马法兰替代环磷酰胺的预处理方案在异基因造血干细胞移植治疗恶性髓系血液病的临床研究

OBJECTIVE: To evaluate melphalan instead of cyclophosphamide in modified busulfancyclophosphamide regimen as a new myeloablative conditioning regimen for the treatment of myeloid malignancies patients receiving allogeneic hematopoietic stem cell transplantation (HSCT). METHODS: The clinic data of 94 myeloid malignancies patients undergoing allogeneic HSCT were analyzed, of which 48 patients received Bu + Cy + Flu + Ara-C, 46 cases Bu + Mel + Flu + Ara-C regimens. Rregimen-related toxicity, engraftment, graft-versus-host disease (GVHD), infection condition, non-relapse mortality, and overall survival were compared between the two groups. RESULTS: All patients achieved neutrophil engraftment. The incidence of grade Ⅲ-Ⅳ oral mucositis and diarrhea in BMFA group was higher than in BCFA group (P<0.05). The incidence of acute GVHD in BMFA group was also higher than in BCFA group but without statistically significant difference (36.5% over 56.5%, P=0.100). With a median follow up of 42 months, the incidence of no relapse mortality in BCFA group was 12.5% and 19.6% in BMFA group (P=0.400). The relapse rate in BMFA group (4.3%) was significantly lower than in BCFA group (25.0%, P=0.009). The overall survival rates were (71.8±6.7)% and (76.1±6.3)% (P=0.852), and diseasefree survival rates were (67.8±8.9)% and (76.1±6.3)% (P=0.567) were comparable between BCFA group and BMFA group. CONCLUSION: Melphalan instead of cyclophosphamide as a new myeloablative conditioning regimen had lower relapse and satisfied disease-free survival rates, but the risk of regimenrelated toxicity and GVHD should be taken into consideration.