BONE-MARROW TRANSPLANTATION IN PATIENTS WITH FANCONI-ANEMIA - EXPERIENCE WITH CYCLOPHOSPHAMIDE AND TOTAL-BODY IRRADIATION CONDITIONING REGIMEN

Eleven patients with Fanconi anemia (FA) underwent bone marrow transpl antation (BMT) between March 1985 and May 1990 in a single institution . Ten patients received bone ma? row from healthy Still human leukocyt e antigen (HLA) matched siblings and one patient from her father (one antigen mismatch). Ten patients were conditioned with cyclophosphamide (Cy) at a dose of 5 mg/kg per day for 4 days followed by total body i rradiation (TBI) for a total of 600 cGy over 3 days. Six of the 11 pat ients are alive and have normal reconstitution of their bone ma?row. M edian follow-lip was 72 months (range 42-84). Three of the 10 patients who received Cy and TBI (two HLA compatible, one antigen, mismatch) h ad graft failure. Five patients developed at least grade III acute gra ft-versus-host disease (GVHD). The rates of graft failure and GVHD are , however, still significantly high. Modification of the conditioning regimen and GVHD prophylaxis is needed to improve the outcome.