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 Univers

SUBMITTER: van den Broek BTA 

PROVIDER: S-EPMC5761624 | biostudies-literature | 2018 Jan

REPOSITORIES: biostudies-literature