Browsing by Subject "Hematopoietic Stem Cell Transplantation"
Now showing items 1-20 of 45
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A Randomized Phase II Crossover Study of Imatinib or Rituximab for Cutaneous Sclerosis after Hematopoietic Cell Transplantation.
(Clin Cancer Res, 2016-01-15)PURPOSE: Cutaneous sclerosis occurs in 20% of patients with chronic graft-versus-host disease (GVHD) and can compromise mobility and quality of life. EXPERIMENTAL DESIGN: We conducted a prospective, multicenter, randomized, ... -
A Randomized, Placebo-controlled Trial of Fidaxomicin for Prophylaxis of Clostridium difficile-associated Diarrhea in Adults Undergoing Hematopoietic Stem Cell Transplantation.
(Clinical infectious diseases : an official publication of the Infectious Diseases Society of America, 2019-01)<h4>Background</h4>Clostridium difficile-associated diarrhea (CDAD) is common during hematopoietic stem-cell transplantation (HSCT) and is associated with increased morbidity and mortality. We evaluated fidaxomicin for prevention ... -
Allogeneic hematopoietic SCT for alpha-mannosidosis: an analysis of 17 patients.
(Bone marrow transplantation, 2012-03)Alpha-mannosidosis is a rare lysosomal storage disease. Hematopoietic SCT (HSCT) is usually recommended as a therapeutic option though reports are anecdotal to date. This retrospective multi institutional analysis describes ... -
Allogeneic stem cell transplantation with omidubicel in sickle cell disease.
(Blood advances, 2021-02)Many patients with sickle cell disease (SCD) do not have HLA-matched related donors for hematopoietic stem cell transplantation (HSCT). Unrelated cord blood (UCB) is an alternative graft option but is historically associated ... -
Brincidofovir for Asymptomatic Adenovirus Viremia in Pediatric and Adult Allogeneic Hematopoietic Cell Transplant Recipients: A Randomized Placebo-Controlled Phase II Trial.
(Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2017-03)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 ... -
Cell therapy for diverse central nervous system disorders: inherited metabolic diseases and autism.
(Pediatric research, 2018-01)The concept of utilizing human cells for the treatment of medical conditions is not new. In its simplest form, blood product transfusion as treatment of severe hemorrhage has been practiced since the 1800s. The advent of ... -
Clinical outcomes of children with abnormal newborn screening results for Krabbe disease in New York State.
(Genetics in medicine : official journal of the American College of Medical Genetics, 2016-12)<h4>Background</h4>Early infantile Krabbe disease is rapidly fatal, but hematopoietic stem cell transplantation (HSCT) may improve outcomes if performed soon after birth. New York State began screening all newborns for Krabbe ... -
Comparative cost-effectiveness analysis of voriconazole and fluconazole for prevention of invasive fungal infection in patients receiving allogeneic hematopoietic cell transplants.
(American journal of health-system pharmacy : AJHP : official journal of the American Society of Health-System Pharmacists, 2013-09)<h4>Purpose</h4>The cost-effectiveness of voriconazole versus fluconazole prophylaxis against fungal infections in hematopoietic cell transplant (HCT) recipients is investigated.<h4>Methods</h4>A decision-analytic model ... -
Consensus guidelines for newborn screening, diagnosis and treatment of infantile Krabbe disease.
(Orphanet journal of rare diseases, 2018-02)BACKGROUND:Krabbe disease is a rare neurodegenerative genetic disorder caused by deficiency of galactocerebrosidase. Patients with the infantile form of Krabbe disease can be treated at a presymptomatic stage with human ... -
Consensus opinion on immune-mediated cytopenias after hematopoietic cell transplant for inherited metabolic disorders.
(Bone marrow transplantation, 2021-06)Hematopoietic stem cell transplantation (HCT) has been increasingly used for patients with inherited metabolic disorders (IMD). Immune mediated cytopenias (IMCs) after HCT, manifesting as hemolytic anemia, thrombocytopenia, ... -
Cord blood is the optimal graft source for the treatment of pediatric patients with lysosomal storage diseases: clinical outcomes and future directions.
(Cytotherapy, 2015-06)Initially used as an alternative hematopoietic stem cell source for patients without a human leukocyte antigen-matched bone marrow or peripheral blood stem cell donor, unrelated cord blood (UCB) is now the preferred donor ... -
Defibrotide for the treatment of severe hepatic veno-occlusive disease and multiorgan failure after stem cell transplantation: a multicenter, randomized, dose-finding trial.
(Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2010-07)Therapeutic options for severe hepatic veno-occlusive disease (VOD) are limited and outcomes are dismal, but early phase I/II studies have suggested promising activity and acceptable toxicity using the novel polydisperse ... -
Ethical considerations of using a single minor donor for three bone marrow harvests for three HLA-matched siblings with primary immunodeficiency.
(Pediatric blood & cancer, 2019-04)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, ... -
Factors associated with mortality in transplant patients with invasive aspergillosis.
(Clin Infect Dis, 2010-06-15)BACKGROUND: Invasive aspergillosis (IA) is an important cause of morbidity and mortality in hematopoietic stem cell transplant (HSCT) and solid organ transplant (SOT) recipients. The purpose of this study was to evaluate ... -
Gene therapy offers new hope for children with metachromatic leukodystrophy.
(Lancet (London, England), 2022-01) -
Guidelines for Pediatric Unrelated Cord Blood Transplantation-Unique Considerations.
(Transplantation and cellular therapy, 2021-12)Cord blood (CB) is the stem cell source of choice for approximately 30% of pediatric patients undergoing hematopoietic cell transplantation. Cord blood is readily available and is a particularly appealing stem cell source ... -
Hematopoietic Stem Cell Transplantation to Treat Leukodystrophies: Clinical Practice Guidelines from the Hunter's Hope Leukodystrophy Care Network.
(Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2019-12)The leukodystrophies are a heterogeneous group of inherited diseases characterized by progressive demyelination of the central nervous system leading to devastating neurologic symptoms and premature death. Hematopoietic ... -
In reply.
(Transfusion, 2014-02) -
Inefficient dystrophin expression after cord blood transplantation in Duchenne muscular dystrophy.
(Muscle & nerve, 2010-06)We report a boy who received two allogeneic stem cell transplantations from umbilical cord donors to treat chronic granulomatous disease (CGD). The CGD was cured after the second transplantation, but 2.5 years later he was ... -
Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study.
(Blood, 2015-03)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 ...
