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

Abstract

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.

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Published Version (Please cite this version)

10.1182/bloodadvances.2017010645

Publication Info

van den Broek, Brigitte TA, Kristin Page, Annalisa Paviglianiti, Janna Hol, Heather Allewelt, Fernanda Volt, Gerard Michel, Miguel Angel Diaz, et al. (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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Kristin Marie Page

Assistant Professor of Pediatrics

Stem cell transplantation and/or cellular therapies can be used to treat a variety of pediatric diseases including malignancies such as leukemia, lymphoma and myelodysplastic syndrome in addition to certain non-malignant conditions (such as immune deficiencies, inherited metabolic diseases, hemoglobinopathies, and bone marrow failure syndromes). As the Director of the Pediatric Transplant and Cellular Therapy Survivorship Clinic, my goal is optimize the care of survivors of pediatric stem cell transplantation, cellular therapies and chemotherapy. To further support this goal, my research interests include improving patient-reported health related quality of life, access to survivorship care, and further understanding the pathophysiology of certain late effects with the ultimate goal of identifying treatments.


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