February 2021 Hematopoietic stem cell transplantation (HCT) is a curative treatment option for patients with hematologic conditions but presents many complications that must be managed as a complex, chronic condition. Mobile health applications (mHealth apps) may permit ... Full text Link to item Cite

Double unrelated umbilical cord blood vs HLA-haploidentical bone marrow transplantation: the BMT CTN 1101 trial.

Journal Article Blood · January 21, 2021 Results of 2 parallel phase 2 trials of transplantation of unrelated umbilical cord blood (UCB) or bone marrow (BM) from HLA-haploidentical relatives provided equipoise for direct comparison of these donor sources. Between June 2012 and June 2018, 368 pati ... Full text Link to item Cite

Phase I dose escalation study of naive T-cell depleted donor lymphocyte infusion following allogeneic stem cell transplantation.

Journal Article Bone Marrow Transplant · January 2021 Prophylactic donor lymphocyte infusions (DLI) are used to augment post-transplant immune recovery to reduce both infectious complications and disease recurrence. Preclinical studies implicate the naive T-cell subset as the primary driver of graft-versus-ho ... Full text Link to item Cite

Impact of Conditioning Intensity and Genomics on Relapse After Allogeneic Transplantation for Patients With Myelodysplastic Syndrome.

Journal Article JCO Precis Oncol · 2021 PURPOSE: Patients with myelodysplastic syndrome (MDS) are at risk of relapse after allogeneic hematopoietic cell transplantation. The utility of ultra-deep genomic testing to predict and the impact of conditioning intensity to prevent MDS relapse are unkno ... Full text Link to item Cite

A phase 2 trial of the somatostatin analog pasireotide to prevent GI toxicity and acute GVHD in allogeneic hematopoietic stem cell transplant.

Journal Article PLoS One · 2021 BACKGROUND: Allogeneic hematopoietic stem cell transplantation (HCT) is an often curative intent treatment, however it is associated with significant gastrointestinal (GI) toxicity and treatment related mortality. Graft-versus-host disease is a significant ... Full text Link to item Cite

Clinical and Neuroimaging Correlates of Post-Transplant Delirium.

Journal Article Biol Blood Marrow Transplant · December 2020 Delirium is common among adults undergoing hematopoietic stem cell transplantation (HCT), although the clinical and neuroimaging correlates of post-HCT delirium have not been adequately delineated. We therefore examined the frequency of delirium and neuroi ... Full text Link to item Cite

Morphologic Leukemia-Free State in Acute Myeloid Leukemia Is Sufficient for Successful Allogeneic Hematopoietic Stem Cell Transplant

Conference Blood · November 5, 2020 Allogeneic hematopoietic cell transplant (HCT) improves survival in patients with relapsed or high risk acute myeloid leukemia (AML). Complete remission (CR) is typically a pre-requisite for transplantation, though many do not achieve a formal CR. ... Full text 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

Treatment with Foscarnet after Allogeneic Hematopoietic Cell Transplant (Allo-HCT) Is Associated with Long-Term Loss of Renal Function.

Journal Article Biol Blood Marrow Transplant · September 2020 Despite a well-established risk of chronic kidney disease (CKD) after allogeneic hematopoietic cell transplant (allo-HCT), the benefits of using nephrotoxic anti-infective agents to treat serious peritransplant infections often outweigh this risk. While th ... Full text Link to item Cite

Double unrelated umbilical cord blood versus HLA-haploidentical bone marrow transplantation (BMT CTN 1101).

Journal Article Blood · August 31, 2020 Results of two parallel phase II trials of transplantation of unrelated umbilical cord blood or bone marrow from HLA-haploidentical relatives provided equipoise for direct comparison of these donor sources. Between June 2012 and June 2018, 368 patients age ... Full text Link to item Cite

Hematopoietic Cell Transplantation, Version 2.2020, NCCN Clinical Practice Guidelines in Oncology.

Journal Article J Natl Compr Canc Netw · May 1, 2020 Hematopoietic cell transplantation (HCT) involves the infusion of hematopoietic progenitor cells into patients with hematologic disorders with the goal of re-establishing normal hematopoietic and immune function. HCT is classified as autologous or allogene ... Full text Link to item Cite

Impact of Conditioning Intensity of Allogeneic Transplantation for Acute Myeloid Leukemia With Genomic Evidence of Residual Disease.

Journal Article J Clin Oncol · April 20, 2020 PURPOSE: Patients with acute myeloid leukemia (AML) in remission remain at risk for relapse even after allogeneic hematopoietic cell transplantation (alloHCT). AML measurable residual disease (MRD) status before alloHCT has been shown to be prognostic. Whe ... Full text Link to item Cite

Cytomegalovirus in Allogeneic Hematopoietic Transplantation: Impact on Costs and Clinical Outcomes Using a Preemptive Strategy.

Journal Article Biol Blood Marrow Transplant · March 2020 Cytomegalovirus (CMV) results in significant morbidity and mortality following hematopoietic cell transplantation (HCT). Establishing the cost and clinical impact is imperative to the selection of appropriate CMV preventative strategies. This is a retrospe ... Full text Link to item Cite

Phase II Trial of Pasireotide to Prevent GI Toxicity and Acute Gvhd in Allogeneic HSCT

Conference BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION · March 1, 2020 Link to item Cite

Pre-transplant hepatic steatosis (fatty liver) is associated with chronic graft-vs-host disease but not mortality.

Journal Article PLoS One · 2020 Allogeneic-HCT (allo-HCT), while potentially curative, can result in significant complications including graft versus host disease (GVHD). Prior studies suggest that metabolic syndrome may be one risk factor for GVHD. We hypothesized that hepatic steatosis ... Full text Link to item Cite

Small-molecule nicotinamide for ex vivo expansion of umbilical cord blood.

