Abstract
Purpose: 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). Patient and 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. Conclusion: Unrelated cord blood transplantation may provide a good source of stem cells for patients with AiD when a matched sibling is not available.
| Original language | English |
|---|---|
| Pages (from-to) | 257-259 |
| Number of pages | 3 |
| Journal | Journal of Pediatric Hematology/Oncology |
| Volume | 20 |
| Issue number | 3 |
| DOIs | |
| State | Published - 1 May 1998 |
| Externally published | Yes |
Keywords
- Adrenoleukodystrophy
- Human umbilical cord blood
- Matched unrelated
- Very-long-chain fatty acids
ASJC Scopus subject areas
- Pediatrics, Perinatology, and Child Health
- Hematology
- Oncology