Matched unrelated human umbilical cord blood transplantation for X-linked adrenoleukodystrophy.
OBJECTIVE
This report evaluates engraftment, immunologic reconstitution, and sera very-long-chain fatty acid levels after matched unrelated human umbilical cord blood transplantation (HUCBT) for adrenoleukodystrophy (ALD).


METHODS
A child with X-linked ALD underwent matched unrelated HUCBT and administration of granulocyte colony stimulating factor in combination with erythropoietin.


RESULTS
Engraftment was complete and stable, with 100% chimerism. Natural killer cell numbers and function were normal more than 3 months after HUCBT. T cell numbers normalized more than 9 months after HUCBT but T cell mitogenic response remained subnormal. Thirteen months after transplantation, the child is in very good clinical condition with normalization of his sera very-long-chain fatty acid levels.


CONCLUSIONS
Unrelated cord blood transplantation may provide a good source of stem cells for patients with ALD when a matched sibling is not available.
Kapelushnik
J
J
Department of Bone Marrow Transplantation, Hadassah University Hospital, Jerusalem, Israel.
Varadi
G
G
Nagler
A
A
D000326:Adrenoleukodystrophy / Q000097:blood / Q000628:therapy*; D001803:Blood Transfusion; D002648:Child; D003131:Combined Modality Therapy; D004921:Erythropoietin / Q000008:administration & dosage; D005227:Fatty Acids / Q000097:blood; D005312:Fetal Blood; D016179:Granulocyte Colony-Stimulating Factor / Q000008:administration & dosage; D006801:Humans; D008297:Male
D005227:Fatty Acids; D004921:Erythropoietin; D016179:Granulocyte Colony-Stimulating Factor