Journal Article Exp Hematol · December 2019 Umbilical cord blood transplant is an alternative graft source for patients lacking a human leukocyte antigen-matched donor; however, delayed engraftment times have historically resulted in transplant-related morbidity and mortality from complications such ... Full text Link to item Cite

Single-Cell RNA-Seq Identifies Potentially Pathogenic B Cell Populations That Uniquely Circulate in Patients with Chronic Gvhd

Conference Blood · November 13, 2019 While B cells are known to contribute to the pathogenesis of chronic graft-versus-host disease (cGVHD) in mice, it has been challenging to elucidate intrinsic mechanisms of tolerance loss in patients. To identify distinct and potentially targetable ... Full text Cite

Evaluation of the Oral SYK Inhibitor Fostamatinib in Patients after Allogeneic Transplantation for Chronic Graft-Versus-Host Disease

Conference Blood · November 13, 2019 Our group previously showed that B cells signal aberrantly through the B cell receptor (BCR) in allogeneic hematopoietic stem cell transplant (HCT) patients with active chronic graft-versus-host disease (cGVHD). Preclinical mouse studies have demon ... Full text Cite

Pre-Transplant Hepatic Steatosis (fatty liver) Predicts Chronic Graft-Vs-Host Disease but Does Not Affect Mortality

Conference Blood · November 13, 2019 Introduction: Allogeneic hematopoietic stem cell transplant (allo-HCT) remains the only curative treatment for many hematologic conditions. However, significant mortality risk and risk of severe graft-vs-host disease (GvHD) limit its usefulness. So ... Full text Cite

Decreased Mortality after the First Year of Allogeneic Hematopoietic Stem Cell Transplant in Recipients of Umbilical Cord Blood Vs. Matched Related or Matched Unrelated Donors

Conference Blood · November 13, 2019 Introduction: Allogeneic hematopoietic stem cell transplantation (allo-HCT) provides a potential cure for a multitude of diseases but can result in high rates of morbidity and mortality. These outcomes have been associated with donor choice, with u ... Full text Cite

Geriatric Assessment Identifies Impairments in Younger Candidates for Allogeneic Hematopoietic Stem Cell Transplantation

Conference Blood · November 13, 2019 Introduction: Geriatric assessment (GA) is a multidimensional evaluation of patient health and function that may detect impairments not identified as part of routine care, predict treatment-related morbidity and mortality, and inform treatment plan ... Full text Cite

Daily Chlorhexidine Gluconate Bathing Reduces the Rate of Bloodstream Infections in Adults Undergoing Inpatient Hematopoietic Stem Cell Transplantation

Conference Blood · November 13, 2019 Introduction: While hematopoietic stem cell transplantation (HSCT) has great therapeutic potential, intensive conditioning regimens and variability in time to stem cell engraftment result in a period of pancytopenia and immunosuppression in which p ... Full text Cite

Interrater Reliability of Clinical Grading Measures for Cutaneous Chronic Graft-vs-Host Disease.

Journal Article JAMA Dermatol · July 1, 2019 IMPORTANCE: Cutaneous chronic graft-vs-host disease (cGVHD) is common after allogeneic hematopoietic stem cell transplant and is often associated with poor patient outcomes. A reliable and practical method for assessing disease severity and response to the ... Full text Link to item Cite

Current and future perspectives on allogeneic transplantation using ex vivo expansion or manipulation of umbilical cord blood cells.

Journal Article Int J Hematol · July 2019 In patients with hematologic malignancies, the outcome of umbilical cord blood transplantation has improved and is now comparable to that of matched unrelated donor transplantation. However, the limitation of using umbilical cord blood has been a delay in ... Full text Link to item Cite

Plerixafor alone for the mobilization and transplantation of HLA-matched sibling donor hematopoietic stem cells.

Journal Article Blood Adv · March 26, 2019 Plerixafor, a direct antagonist of CXCR4/stromal-derived factor 1, can safely and rapidly mobilize allografts without the use of granulocyte colony-stimulating factor (G-CSF). We conducted a phase 2, multicenter, prospective study of plerixafor-mobilized H ... Full text 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

Treatment with Foscarnet after Allogeneic Hematopoietic-Cell Transplant (AlloHCT) Is Associated with Long-Term Loss of Renal Function

Conference Blood · November 29, 2018 AbstractIntroduction: Despite a well-established risk of chronic kidney disease (CKD) in 20-30% of patients undergoing AlloHCT, the benefits of treating serious infections, such as cytomegalovirus (CMV), wit ... Full text Cite

Rapid and Robust CD4+ and CD8+ T-, NK-, B- and Monocyte Cell Reconstitution after Nicotinamide-Expanded Cord Blood Transplantation

Conference Blood · November 29, 2018 AbstractIntroductionNicotinamide-expanded cord blood (NiCord) is a promising alternative source for allogeneic hematopoietic cell transplantation (HCT) when an HLA-matched do ... Full text Cite

Use of a Processed Hematopoietic Stem Cell Product (FCRx) in Unmatched Related and Unrelated Donor — Recipient Pairs Is Associated with High Levels of Donor Chimerism and Donor-Specific Tolerance to Kidney Allografts

Conference Blood · November 29, 2018 AbstractThe prospect of eliminating the long term costs and side effects of drug-based immunosuppression (IS) is a compelling reason to pursue the establishment of transplant tolerance. Tolerance in kidney t ... Full text Cite

Easy-to-Read Informed Consent Form for Hematopoietic Cell Transplantation Clinical Trials: Results from the Blood and Marrow Transplant Clinical Trials Network 1205 Study.

