摘要目的:探讨非清髓自体外周造血干细胞（HSC）与单次异体脐带间充质干细胞（MSC）移植治疗1型糖尿病（T1DM）患者的疗效和安全性差异。方法:为回顾性病例对照研究。选取2006年8月至2010年12月于徐州医科大学鼓楼临床学院内分泌科接受HSC移植的T1DM患者9例及MSC移植的患者8例作为研究对象，筛选同时期仅接受常规胰岛素治疗且临床特征相匹配的T1DM患者10例作为对照。收集患者移植前及移植后12个月的胰岛素注射剂量、糖化血红蛋白（HbA 1c）、空腹C肽（FCP）及餐后2 h C肽（2hCP），并计算各指标的变化幅度。将患者移植后12个月的血清FCP和（或）2hCP较移植前增加10%以上定义为临床缓解。观察两种干细胞移植过程中及随访期间患者不良反应的发生情况以判断其安全性。采用 t检验或单因素方差分析、Mann‐Whitney U检验或Kruskal-Wallis H检验、Fisher精确检验进行组间比较。 结果:移植后12个月，HSC移植组、MSC移植组和对照组达到临床缓解的占比分别为7/9、2/8和1/10，HSC移植组临床缓解的占比高于对照组（ P=0.005）；3组的HbA 1c分别为（6.5±1.3）%、（7.7±1.5）%和（9.2±3.5）%；HSC移植组胰岛素注射剂量的下降幅度、FCP的增高幅度均大于对照组，且其2hCP的增高幅度大于MSC移植组和对照组（ P<0.05）。HSC移植组有2例患者脱离胰岛素，MSC移植组和对照组均无患者脱离胰岛素。与移植前相比，移植后12个月HSC移植组的HbA 1c（ t=-3.85， P=0.008）和胰岛素注射剂量（ t=-2.47， P=0.039）均下降，而2hCP（ Z=-2.07， P=0.039）升高；MSC移植组HbA 1c（ t=-3.11， P=0.017）和2hCP（ Z=-2.38， P=0.016）均下降。安全性方面，HSC移植组患者均发生轻度不良反应，而MSC移植组患者无不良反应发生。 结论:两种干细胞技术均有助于改善T1DM患者血糖及保护胰岛功能，HSC移植在改善胰岛功能疗效方面有优于MSC移植的趋势，而MSC移植安全性更高。

abstractsObjective:To explore the efficacy and safety of non-myeloablative autologous peripheral hematopoietic stem cell (HSC) and single allogeneic umbilical cord mesenchymal stem cell (MSC) transplantation in the treatment of patients with type 1 diabetes mellitus (T1DM).Methods:This was a retrospective case-control study. Nine participants receiving HSC transplantation and 8 participants receiving MSC transplantation during August 2006 and December 2010 in Department of Endocrinology, Nanjing Drum Tower Hospital Clinical College of Xuzhou Medical University were selected for analysis, meanwhile 10 matched participants who received insulin therapy alone during the same time period were enrolled as the control. Insulin injection dose, glycated hemoglobin A 1c (HbA 1c), fasting C-peptide (FCP) and 2h-postprandial C-peptide (2hCP) were collected before and 12 months after transplantation, and the change range of each index was calculated. Clinical remission was defined as a 10% increase from baseline in the levels of FCP and/or 2hCP. The adverse reactions of patients during the two kinds of stem cell transplantation and follow-up were observed to judge their safety. T-test, one-way analysis of variance, Mann-Whitney U test, Kruskal-Wallis H test, Fisher′s exact test were used to compare between the groups. Results:At the 12 th month of follow-up, the ratio of clinical remission in the HSC-treated, MSC-treated and control group were 7/9, 2/8, and 1/10 respectively, with HSC-treated group showing significantly higher ratio than that of control group ( P=0.005). By the end of follow-up, the HbA 1c of the three groups were (6.5±1.3)%, (7.7±1.5)% and (9.2±3.5)%, respectively. The decrease of insulin injection dose and increase of FCP in HSC transplantation group were significantly higher than those in control group, and the increase of 2hCP in HSC transplantation group was significantly higher than that in MSC transplantation group and control group ( P<0.05). Two recipients of HSC-treated group achieved insulin independence, but none in the MSC-treated group or control group. Compared to baseline in each group, HbA 1c ( t=-3.85, P=0.008) and insulin injection dose ( t=-2.47, P=0.039) were decreased and 2hCP ( Z=-2.07, P=0.039) were increased at the end of follow-up in HSC-treated group, while HbA 1c ( t=-3.11, P=0.017) and 2hCP ( Z=-2.38, P=0.016) were decreased in MSC-treated group. Considering the safety of stem cell transplantation, all recipients in the HSC-treated group experienced some adverse events, while none presented in the MSC-treated group. Conclusions:Both stem cell therapies improved glycemic control and preserved residue β-cell function in T1DM patients. HSC transplantation had a tendency to be superior to MSC transplantation in improving β-cell function, while the latter presented a higher safety profile.