异基因造血干细胞移植患者白消安每日一次静脉滴注联合环磷酰胺预处理方案的疗效及安全性

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。

Efficacy and safety of pretransplant conditioning regimens with intravenous infusion of busulfan once daily and cyclophosphamide in patients undergoing allogeneic stem cell transplantation

Objective To evaluate the efficacy and safety of pretransplant conditioning regimens with intravenous （IV） infusion of busulfan once daily and cyelophosphamide 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 mueositis, 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.