Journal Article Biol Blood Marrow Transplant · October 2018 Because of the complexity of hematopoietic cell transplant trial treatments, informed consent forms are often long and difficult to read. We evaluated a 2-column easy-to-read informed consent (ETRIC) form that incorporates elements of health literacy and r ... Full text Link to item Cite

Plerixafor Plus Granulocyte Colony-Stimulating Factor for Patients with Non-Hodgkin Lymphoma and Multiple Myeloma: Long-Term Follow-Up Report.

Journal Article Biol Blood Marrow Transplant · June 2018 The purpose of this report is to analyze long-term clinical outcomes of patients exposed to plerixafor plus granulocyte colony-stimulating factor (G-CSF) for stem cell mobilization. This was a study of patients with non-Hodgkin lymphoma (NHL; n = 167) and ... Full text Link to item Cite

Shared Decision-Making in Hematopoietic Stem Cell Transplantation for Sickle Cell Disease.

Journal Article Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation · May 2018 Full text Cite

Stem Cell Transplantation and Informatics: Current Considerations.

Journal Article Biol Blood Marrow Transplant · April 2018 Informatics strategies and applications available to stem cell transplant (SCT) programs are diverse and changing rapidly. Although most hospitals have electronic medical records (EMRs), few are equipped with specialized SCT applications. Most EMRs do not ... Full text Link to item Cite

High-dose BCNU/Melphalan conditioning regimen before autologous stem cell transplantation in newly diagnosed multiple myeloma.

Journal Article Bone Marrow Transplant · January 2018 Single-agent high-dose melphalan (HDM, 200 mg/m2) has been the most commonly used conditioning regimen prior to autologous stem cell transplant, since its introduction in 1992. We used a more aggressive alkylator-based conditioning regimen in an attempt to ... Full text Link to item Cite

HSCT-GAVE as a Manifestation of Chronic Graft versus Host Disease: A Case Report and Review of the Existing Literature.

Journal Article Case Rep Transplant · 2018 Gastric antral vascular ectasia or "watermelon stomach" is a significant cause of nonvariceal upper GI bleeding and is characterized by red, tortuous ectatic vessels along longitudinal folds in the gastric antrum. The existing literature links GAVE to pati ... Full text Open Access Link to item Cite

Prospective, Randomized, Double-Blind, Phase III Clinical Trial of Anti-T-Lymphocyte Globulin to Assess Impact on Chronic Graft-Versus-Host Disease-Free Survival in Patients Undergoing HLA-Matched Unrelated Myeloablative Hematopoietic Cell Transplantation.

Journal Article J Clin Oncol · December 20, 2017 Purpose Several open-label randomized studies have suggested that in vivo T-cell depletion with anti-T-lymphocyte globulin (ATLG; formerly antithymocyte globulin-Fresenius) reduces chronic graft-versus-host disease (cGVHD) without compromising survival. We ... Full text Link to item Cite

Efficacy and safety of high-dose chemotherapy with autologous stem cell transplantation in senior versus younger adults with newly diagnosed multiple myeloma.

Journal Article Hematol Oncol · December 2017 We retrospectively studied 340 fit patients with multiple myeloma (MM) who underwent autologous stem cell transplantation (ASCT). We hypothesized that progression-free survival (PFS) of older patients was non-inferior to that of younger patients after ASCT ... Full text Link to item Cite

An aberrant NOTCH2-BCR signaling axis in B cells from patients with chronic GVHD.

Journal Article Blood · November 9, 2017 B-cell receptor (BCR)-activated B cells contribute to pathogenesis in chronic graft-versus-host disease (cGVHD), a condition manifested by both B-cell autoreactivity and immune deficiency. We hypothesized that constitutive BCR activation precluded function ... Full text Link to item Cite

Adult Umbilical Cord Blood Transplantation Using Myeloablative Thiotepa, Total Body Irradiation, and Fludarabine Conditioning.

Conference Biol Blood Marrow Transplant · November 2017 Treatment-related mortality (TRM) remains elevated in adult patients undergoing umbilical cord blood transplantation (UCBT), including an early rise in TRM suggestive of excessive toxicity associated with the standard myeloablative total body irradiation ( ... Full text Link to item Cite

Evaluation of Hematopoietic Stem Cell Mobilization Rates with Early Plerixafor Administration for Adult Stem Cell Transplantation.

Journal Article Biol Blood Marrow Transplant · August 2017 The addition of plerixafor to high-dose colony-stimulating growth factor has been shown to improve stem cell mobilization rates in autologous transplant patients with multiple myeloma and non-Hodgkin lymphoma. This study evaluates the change in administrat ... Full text Link to item Cite

Transplantation of Ex Vivo Expanded Umbilical Cord Blood (NiCord) Decreases Early Infection and Hospitalization.

Journal Article Biol Blood Marrow Transplant · July 2017 Delayed hematopoietic recovery contributes to increased infection risk following umbilical cord blood (UCB) transplantation. In a Phase 1 study, adult recipients of UCB stem cells cultured ex vivo for 3 weeks with nicotinamide (NiCord) had earlier median n ... Full text 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

Myeloablative Versus Reduced-Intensity Hematopoietic Cell Transplantation for Acute Myeloid Leukemia and Myelodysplastic Syndromes.

Journal Article J Clin Oncol · April 10, 2017 Purpose The optimal regimen intensity before allogeneic hematopoietic cell transplantation (HCT) is unknown. We hypothesized that lower treatment-related mortality (TRM) with reduced-intensity conditioning (RIC) would result in improved overall survival (O ... Full text Link to item Cite

Outcomes of Maintenance Therapy with Bortezomib after Autologous Stem Cell Transplantation for Patients with Multiple Myeloma.

Journal Article Biol Blood Marrow Transplant · February 2017 Comprehensive recommendations for maintenance therapy after autologous stem cell transplantation (ASCT) for patients with multiple myeloma (MM) have yet to be defined. Bortezomib has been utilized as maintenance therapy after ASCT, but data attesting to th ... Full text Link to item Cite

Reply to Strahilevitz and Shapira.

