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 marrow transplantation.

PATIENTS AND METHODS

They were conditioned with cyclophosphamide (CY) (20-150 mg/kg), antilymphocyte globulin, and thoracoabdominal irradiation (4-6 Gy). The dose of CY for preconditioning was adjusted individually, based on the in vitro effect of CY metabolites on the chromosomes of patients with Fanconi anemia. Four patients received marrow from human leukocyte antigen (HLA)-identical siblings, and one received marrow from his HLA phenotypically identical father.

RESULTS

All patients achieved engraftment, and acute graft-versus-host disease (GVHD) grade II or more was not observed. Three developed chronic GVHD. All patients are surviving 2-5 years after grafting, with hematological improvement.

CONCLUSIONS

These results indicate that the individual dose adjustment of CY used for preconditioning may prevent graft failure and severe acute GVHD.