Early and late outcomes after cord blood transplantation for pediatric patients with inherited leukodystrophies.
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Leukodystrophies (LD) are devastating inherited disorders leading to rapid neurological deterioration and premature death. Hematopoietic stem cell transplantation (HSCT) can halt disease progression for selected LD. Cord blood is a common donor source for transplantation of these patients because it is rapidly available and can be used without full HLA matching. However, precise recommendations allowing care providers to identify patients who benefit from HSCT are lacking. In this study, we define risk factors and describe the early and late outcomes of 169 patients with globoid cell leukodystrophy, X-linked adrenoleukodystrophy, and metachromatic leukodystrophy undergoing cord blood transplantation (CBT) at an European Society for Blood and Marrow Transplantation center or at Duke University Medical Center from 1996 to 2013. Factors associated with higher overall survival (OS) included presymptomatic status (77% vs 49%;P= .006), well-matched (≤1 HLA mismatch) CB units (71% vs 54%;P= .009), and performance status (PS) of >80 vs <60 or 60 to 80 (69% vs 32% and 55%, respectively;P= .003). For patients with PS≤60 (n = 20) or 60 to 80 (n = 24) pre-CBT, only 4 (9%) showed improvement. Of the survivors with PS >80 pre-CBT, 50% remained stable, 20% declined to 60 to 80, and 30% to <60. Overall, an encouraging OS was found for LD patients after CBT, especially for those who are presymptomatic before CBT and received adequately dosed grafts. Early identification and fast referral to a specialized center may lead to earlier treatment and, subsequently, to improved outcomes.
Published Version (Please cite this version)10.1182/bloodadvances.2017010645
Publication InfoAl-Seraihy, A; Allewelt, Heather; Boelens, JJ; Bordon, V; Diaz, MA; Gennery, AR; ... Volt, F (2018). Early and late outcomes after cord blood transplantation for pediatric patients with inherited leukodystrophies. Blood Adv, 2(1). pp. 49-60. 10.1182/bloodadvances.2017010645. Retrieved from https://hdl.handle.net/10161/16160.
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Jerome S. Harris Professor of Pediatrics
Dr. Kurtzberg conducts both clinical and laboratory-based translational research efforts, all involving various aspects of normal and malignant hematopoiesis. In the laboratory, her early work focused on studies determining the mechanisms that regulate the choice between the various pathways of differentiation available to the pluripotent hematopoietic stem cell. Her laboratory established a CD7+ cell line, DU.528, capable of multilineage differentiation as well as self-renewal, and subse
Assistant Professor of Pediatrics
Stem cell transplantation and cellular therapies to treat a variety of pediatric diseases including malignant (leukemia, lymphoma and myelodysplastic syndrome) and non-malignant conditions (such as leukodystrophies, hemoglobinopathies, bone marrow failure syndromes and immune deficiencies). My clinical interests includes optimizing care for survivors of pediatric stem cell transplantation and chemotherapy. My research interests include the use of cord blood as a graft source and the investigatin
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