Journal Article Clin Infect Dis · December 12, 2016 Full text Link to item Cite

Adult Umbilical Cord Blood Transplantation Using Myeloablative Total Body Irradiation, Fludarabine, and Thiotepa Conditioning

Conference Blood · December 2, 2016 AbstractIntroduction: Umbilical cord blood (UCB) extends the curative potential of stem cell transplantation to adult patients without a suitable donor. The most commonly used myeloablative preparative regim ... Full text Cite

A Prospective Randomized Double Blind Phase 3 Clinical Trial of Anti- T Lymphocyte Globulin (ATLG) to Assess Impact on Chronic Graft-Versus-Host Disease (cGVHD) Free Survival in Patients Undergoing HLA Matched Unrelated Myeloablative Hematopoietic Cell Transplantation (HCT)

Conference Blood · December 2, 2016 AbstractBackground: Chronic GVHD contributes to morbidity and mortality after allogeneic transplantation. Several phase 2 and 3 open label studies suggested that ATLG (formerly refe ... 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

Universal Mask Usage for Reduction of Respiratory Viral Infections After Stem Cell Transplant: A Prospective Trial.

Journal Article Clin Infect Dis · October 15, 2016 BACKGROUND: Respiratory viral infections (RVIs) are frequent complications of hematopoietic stem cell transplant (HSCT). Surgical masks are a simple and inexpensive intervention that may reduce nosocomial spread. METHODS: In this prospective single-center ... Full text Link to item Cite

Plerixafor (a CXCR4 antagonist) following myeloablative allogeneic hematopoietic stem cell transplantation enhances hematopoietic recovery.

Journal Article J Hematol Oncol · August 17, 2016 BACKGROUND: The binding of CXCR4 with its ligand (stromal-derived factor-1) maintains hematopoietic stem/progenitor cells (HSPCs) in a quiescent state. We hypothesized that blocking CXCR4/SDF-1 interaction after hematopoietic stem cell transplantation (HSC ... Full text Open Access Link to item Cite

Efficacy of Pharmacokinetics-Directed Busulfan, Cyclophosphamide, and Etoposide Conditioning and Autologous Stem Cell Transplantation for Lymphoma: Comparison of a Multicenter Phase II Study and CIBMTR Outcomes.

Journal Article Biol Blood Marrow Transplant · July 2016 Busulfan, cyclophosphamide, and etoposide (BuCyE) is a commonly used conditioning regimen for autologous stem cell transplantation (ASCT). This multicenter, phase II study examined the safety and efficacy of BuCyE with individually adjusted busulfan based ... Full text Open Access Link to item Cite

Efficacy of Autologous Stem Cell Transplantation in Older Multiple Myeloma Patients

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

Ex Vivo Expansion or Manipulation of Stem Cells to Improve Outcome of Umbilical Cord Blood Transplantation.

Journal Article Curr Hematol Malig Rep · February 2016 The outcome of umbilical cord blood transplantation for adult patients with hematologic malignancies now rivals that of matched unrelated donor transplantation. However, delayed hematopoietic and immunologic recovery remains a source of significant morbidi ... Full text Link to item Cite

Myeloablative conditioning with total body irradiation for AML: Balancing survival and pulmonary toxicity.

Conference Adv Radiat Oncol · 2016 PURPOSE: The purpose of this study was to compare leukemia-free survival (LFS) and other clinical outcomes in patients with acute myelogenous leukemia who underwent a myeloablative allogeneic stem cell transplant with and without total body irradiation (TB ... Full text Open Access Link to item Cite

Results of a Phase III Randomized, Multi-Center Study of Allogeneic Stem Cell Transplantation after High Versus Reduced Intensity Conditioning in Patients with Myelodysplastic Syndrome (MDS) or Acute Myeloid Leukemia (AML): Blood and Marrow Transplant Clinical Trials Network (BMT CTN) 0901

Conference Blood · December 3, 2015 AbstractAlthough reduced intensity conditioning (RIC) has a lower toxicity profile and lower treatment related mortality (TRM), retrospective analyses have shown higher relapse rates and similar overall surv ... Full text Cite

Cyclophosphamide conditioning in patients with severe aplastic anaemia given unrelated marrow transplantation: a phase 1-2 dose de-escalation study.

Journal Article Lancet Haematol · September 2015 BACKGROUND: The optimum preparative regimen for unrelated donor marrow transplantation in patients with severe aplastic anaemia remains to be established. We investigated whether the combination of fludarabine, anti-thymocyte globulin, and total body irrad ... Full text Link to item Cite

Cyclophosphamide-based hematopoietic stem cell mobilization before autologous stem cell transplantation in newly diagnosed multiple myeloma.

Journal Article J Clin Apher · June 2015 High-dose cyclophosphamide (Cy) is frequently employed for peripheral blood mobilization of hematopoietic stem cells before high-dose chemotherapy with autologous stem cell transplantation (ASCT) in multiple myeloma (MM). The benefit of mobilization with C ... Full text Link to item Cite

Improving the outcome of umbilical cord blood transplantation through ex vivo expansion or graft manipulation.

Journal Article Cytotherapy · June 2015 The outcome of umbilical cord blood transplantation for adult patients with hematologic malignancies now rivals that of matched unrelated donor transplantation. However, relatively low lymphocyte and hematopoietic stem and progenitor cell dose is a source ... Full text Link to item Cite

Results of a prospective multicentre myeloablative double-unit cord blood transplantation trial in adult patients with acute leukaemia and myelodysplasia.

