Expanded Umbilical Cord Blood Transplantation in Cerebral X-Linked Adrenoleukodystrophy: A Case Report.
This case report describes the first reported successful ex-vivo expanded umbilical cord blood transplantation in an adolescent with cerebral X-linked adrenoleukodystrophy who lacked an alternative donor or treatment option and required urgent transplantation for neurologic preservation.
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This case report describes the first reported successful ex-vivo expanded umbilical cord blood transplantation in an adolescent with cerebral X-linked adrenoleukodystrophy who lacked an alternative donor or treatment option and required urgent transplantation for neurologic preservation.
Research significance
The reported case provides preliminary human evidence that ex-vivo expanded cord blood can enable timely hematopoietic transplantation for cerebral adrenoleukodystrophy when conventional donors are unavailable; it remains an inference, not established evidence, that this strategy reliably improves neurologic outcomes or transplant safety across patients.
Source abstract
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.