Allogeneic haematopoietic cell transplantation from alternative donors with a conditioning regimen of low-dose irradiation, fludarabine and cyclophosphamide in Fanconi anaemia

Br J Haematol. 2006 Jul;134(2):208-12. doi: 10.1111/j.1365-2141.2006.06128.x.

Abstract

A pilot study was undertaken using a fludarabine-based conditioning regimen to improve haematopoietic cell transplantation (HCT) from alternative donors in 27 Fanconi anaemia (FA) patients. Patients were conditioned with 150-180 mg/m2 of fludarabine, 40 mg/kg of cyclophosphamide, 5-10 mg/kg of antithymocyte globulin, and 300-450 cGy of thoracoabdominal/total body irradiation. One patient who received unrelated cord blood transplantation failed to engraft, another patient died of sepsis. The 1-year overall survival was 96.3% (95% CI, 89-100). This conditioning regimen exerted an immunosuppressive effect that enabled durable engraftment in alternative donor HCT without severe toxicity.

Publication types

  • Evaluation Study
  • Multicenter Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Child
  • Child, Preschool
  • Combined Modality Therapy
  • Cyclophosphamide / therapeutic use
  • Drug Therapy, Combination
  • Fanconi Anemia / radiotherapy
  • Fanconi Anemia / therapy*
  • Female
  • Graft vs Host Disease / etiology
  • Hematopoietic Stem Cell Transplantation / methods*
  • Humans
  • Infant
  • Male
  • Pilot Projects
  • Transplantation Conditioning / methods*
  • Treatment Outcome
  • Vidarabine / analogs & derivatives
  • Vidarabine / therapeutic use
  • Whole-Body Irradiation

Substances

  • Cyclophosphamide
  • Vidarabine
  • fludarabine