Journal Article Br J Haematol · February 2015 Double-unit cord blood (CB) grafts may improve engraftment and relapse risk in adults with haematological malignancies. We performed a prospective high-dose myeloablative double-unit CB transplantation (CBT) trial in adults with high-risk acute leukaemia o ... Full text Link to item Cite

Cardiopulmonary exercise testing prior to myeloablative allo-SCT: a feasibility study.

Journal Article Bone Marrow Transplant · October 2014 The feasibility of symptom-limited cardiopulmonary exercise testing (CPET) prior to allo-SCT was assessed in addition to the prognostic value of CPET-derived measures. CPET was performed prospectively on 21 patients with hematologic malignancies, with asse ... Full text 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

Increased BCR responsiveness in B cells from patients with chronic GVHD.

Journal Article Blood · March 27, 2014 Although B cells have emerged as important contributors to chronic graft-versus-host-disease (cGVHD) pathogenesis, the mechanisms responsible for their sustained activation remain unknown. We previously showed that patients with cGVHD have significantly in ... Full text Link to item Cite

Reduced-intensity allogeneic transplantation using alemtuzumab from HLA-matched related, unrelated, or haploidentical related donors for patients with hematologic malignancies.

Journal Article Biol Blood Marrow Transplant · February 2014 We present a comparative study on 124 patients with hematologic malignancies who had undergone reduced-intensity conditioning and then received a transplant from an HLA-matched related (MRD), an HLA-matched unrelated (MUD), or an HLA-haploidentical related ... Full text Link to item Cite

Outcomes and costs of autologous stem cell mobilization with chemotherapy plus G-CSF vs G-CSF alone

Journal Article Bone Marrow Transplantation · November 1, 2013 Chemotherapy plus G-CSF (C+G) and G-CSF alone are two of the most common methods used to mobilize CD34 + cells for autologous hematopoietic SCT (AHSCT). In order to compare and determine the real-world outcomes and costs of these strategies, we performed a ... Full text Cite

Outcomes and costs of autologous stem cell mobilization with chemotherapy plus G-CSF vs G-CSF alone.

Journal Article Bone Marrow Transplant · November 2013 Chemotherapy plus G-CSF (C+G) and G-CSF alone are two of the most common methods used to mobilize CD34(+) cells for autologous hematopoietic SCT (AHSCT). In order to compare and determine the real-world outcomes and costs of these strategies, we performed ... 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

A Phase I study of arsenic trioxide (Trisenox), ascorbic acid, and bortezomib (Velcade) combination therapy in patients with relapsed/refractory multiple myeloma.

Journal Article Cancer Invest · March 2013 PURPOSE: This Phase I study assessed the feasibility of concomitant arsenic trioxide (ATO), ascorbic acid (AA), and bortezomib (Velcade™) (AAV) for patients with relapsed/refractory multiple myeloma. EXPERIMENTAL DESIGN: ATO (0.25 mg/kg) and AA (1 g) were ... Full text Link to item Cite

Surgical Mask Usage Reduces the Incidence of Parainfluenza Virus 3 in Recipients of Stem Cell Transplantation

Conference Blood · November 16, 2012 AbstractAbstract 462Respiratory viral infections (RVI) are frequent complications of hematopoietic stem cell transplantation (HSCT). Parainfluenza virus 3 (PIV3) in particula ... Full text Cite

Immune recovery in adult patients after myeloablative dual umbilical cord blood, matched sibling, and matched unrelated donor hematopoietic cell transplantation.

Journal Article Biol Blood Marrow Transplant · November 2012 Immunologic reconstitution after allogeneic hematopoietic cell transplantation is a critical component of successful outcome. Umbilical cord blood (UCB) transplantation in adult recipients is associated with slow and often inadequate immune recovery. We ch ... Full text Link to item Cite

Efficacy and safety of hematopoietic stem cell remobilization with plerixafor+G-CSF in adult patients with germ cell tumors.

Journal Article Bone Marrow Transplant · October 2012 Autologous hematopoietic SCT (auto-HSCT) can be curative for patients with germ cell tumors. Poor stem cell mobilization jeopardizes the ability to deliver this therapy. Herein, we describe a retrospective study examining safety and efficacy of plerixafor ... Full text 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

Preemptive dosing of plerixafor given to poor stem cell mobilizers on day 5 of G-CSF administration.

Journal Article Bone Marrow Transplant · August 2012 Plerixafor, given on day 4 of G-CSF treatment is more effective than G-CSF alone in mobilizing hematopoietic progenitor cells. We tested a strategy of preemptive plerixafor use following assessment of the peak mobilization response to 5 days of G-CSF. Pati ... Full text Link to item Cite

Fludarabine-based conditioning for marrow transplantation from unrelated donors in severe aplastic anemia: early results of a cyclophosphamide dose deescalation study show life-threatening adverse events at predefined cyclophosphamide dose levels.

Journal Article Biol Blood Marrow Transplant · July 2012 Excessive adverse events were encountered in a Phase I/II study of cyclophosphamide (CY) dose deescalation in a fludarabine-based conditioning regimen for bone marrow transplantation from unrelated donors in patients with severe aplastic anemia. All patien ... Full text 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

Outcomes of a 1-day nonmyeloablative salvage regimen for patients with primary graft failure after allogeneic hematopoietic cell transplantation.

Journal Article Bone Marrow Transplant · May 2012 Primary graft failure after allogeneic hematopoietic cell transplantation is a life-threatening complication. A shortened conditioning regimen may reduce the risk of infection and increase the chance of survival. Here, we report the outcome of 11 patients ... Full text Link to item Cite

Severe pulmonary toxicity after myeloablative conditioning using total body irradiation: an assessment of risk factors.

