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

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...

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Bibliographic Details
Published in:Blood advances 2018-01, Vol.2 (1), p.49-60
Main Authors: van den Broek, Brigitte T.A., Page, Kristin, Paviglianiti, Annalisa, Hol, Janna, Allewelt, Heather, Volt, Fernanda, Michel, Gerard, Diaz, Miguel Angel, Bordon, Victoria, O'Brien, Tracey, Shaw, Peter J., Kenzey, Chantal, Al-Seraihy, Amal, van Hasselt, Peter M., Gennery, Andrew R., Gluckman, Eliane, Rocha, Vanderson, Ruggeri, Annalisa, Kurtzberg, Joanne, Boelens, Jaap Jan
Format: Article
Language:English
Subjects:
Adrenoleukodystrophy - mortality
Adrenoleukodystrophy - therapy
Child
Child, Preschool
Cord Blood Stem Cell Transplantation
Female
Humans
Infant
Leukodystrophy, Globoid Cell - mortality
Leukodystrophy, Globoid Cell - therapy
Leukodystrophy, Metachromatic - mortality
Leukodystrophy, Metachromatic - therapy
Male
Risk Factors
Survival Analysis
Time Factors
Transplantation
Treatment Outcome
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Summary: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 80 pre-CBT, 50% remained stable, 20% declined to 60 to 80, and 30% to
ISSN:2473-9529
2473-9537
DOI:10.1182/bloodadvances.2017010645