A fludarabine-based protocol for bone marrow transplantation in Fanconi's anemia

Bone Marrow Transplant. 1997 Dec;20(12):1109-10. doi: 10.1038/sj.bmt.1701016.

Abstract

Allogeneic bone marrow transplantation (BMT) is an effective therapy for Fanconi's anemia (FA). However, mortality and transplant-related complications are usually high due to increased sensitivity to the alkylating agents and radiation commonly used for pre-transplant conditioning. Fludarabine monophosphate is a purine analogue that has been proven effective as a conditioning agent for chronic lymphocytic leukemia patients. We report a child with FA in leukemic transformation with thrombocytopenia and 20% myeloblasts who underwent successful BMT following conditioning with fludarabine/ATG/cyclophosphamide. The regimen was well tolerated, no transplant-related complications were observed, and engraftment was rapid. The child is currently 10 months post-BMT, in excellent clinical condition with a normal blood count, 100% chimerism and no sign of graft-versus-host disease (GVHD). We suggest that this fludarabine-based regimen may be effective in the conditioning of standard, as well as transforming, FA patients for BMT.

Publication types

  • Case Reports

MeSH terms

  • Antineoplastic Agents / therapeutic use*
  • Child
  • Cyclophosphamide / administration & dosage
  • Cyclophosphamide / therapeutic use
  • Fanconi Anemia / drug therapy
  • Fanconi Anemia / therapy*
  • Female
  • Humans
  • Leukemia, Myeloid / etiology
  • Leukemia, Myeloid / therapy*
  • Male
  • Preleukemia / etiology
  • Preleukemia / therapy*
  • Remission Induction
  • Risk Factors
  • Transplantation Conditioning*
  • Vidarabine / analogs & derivatives*
  • Vidarabine / therapeutic use

Substances

  • Antineoplastic Agents
  • Cyclophosphamide
  • Vidarabine
  • fludarabine