Journal Article Int J Radiat Oncol Biol Phys · November 1, 2011 PURPOSE: To assess factors associated with severe pulmonary toxicity after myeloablative conditioning using total body irradiation (TBI) followed by allogeneic stem cell transplantation. METHODS AND MATERIALS: A total of 101 adult patients who underwent TB ... Full text Link to item Cite

Early post transplant (F-18) 2-fluoro-2-deoxyglucose positron emission tomography does not predict outcome for patients undergoing auto-SCT in non-Hodgkin and Hodgkin lymphoma.

Journal Article Bone Marrow Transplant · June 2011 Positron emission tomography (PET) in conjunction with computed tomography is a frequently used modality for staging patients with lymphoma. Utility of PET-computed tomography before or early following auto-SCT has not been as rigorously evaluated. We retr ... Full text Link to item Cite

Adult dual umbilical cord blood transplantation using myeloablative total body irradiation (1350 cGy) and fludarabine conditioning.

Journal Article Biol Blood Marrow Transplant · June 2011 High treatment-related mortality (TRM) and high graft failure rate are serious concerns in HLA-mismatched umbilical cord blood (UCB) transplantation with myeloablative conditioning. We conducted a prospective trial of dual UCB transplantation using modifie ... Full text Link to item Cite

Donor cell-derived leukemias/myelodysplastic neoplasms in allogeneic hematopoietic stem cell transplant recipients: a clinicopathologic study of 10 cases and a comprehensive review of the literature.

Journal Article Am J Clin Pathol · April 2011 We report 10 cases of donor cell leukemia (DCL). All cases except the case of chronic lymphocytic leukemia had anemia, neutropenia, and/or thrombocytopenia when DCL was diagnosed. Eight cases with sex-mismatched hematopoietic stem cell transplant (HCT) sho ... Full text Link to item Cite

Renal shielding and dosimetry for patients with severe systemic sclerosis receiving immunoablation with total body irradiation in the scleroderma: cyclophosphamide or transplantation trial.

Journal Article Int J Radiat Oncol Biol Phys · March 15, 2011 PURPOSE: To describe renal shielding techniques and dosimetry in delivering total body irradiation (TBI) to patients with severe systemic sclerosis (SSc) enrolled in a hematopoietic stem cell transplant protocol. METHODS AND MATERIALS: The Scleroderma: Cyc ... Full text Link to item Cite

Reduced intensity versus myeloablative allogeneic stem cell transplantation for the treatment of acute myeloid leukemia, myelodysplastic syndrome and acute lymphoid leukemia.

Journal Article Curr Opin Oncol · March 2011 PURPOSE OF REVIEW: Use of a reduced intensity conditioning (RIC) regimen has now become standard practice among older or more infirmed stem cell transplantation candidates. Encouraging outcome in this population has led to the question of whether RIC shoul ... Full text Link to item Cite

Clinical outcomes following hematopoietic stem cell transplantation for the treatment of mucopolysaccharidosis VI.

Journal Article Mol Genet Metab · February 2011 Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome) is one of approximately 50 known lysosomal storage disorders. MPS VI is characterized by an absence or deficiency of N-acetylgalactosamine 4-sulfatase (arylsulfatase B) resulting in accumulation of ... Full text Link to item Cite

Feasibility of low-dose interleukin-2 therapy following T-cell-depleted nonmyeloablative allogeneic hematopoietic stem cell transplantation from HLA-matched or -mismatched family member donors.

Journal Article Cancer Invest · January 2011 INTRODUCTION: High relapse rates and infections remain primary causes of failure in nonmyeloablative transplantation. Interleukin-2 (IL-2) may stimulate the immune system and improve outcomes. The primary objective of this pilot study was to evaluate the f ... Full text Link to item Cite

Umbilical cord blood transplantation for treatment of non-malignant disorders

Journal Article Cellular Therapy and Transplantation · December 1, 2010 As the outcomes of umbilical cord blood transplantation improve, the risk versus benefit considerations with respect to treatment of non-malignant disorders must be reassessed. Recent data would suggest that the outcome of umbilical cord blood transplantat ... Full text Cite

Natural killer cell-enriched donor lymphocyte infusions from A 3-6/6 HLA matched family member following nonmyeloablative allogeneic stem cell transplantation.

Journal Article Biol Blood Marrow Transplant · August 2010 Infusing natural killer (NK) cells following transplantation may allow less infections and relapse with little risk of acute graft-versus-host disease (aGVHD). We delivered 51 total NK cell-enriched donor lymphocyte infusions (DLIs) to 30 patients followin ... Full text Link to item Cite

Overcoming drug resistance in mantle cell lymphoma using a combination of dose-dense and intense therapy.

Journal Article Cancer Invest · July 2010 We present a study of the prevalence of genetic polymorphisms and expression of genes encoding the drug-resistance proteins glutathione S-transferases (GSTs) in order to gain insights into the pattern of failure evident in mantle cell lymphoma. We note a h ... Full text Link to item Cite

Non-myeloablative umbilical cord blood transplantation.

Journal Article Best Pract Res Clin Haematol · June 2010 Allogeneic umbilical cord blood (UCB) transplantation is an established alternative to unrelated bone marrow or peripheral blood transplantation for treatment of high-risk hematologic disorders. There is growing evidence that non-myeloablative bone marrow ... Full text Link to item Cite

"Short course" bortezomib plus melphalan and prednisone as induction prior to transplant or as frontline therapy for nontransplant candidates in patients with previously untreated multiple myeloma.

Journal Article Biol Blood Marrow Transplant · January 2010 The purpose of this study was to evaluate the efficacy and safety of short-course bortezomib, melphalan, prednisone (VMP) in previously untreated multiple myeloma as frontline therapy for transplant-ineligible patients and induction prior to autologous ste ... Full text Link to item Cite

Plerixafor and G-CSF versus placebo and G-CSF to mobilize hematopoietic stem cells for autologous stem cell transplantation in patients with multiple myeloma.

