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目的 评价异基因造血干细胞移植(Allo-HSC)治疗中应用白消安每日1次静脉滴注联合环磷酰胺预处理方案的疗效和安全性. 方法 收集首都医科大学宣武医院2004年1月至2012年6月以白消安/环磷酰胺(Bu/Cy)为预处理方案进行Allo-HSC治疗连续病例的病历资料进行回顾性分析.将患者分为口服组[2004年1月至2006年6月住院患者,口服白消安4.0 mg/(kg·d),共3 d]和静脉组[2006年7月至2012年6月住院患者,静脉滴注白消安3.2 mg/(kg·d),共3 d].记录2组患者不良反应发生率、造血功能重建情况及生存率. 结果 共收集到患者50例,静脉组34例,口服组16例.2组患者性别、年龄、输入细胞数差异无统计学意义(P＞0.05).静脉组患者口腔黏膜炎、胃肠道反应、肝损害发生率均明显低于口服组[11.7％ (4/34)比43.8％ (7/16),17.6％ (6/34)比50.0％(8/16),20.6％(7/34)比50.0％(8/16),均P＜0.05]；未发现肝静脉闭塞病及癫痫等不良反应发生.静脉组和口服组患者均获得造血重建,2组外周血中性粒细胞计数≥0.5×109/L和血小板计数≥20×109/L所需时间差异无统计学意义[(14.3 ±3.5)d比(15.6±4.0)d,(17.5±5.0)d比(19.0±6.7)d,均P＞0.05].静脉组和口服组患者5年生存率分别为(69.5±12.1)％和(62.5±12.1)％,组间差异无统计学意义(P＞0.05). 结论 恶性血液病的Allo-HSC治疗中预处理方案采用白消安每日1次静脉滴注联合环磷酰胺较口服白消安有更好的安全性,而且疗效确切.“,”Objective To evaluate the efficacy and safety of pretransplant conditioning regimens with intravenous (Ⅳ) infusion of busulfan once daily and cyclophosphamide in patients undergoing allogeneic stem cell transplantation allo-HSCT.Methods The data of consecutive patients treated with a conditioning regimen with busulfan and cyclophosphamide (Bu/Cy) before undergoing allo-HSCT in Xuanwu Hospital of Capital Medical University from January 2004 to June 2012 were collected and retrospectively analyzed.All patients were divided into the oral group [the inpatients were treated with oral Bu 4.0 mg/(kg · d) for 3 days from January 2004 to June 2006] and the Ⅳ infusion group [the inpatients were treated with Ⅳ infusion of Bu 3.2 mg/(kg · d) for 3 days from July 2006 to June 2012].The incidence of adverse reactions,the situation of hematopoietic reconstitution,and survival rate in the two groups were recorded.Results A total of 50 patients were collected.Of them,34 patients were in the Ⅳ infusion group and 16 patients were in the oral group.There was no significance differences in gender,age,and numbers of transplanted stem cells between the two groups (P ＞ 0.05).The incidence of oral mucositis,gastrointestinal reactions,and liver damage in the Ⅳ infusion group were lower than those in the oral group [11.7％ (4/34) vs.43.8％ (7/16),17.6％ (6/34) vs.50.0％ (8/16),20.6％(7/34)vs.50.0％ (8/16),respectively,all P ＜ 0.05] Adverse reactions such as hepatic veno-occlusive disease or epilepsy were not found.The hematopoietic reconstitutions were achieved in all patients in the two groups.The time that peripheral blood neutrophil count ≥0.5 × 109/L and platelet count ≥20 × 109/L required were (14.3 ± 3.5)d and (15.6 ±4.0)d,(17.5 ±5.0)d and (19.0 ±6.7)d in the Ⅳ infusion and the oral groups,respectively.There were no differences between the two groups (P ＞ 0.05).The 5-year survival rates were (69.5 ± 12.1) ％ and (62.5 ± 12.1) ％ in the Ⅳ infusion and the oral groups,respectively.There were no differences between the two groups (P ＞ 0.05).Conclusion Pretransplant conditioning regimen with Ⅳ infusion of busulfan once daily and cyclophosphamide in patients with hematological malignancies and undergoing allogeneic stem cell transplantation is safer than that with oral busulfan and its efficacy is clear.