减低剂量环磷酰胺的预处理方案对重型再生障碍性贫血同胞全相合外周血造血干细胞移植疗效的影响

OBJECTIVE: To respectively analyze the impact of conditioning regimens with a dose-decreased cyclophosphamide (Cy) on the outcome in fully matched sibling donor (MSD) peripheral blood stem cell transplantation (PBSCT) for severe aplastic anemia (SAA). METHODS: Two conditioning regimens with different doses of Cy (150 mg/kg or 120 mg/kg) in combination with fludarabine (Flu) and antithymocyte globulin (ATG) for MSD-PBSCT were investigated in 51 patients with acquired SAA. RESULTS: Overall survival and failure-free survival in patients received 150 mg/kg of Cy (Cy150 cohort) or 120 mg/kg (Cy120 cohort) were 93.5% vs 90.0% (χ(2)=0.170, P=0.680) and 90.3% vs 85.0% (χ(2)=0.285, P= 0.594) respectively. However, either acute or chronic graft-versus-host disease risks were higher in Cy120 cohort than in Cy150 cohort (HR=3.89, 95% CI 1.21–12.53, P=0.023; HR=4.48, 95% CI 1.40–14.32, P= 0.011, respectively). No difference was found for opportunistic infections or graft failure between two cohorts. CONCLUSION: Cy at a dose of 150 mg/kg, in combination with Flu and ATG, was more effective than that of 120 mg/kg Cy to produce improved clinical outcome in the setting of acquired SAA patients after MSD-PBSCT.