Journal Article Blood · June 4, 2009 This phase 3, multicenter, randomized (1:1), double-blind, placebo-controlled study evaluated the safety and efficacy of plerixafor with granulocyte colony-stimulating factor (G-CSF) in mobilizing hematopoietic stem cells in patients with multiple myeloma. ... Full text Link to item Cite

Allogeneic hematopoietic stem-cell transplantation for leukocyte adhesion deficiency.

Journal Article Pediatrics · March 2009 OBJECTIVES: Leukocyte adhesion deficiency is a rare primary immune disorder caused by defects of the CD18 beta-integrin molecule on immune cells. The condition usually presents in early infancy and is characterized by deep tissue infections, leukocytosis w ... Full text Link to item Cite

Response and toxicity of donor lymphocyte infusions following T-cell depleted non-myeloablative allogeneic hematopoietic SCT from 3-6/6 HLA matched donors.

Journal Article Bone Marrow Transplant · February 2009 We report the outcome of early donor lymphocyte infusions (DLIs) after T-cell depleted non-myeloablative transplantation using stem cells from HLA-matched or mismatched donors. Sixty-nine patients with high-risk hematologic malignancies received DLI follow ... Full text Link to item Cite

Myeloablative intravenous busulfan/fludarabine conditioning does not facilitate reliable engraftment of dual umbilical cord blood grafts in adult recipients.

Journal Article Biol Blood Marrow Transplant · May 2008 The efficacy of once-daily intravenous busulfan with fludarabine as a preparative regimen for partially matched umbilical cord blood transplantation has not been formally studied. We randomized 10 adult patients with myeloid malignancies to receive either ... Full text Link to item Cite

Outcomes of a second non-myeloablative allogeneic stem cell transplantation following graft rejection.

Journal Article Bone Marrow Transplant · January 2008 Following initial graft rejection, a second attempt at allogeneic immunotherapy is often contemplated, but data on the success is limited. We therefore report on 11 patients with hematologic malignancies, renal cell cancer or marrow failure who underwent a ... Full text Link to item Cite

Reversion mutations in patients with leukocyte adhesion deficiency type-1 (LAD-1).

Journal Article Blood · January 1, 2008 Leukocyte adhesion deficiency type-1 (LAD-1) is an autosomal recessive immunodeficiency caused by mutations in the beta2 integrin, CD18, that impair CD11/CD18 heterodimer surface expression and/or function. Absence of functional CD11/CD18 integrins on leuk ... Full text Link to item Cite

Fludarabine-based nonmyeloablative stem cell transplantation for sickle cell disease with and without renal failure: clinical outcome and pharmacokinetics.

Journal Article Biol Blood Marrow Transplant · December 2007 End-organ damage is common in patients with sickle cell disease (SCD) thereby limiting the use of allogeneic stem cell transplantation (SCT). We report the outcome of 2 adult SCD patients, 1 with end-stage renal disease (ESRD), who underwent fludarabine-ba ... Full text Link to item Cite

Sources of human and murine hematopoietic stem cells.

Journal Article Curr Protoc Immunol · November 2007 This unit describes various sites from which human and murine hematopoietic stem cells (HSC) can be collected, including bone marrow, peripheral blood, and, in the case of human HSC, umbilical cord blood. Guidelines related to each of the cell harvesting p ... 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

Partially matched, nonmyeloablative allogeneic transplantation: clinical outcomes and immune reconstitution.

Journal Article J Clin Oncol · February 20, 2007 PURPOSE: Allogeneic transplantation is typically limited to younger patients having a matched donor. To allow a donor to be found for nearly all patients, we have used a nonmyeloablative conditioning regimen in conjunction with stem cells from a related do ... Full text Link to item Cite

Polyclonal long-term MFGS-gp91phox marking in rhesus macaques after nonmyeloablative transplantation with transduced autologous peripheral blood progenitor cells.

Journal Article Mol Ther · August 2006 We have recently reported that the RD114-pseudotyped MFGS-gp91phox vector achieves unprecedented levels of correction of the NADPH-oxidase gp91phox (approved gene symbol CYBB) defect in CD34(+) cells from patients with X-linked chronic granulomatous diseas ... Full text Link to item Cite

Patients with chronic granulomatous disease have a reduced peripheral blood memory B cell compartment.

Journal Article J Immunol · June 1, 2006 In this study, we have identified an altered B cell compartment in patients with chronic granulomatous disease (CGD), a disorder of phagocyte function, characterized by pyogenic infections and granuloma formation caused by defects in NADPH activity. This i ... Full text Link to item Cite

Unique abnormalities of CD4(+) and CD8(+) central memory cells associated with chronic graft-versus-host disease improve after extracorporeal photopheresis.

Journal Article Biol Blood Marrow Transplant · January 2006 Chronic graft-versus-host disease (cGVHD) remains a problematic complication of allogeneic hematopoietic stem cell transplantation. Laboratory parameters correlated with cGVHD have not been fully defined, although changes in CD4/CD8 ratios occur and a decr ... Full text Link to item Cite

Chronic graft-versus-host disease.

Journal Article Blood Rev · January 2006 Chronic graft versus host disease (GVHD) remains today one of the most vexing late complications of allogeneic stem cell transplantation. Occurring a minimum of 100 days following stem cell transplantation, approximately 50% of patients will experience som ... Full text Link to item Cite

Hematopoietic stem cell transplantation for complete IFN-gamma receptor 1 deficiency: a multi-institutional survey.

Journal Article J Pediatr · December 2004 Featured Publication OBJECTIVES: To evaluate the outcome of hematopoietic stem cell transplantation (HSCT) in a series of patients with inherited complete IFN-gamma receptor 1 (IFNgammaR1) deficiency. STUDY DESIGN: We report 8 patients who received altogether 11 HSCT from fami ... Full text Link to item Cite

Campath-1H May Have Activity in the Treatment of Multiple Myeloma.

