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Peer-reviewedCC BYeJHaem

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

  • Rachel E. Granberg1
  • Thomas Pfeiffer1
  • Sanil Sansar2
  • Rafael Galindo3
  • Shalini Shenoy1
  1. 1Division of Hematology/Oncology Department of Pediatrics Washington University School of Medicine St. Louis Missouri USA
  2. 2Gamida Cell Therapeutics Naples Florida USA
  3. 3Division of Pediatric & Developmental Neurology Department of Neurology Washington University School of Medicine St. Louis Missouri USA
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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.

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Cite

BibTeXRIS
BibTeX
@article{granberg2026expanded,
  title = {{Expanded Umbilical Cord Blood Transplantation in Cerebral X‐Linked Adrenoleukodystrophy: A Case Report}},
  author = {Rachel E. Granberg and Thomas Pfeiffer and Sanil Sansar and Rafael Galindo and Shalini Shenoy},
  journal = {eJHaem},
  year = {2026},
  month = oct,
  doi = {10.1002/jha2.70419},
  url = {https://doi.org/10.1002/jha2.70419},
  note = {CC BY},
}
RIS
TY  - JOUR
TI  - Expanded Umbilical Cord Blood Transplantation in Cerebral X‐Linked Adrenoleukodystrophy: A Case Report
AU  - Rachel E. Granberg
AU  - Thomas Pfeiffer
AU  - Sanil Sansar
AU  - Rafael Galindo
AU  - Shalini Shenoy
PY  - 2026
DA  - 2026/10/27
JO  - eJHaem
DO  - 10.1002/jha2.70419
UR  - https://doi.org/10.1002/jha2.70419
AB  - 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.
SN  - 2688-6146
ER  -