Matched Unrelated Human Umbilical Cord Blood Transplantation for X-Linked Adrenoleukodystrophy

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Abstract
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). A child with X-linked ALD underwent matched unrelated HUCBT and administration of granulocyte colony stimulating factor in combination with erythropoietin. 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. Unrelated cord blood transplantation may provide a good source of stem cells for patients with ALD when a matched sibling is not available.