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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https://hdl.handle.net/10161/16160Published Version (Please cite this version)
10.1182/bloodadvances.2017010645Publication Info
van den Broek, Brigitte TA; Page, Kristin; Paviglianiti, Annalisa; Hol, Janna; Allewelt,
Heather; Volt, Fernanda; ... Boelens, Jaap Jan (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.This is constructed from limited available data and may be imprecise. To cite this
article, please review & use the official citation provided by the journal.
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Show full item recordScholars@Duke
Joanne Kurtzberg
Jerome S. Harris Distinguished Professor of Pediatrics
Dr. Kurtzberg is an internationally renowned expert in pediatric hematology/oncology,
pediatric blood and marrow transplantation, umbilical cord blood banking and transplantation,
and novel applications of cord blood and birthing tissues in the emerging fields of
cellular therapies and regenerative medicine. Dr. Kurtzberg serves as the Director
of the Marcus Center for Cellular Cures (MC3), Director of the Pediatric Transplant
and Cellular Therapy Program, Director of the Carolina
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 t
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