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

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Abstract
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.

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