目的：分析比较单份与双份非亲缘脐血造血干细胞移植治疗血液疾病的临床效果。方法：回顾性分析2006 年至2016 年国内8 家移植中心185 例单份脐血干细胞移植（single-unit umbilical cord blood transplantation，SU-UCBT）与66 例双份脐血干细胞移植（double-unit umbilical cord blood transplantation，DU-UCBT）病例临床资料，比较SU-UCBT和DU-UCBT 2 种移植方式的植入率、中性粒细胞和血小板植入时间，恶性和非恶性血液病患者接受SU-UCBT和DU-UCBT 后急性移植物抗宿主病（acute graft vs host disease，aGVHD）发生情况和受者生存率。结果: SU-UCBT和DU-UCBT受者植入率分别为78.9%和70.7%，其中恶性血液病患者植入率SU-UCBT为89.1%，DU-UCBT为82.5%。非恶性血液病患者SU-UCBT和DU-UCBT植入率分别为64.0%和53.8%，SU-UCBT和DU-UCBT的植入率相似（P>0.05）。SU-UCBT和DU-UCBT受者中性粒细胞植入时间中位数都为18 d，血小板植入时间中位数分别为40 d 和27 d，无显著差异（P>0.05）。SU-UCBT和DU-UCBT受者Ⅰ、Ⅱ、Ⅲ和Ⅳ度aGVHD发生率分别为22.6%、22.6%、8.2%、12.3%和21.3%、14.9%、10.6%、10.6%，均无显著差异（均P>0.05）。SU-UCBT和DU-UCBT受者中位生存时间分别为1 460 d（17~2 200 d）和988 d（21~2 200 d），6 年累积存活率分别为42.5%和40.4%，无显著差异（P>0.05）。结论：SU-UCBT和DU-UCBT植入成功患者的中性粒细胞和血小板植入时间、aGVHD发生率、存活时间及6 年累积存活率均无显著差异，提示DU-UCBT是安全有效的移植选择。但仍需完善SU-UCBT和DU-UCBT评价体系及进一步探究DU-UCBT的植入机制，以便指导科学地选择UCBT方式。

Objective: To comparatively analyze clinical efficacy of single- unit and double- unit nonrelative cord blood transplantation for treatment of hematologic diseases. Methods: Clinical data of the 185 patients received single-unit umbilical cord blood transplantation (SU-UCBT) and the 66 patients received double-unit umbilical cord blood transplantation (DU-UCBT) in 8 domestic transplant centers during 2006 to 2016 were retrospectively analyzed.It was compared that implantation rates, implantation time of neutrophil granulocyte and platelet between SUUCBT and DU-UCBT, as well as occurrence of acute graft vs host disease (aGVHD) and survival rates of the patients with malignant hematonosis and with non malignant hematonosis after SU-UCBT and DU-UCBT. Results:Implantation rates of the patients received SU-UCBT and DU-UCBT were 78.9% and 70.7% respectively, among them implantation rates of the patients with malignant hematonosis were 89.1% for SU-UCBT and 82.5% for DUUCBT as well as implantation rates of the patients with non malignant hematonosis were 64.0% for SU-UCBT and 53.8% for DU-UCBT, implantation rates of SU-UCBT and DU-UCBT were similar (P>0.05). In the patients received SU-UCBT and DU-UCBT, median implantation times of neutrophil granulocyte were all 18 d, median implantation times of platelet were 40 d and 27 d respectively, the difference was not remarkable (P>0.05). Incidence rates of aGVHD at I, II, III, IV grades in the patients received SU-UCBT and DU-UCBT were 22.6%, 22.6%, 8.2%,12.3% and 21.3%, 14.9%, 10.6%, 10.6% respectively, all of the differences were not evident (all P>0.05). Median survival time in the recipients of SU-UCBT and DU-UCBT were 1 460 d (17~2 200 d) and 988 d (21~2 200 d), and their cumulative survival rate of 6 years were respectively 42.5% and 40.4%, all differences were not obvious (P>0.05). Conclusion: Between the patients successfully received SU-UCBT and DU-UCBT, implantation times of neutrophil granulocyte and platelet, incidence rate of aGVHD, survival time and cumulative survival rate of 6 years were not significant, which suggests that DU-UCBT could be a safe and effective selection of UCBT. However, the evaluation system of SU-UCBT and DU-UCBT still needs to be perfected and implantation mechanism of DUUCBT needs to make a thorough inquiry, so as to scientifically guide selection of UCBT.

上海市自然科学基金资助项目（No. 16ZR1429200）；上海市科委研发平台专项资助项目（No.16DZ2293000）；2016 年上海市专利试点企业项目资助（No.001083816000041）；上海张江国家自主创新示范区专项发展资金重大项目资助（No. ZJ2017-ZD-010）