title: Matched unrelated human umbilical cord blood transplantation for X-linked adrenoleukodystrophy.
abstract: 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.
authors.affiliation: Department of Bone Marrow Transplantation, Hadassah University Hospital, Jerusalem, Israel.
authors.forename: J
authors.initials: J
authors.lastname: Kapelushnik
authors.forename: G
authors.initials: G
authors.lastname: Varadi
authors.forename: A
authors.initials: A
authors.lastname: Nagler
mesh_terms: 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
chemical_list: D005227:Fatty Acids; D004921:Erythropoietin; D016179:Granulocyte Colony-Stimulating Factor