Marrow transplantation for Fanconi anaemia: Conditioning with reduced doses of cyclophosphamide without radiation

Journal Article

Nine patients with Fanconi anaemia (FA) were conditioned for HLA-identical sibling bone marrow transplant (BMT) with reduced dose of cyclophosphamide (Cy) without radiation or antithymocyte globulin (ATG). The total dose of Cy was 140 mg/kg (n=2) or 120 mg/kg (n=7). The median patient age was 8 years (range 4-19). Graft-versus-host disease (GVHD) prophylaxis was with methotrexate and cyclosporine (n = 8) or cyclosporine alone (n = 1). All patients had sustained engraftment and two developed grade ≥ II acute GVHD. Cy toxicity included grade ≥2 mucositis seen in all evaluable patients and haemorrhagic cystitis in two patients. The Kaplan-Meier survival estimate is 89% with a median follow-up of 285 d (range 56-528). For the purpose of comparison, this report also reviews and updates long-term follow-up data on 32 previously reported FA patients conditioned with 140-200 mg Cy/kg without radiation. The lowest dose of Cy (without radiation or ATG) after which HLA-identical sibling marrow transplant can be successfully performed in FA patients has yet to be determined, but it appears that uniform and sustained engraftment can be achieved with a Cy dose of as low as 120 mg/kg.

Duke Authors

Cited Authors

  • Flowers, MED; Zanis, J; Pasquini, R; Deeg, HJ; Ribeiro, R; Longton, G; Medeiros, CR; Doney, K; Sanders, J; Bryant, E; Hansen, J; Sullivan, KM; Appelbaum, F; Thomas, ED; Storb, R

Published Date

  • 1996

Published In

Volume / Issue

  • 92 / 3

Start / End Page

  • 699 - 706

PubMed ID

  • 8616040

International Standard Serial Number (ISSN)

  • 0007-1048