[Treatment of Fanconi anemia by bone marrow transplantation]

We report on our experience with allogenic bone marrow transplantation in the treatment of Fanconi anemia. Eight patients were treated, ranging in age from 5 to 17 years. Beside severe hemopoietic insufficiency, all patients exhibited typical cytogenetic abnormalities with an increased rate of chromosomal breaks, while constitutional signs of the disorder were rather variable. Marrow donors were HLA-identical siblings. For conditioning, we used cyclophosphamide at 5 mg/kg on 4 consecutive days followed by thoraco-abdominal irradiation at 5 Gy with full lung shielding. For prophylaxis of graft versus host disease, cyclosporin A was given except in 3 cases who received T-cell depleted marrow. In 2 of the latter cases, graft failure was observed, successfully reversed in one by retransplantation. All others showed prompt and stable engraftment of donor cells. Complications of graft versus host disease developed in 2, requiring prolonged immunosuppressive treatment. Of 8 transplanted patients, 7 survive. With the exception of a recently treated girl, they have normal stable marrow functions. Our results confirm that successful treatment of Fanconi anemia is possible in a majority of patients with HLA-identical donors.