Expanded Umbilical Cord Blood Transplantation in Cerebral X-Linked Adrenoleukodystrophy: A Case Report.

Umbilical cord blood transplantation is a viable source of stem cells due to accessibility and low incidence of chronic graft-versus-host disease despite human-leukocyte-antigen mismatching. Disadvantages of low stem cell dose in larger recipients include delayed immune reconstitution, graft rejection, and mortality. Successful transplants with ex-vivo expanded cords resulted in approval in malignant disorders and aplastic anemia. We report the first successful expanded cord transplantation in an adolescent with cerebral adrenoleukodystrophy who had no alternative donor/treatment options and required transplant emergently for neurologic preservation. This report highlights a mechanism for timely transplant in metabolic disorders, and preservation of functional advantages despite ex-vivo expansion. Trial Registration: The authors have confirmed clinical trial registration is not needed for this submission.