Abstract
Given the profound sensitivity of patients with Fanconi anemia to conventional conditioning regimens before hematopoietic stem cell transplantation (HSCT), we developed a minimally toxic regimen consisting of 2 Gy total body irradiation, 90 mg/m2 fludarabine, and postgrafting immunosuppression with cyclosporine and mycophenolate to treat FA patients undergoing HSCT from HLA-matched unrelated donors. With over 10 months follow-up, our first patient has complete and sustained engraftment. Graft-versus-host disease was limited to mild skin and liver and moderate gut manifestations. We conclude that the approach is well tolerated and ideally suited to reduce regimen-related toxicities while achieving sustained engraftment.
Original language | English (US) |
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Pages (from-to) | 581-583 |
Number of pages | 3 |
Journal | Journal of Pediatric Hematology/Oncology |
Volume | 25 |
Issue number | 7 |
DOIs | |
State | Published - Jul 2003 |
Externally published | Yes |
Keywords
- Fanconi anemia
- Minimal toxicity conditioning regimen
- Stem cell transplantation
ASJC Scopus subject areas
- Pediatrics, Perinatology, and Child Health
- Hematology
- Oncology