BONE-MARROW TRANSPLANTATION FOR FANCONI-ANEMIA - ADJUSTMENT OF THE DOSE OF CYCLOPHOSPHAMIDE FOR PRECONDITIONING

Purpose: Five patients with Fanconi anemia have been treated by bone m arrow transplantation. Patients and Methods: They were conditioned wit h cyclophosphamide (CY) (20-150 mg/kg), antilymphocyte globulin, and t horacoabdominal irradiation (4-6 Gy). The dose of CY for preconditioni ng was adjusted individually, based on the in vitro effect of CY metab olites on the chromosomes of patients with Fanconi anemia. Four patien ts received marrow from human leukocyte antigen (HLA)-identical siblin gs, and one received marrow from his HLA phenotypically identical fath er. Results: All patients achieved engraftment, and acute graft-versus -host disease (GVHD) grade II or more was not observed. Three develope d chronic GVHD. All patients are surviving 2-5 years after grafting, w ith hematological improvement. Conclusions: These results indicate tha t the individual dose adjustment of CY used for preconditioning may pr event graft failure and severe acute GVHD.