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

J Pediatr Hematol Oncol. 2003 Jul;25(7):581-3. doi: 10.1097/00043426-200307000-00019.

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.

Publication types

  • Case Reports
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Adult
  • Child
  • Fanconi Anemia / therapy*
  • Female
  • Graft Survival
  • Histocompatibility Testing
  • Humans
  • Immunosuppression Therapy / methods
  • Living Donors*
  • Male
  • Stem Cell Transplantation* / adverse effects
  • Treatment Outcome
  • Whole-Body Irradiation