BUSULFAN CYCLOPHOSPHAMIDE AS CONDITIONING REGIMEN FOR ALLOGENEIC BONE-MARROW TRANSPLANTATION FOR MYELODYSPLASIA/

Purpose: A non-radiation-containing regimen of busulfan and cyclophosp hamide (BU/CY) was evaluated for toxicity, relapse, and long-term surv ival in patients who received allogeneic bone marrow transplantation ( BMT) for myelodysplasia (MDS). Patients and Methods: Thirty-eight pati ents with MDS, including eight with therapy-related MDS, were prepared for BMT using BU/CY. Results: Fourteen patients remain in first remis sion 18 to 60 months posttransplant. Five patients relapsed after BMT, and four of these patients died. Eight additional patients died of ac ute or chronic graft-versus-host disease (GVHD), and 11 died of regime n-related toxicity, primarily systemic mycoses. Overall survival rate at 2 years was 45% (95% confidence interval [CI], 0.30 to 0.61), with a 24% probability of relapse (95% CI, 0.10 to 0.49). Regimen-related t oxicity was manifested primarily as hepatic dysfunction in 72% of pati ents, with 16% developing overt venoocclusive disease (VOD). Conclusio n: Non-radiation-containing preparative regimens offer long-term survi val in allogeneic BMT for MDS that is comparable to that of radiation- containing regimens, and are useful in patients with therapy-related M DS. Monitoring BU levels may reduce regimen-related mortality and impr ove survival. (C) 1995 by American Society of Clinical Oncology.