Conference Blood · November 16, 2004 AbstractPreviously we have demonstrated that multiple myeloma cell lines and primary cells express CD52, the antigenic target of Campath-1H, and that they undergo apoptosis following treatment with alemtuzum ... Full text Cite

Hematopoietic stem cell transplantation.

Journal Article Curr Opin Hematol · November 2004 Full text Link to item Cite

Adult recipients of umbilical cord blood transplants after nonmyeloablative preparative regimens.

Journal Article Biol Blood Marrow Transplant · August 2004 We report the outcome of 13 patients with advanced malignancies who underwent nonmyeloablative conditioning therapy followed by infusion of partially matched unrelated cord blood cells. The median age of these patients was 49 years, and their median weight ... Full text Link to item Cite

Severe chronic graft-versus-host disease is characterized by a preponderance of CD4(+) effector memory cells relative to central memory cells.

Journal Article Blood · May 15, 2004 Featured Publication Donor alloreactive CD4(+) T cells are important to the pathogenesis of chronic graft-versus-host disease (cGVHD), but specific subsets of CD4(+) T cells responsible for GVHD have not been defined. We hypothesized that cGVHD might be associated with a prepo ... Full text Link to item Cite

Pediatric large-volume leukapheresis: a single institution experience with heparin versus citrate-based anticoagulant regimens.

Journal Article Transfusion · February 2004 BACKGROUND: Anticoagulant-associated toxicity may exert significant effects on the safety and efficacy of large-volume leukapheresis (LVL) in children, however, few studies specifically address management of this issue. STUDY DESIGN AND METHODS: Seventy-fo ... Full text Link to item Cite

Concentrated RD114-pseudotyped MFGS-gp91phox vector achieves high levels of functional correction of the chronic granulomatous disease oxidase defect in NOD/SCID/beta -microglobulin-/- repopulating mobilized human peripheral blood CD34+ cells.

Journal Article Blood · October 15, 2003 In previous studies amphotropic MFGS-gp91phox (murine onco-retrovirus vector) was used in a clinical trial of X-linked chronic granulomatous disease (X-CGD) gene therapy to achieve transient correction of oxidase activity in 0.1% of neutrophils. We later s ... Full text Link to item Cite

Persistent Mycobacterium avium infection following nonmyeloablative allogeneic peripheral blood stem cell transplantation for interferon-gamma receptor-1 deficiency.

Journal Article Blood · October 1, 2003 Featured Publication Interferon-gamma receptor-1 (IFNgammaR1) deficiency is a rare inherited immunodeficiency. We performed a nonmyeloablative allogeneic stem cell transplantation on a boy with complete IFNgammaR1 deficiency and refractory disseminated Myco- bacterium avium in ... Full text Link to item Cite

Immunomodulation in stem-cell transplantation.

Journal Article Curr Opin Pharmacol · August 2002 Acute graft-versus-host disease is a complication that affects 30-60% of patients undergoing allogeneic stem-cell transplantation. The standard for prophylaxis for graft-versus-host disease has historically been the combination of cyclosporine and methotre ... Full text Link to item Cite

Autologous stem cell transplantation for pediatric rheumatic diseases.

Journal Article J Rheumatol · October 2001 The National Institute of Allergy and Infectious Disease, National Institutes of Health, convened a workshop entitled The Next Step: Protocol Development for Autologous Stem Cell Transplantation for Pediatric Rheumatic Disease, June 2000, co-chaired by Drs ... Link to item Cite

Lymphocyte reconstitution following non-myeloablative hematopoietic stem cell transplantation follows two patterns depending on age and donor/recipient chimerism.

Journal Article Bone Marrow Transplant · September 2001 Featured Publication The effect of mixed chimerism on the pace of post-transplant immune reconstitution is unknown. Using flow cytometry, recall and neo-antigen vaccine responses, and T cell receptor recombination excision circle (TREC) quantification, we evaluated phenotypic ... Full text Link to item Cite

Treatment of chronic granulomatous disease with nonmyeloablative conditioning and a T-cell-depleted hematopoietic allograft.

Journal Article N Engl J Med · March 22, 2001 Featured Publication BACKGROUND: The treatment of chronic granulomatous disease with conventional allogeneic hematopoietic stem-cell transplantation carries a high risk of serious complications and death. We investigated the feasibility of stem-cell transplantation without abl ... Full text Link to item Cite

Stem-cell transplantation for inherited immunodeficiency disorders.

Journal Article Pediatr Clin North Am · December 2000 Featured Publication For patients with well-characterized, rapidly fatal, nonmalignant immunodeficiency disorders, such as SCID, the decision to proceed with allogeneic SCT is clear-cut. For patients with many other disorders, this decision can be extremely difficult. Disorder ... Full text Link to item Cite

The gene for familial Mediterranean fever, MEFV, is expressed in early leukocyte development and is regulated in response to inflammatory mediators.

Journal Article Blood · May 15, 2000 Familial Mediterranean fever (FMF) is a recessive disorder characterized by episodes of fever and neutrophil-mediated serosal inflammation. We recently identified the gene causing FMF, designated MEFV, and found it to be expressed in mature neutrophils, su ... Link to item Cite

Granulocyte colony-stimulating factor mobilized peripheral blood stem cells enter into G1 of the cell cycle and express higher levels of amphotropic retrovirus receptor mRNA.

Journal Article Exp Hematol · July 1999 Featured Publication We compared the cell cycle status and expression of mRNA for the amphotropic retroviral receptor in hematopoietic stem cells isolated from bone marrow and cytokine mobilized peripheral blood. CD34+ cells from six normal volunteers were enriched by immune s ... Full text Link to item Cite