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) |
|---|---|
| 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 |
Keywords
- Fanconi anemia
- Minimal toxicity conditioning regimen
- Stem cell transplantation
ASJC Scopus subject areas
- Pediatrics, Perinatology, and Child Health
- Hematology
- Oncology
Fingerprint Dive into the research topics of 'Reduced toxicity and prompt engraftment after minimal conditioning of a patient with fanconi anemia undergoing hematopoietic stem cell transplantation from an HLA-matched unrelated donor'. Together they form a unique fingerprint.
Cite this
Kurre, P., Pulsipher, M., Woolfrey, A., Maris, M., Sandmaier, B., Kiem, H. P., & Storb, R. (2003). Reduced toxicity and prompt engraftment after minimal conditioning of a patient with fanconi anemia undergoing hematopoietic stem cell transplantation from an HLA-matched unrelated donor. Journal of Pediatric Hematology/Oncology, 25(7), 581-583. https://doi.org/10.1097/00043426-200307000-00019