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[自体外周血造血干细胞移植治疗1型糖尿病:附16例报告]

[Autologous peripheral blood hematopoietic stem cell transplantation in the treatment of type 1 diabetic mellitus: a report of 16 cases].

作者信息

Feng Kai, Xu Yi-wei, Ye Fu-guang, Xiao Li, Ma Xi-hui, Gao Yu, Zhang Xin, Yao Shang-zhi, Shi Bing-yi

机构信息

Department of Cellular Therapy, PLA Organ Transplantation Centre, Beijing, China.

出版信息

Zhonghua Yi Xue Za Zhi. 2011 Jul 26;91(28):1966-9.

Abstract

OBJECTIVE

To explore the efficacy and safety of autologous peripheral blood hematopoietic stem cell transplantation (APBHST) in patients with type 1 diabetes mellitus.

METHODS

Hematopoietic stem cells were mobilized with cyclophosphamide and granulocyte colony stimulating factor for 16 patients with type 1 diabetes mellitus who admitted to our department during November 2009 to August 2010. And then stem cells were collected from peripheral blood by leukapheresis and cryopreservation. The cells were infused intravenously after conditioning with cyclophosphamide and antithymocyte globulin. To compare the daily dose of exogenous insulin requirements, the serum levels of hemoglobin A1c (HbA1c), C-peptide, islet cell function during the mixed meal tolerance test were measured before and at different times after APBHST. Blood glucose was monitored 7 times a day before and after APBHST. And the adverse effects were recorded during and after APBHST.

RESULTS

The median follow-up was 28 weeks (range: 8 - 44 weeks). Twelve of 16 patients stayed free from insulin at 3 - 20 days post APBHST. And islet cell function greatly improved after APBHST. Four of 16 patients required exogenous insulin but the dosage decreased. And all 4 patients had a poor level of C-peptide before APBHSCT. There were no such severe adverse effects as myelosuppression.

CONCLUSION

Very encouraging results have been obtained in the patients treated with APBHST. There is definite therapeutic effects and safety in a short term. But further follow-up is necessary to confirm the duration of insulin independence and the mechanisms of action.

摘要

目的

探讨自体外周血造血干细胞移植(APBHST)治疗1型糖尿病患者的疗效和安全性。

方法

对2009年11月至2010年8月期间入住我科的16例1型糖尿病患者,采用环磷酰胺和粒细胞集落刺激因子动员造血干细胞。然后通过白细胞分离术从外周血中采集干细胞并进行冷冻保存。在用环磷酰胺和抗胸腺细胞球蛋白预处理后,将细胞静脉输注。比较APBHST前后不同时间点外源性胰岛素的每日需求量、糖化血红蛋白(HbA1c)、C肽血清水平以及混合餐耐量试验中的胰岛细胞功能。在APBHST前后每天监测7次血糖。并记录APBHST期间及之后的不良反应。

结果

中位随访时间为28周(范围:8 - 44周)。16例患者中有12例在APBHST后3 - 20天无需使用胰岛素。并且APBHST后胰岛细胞功能有显著改善。16例患者中有4例需要外源性胰岛素,但剂量减少。并且这4例患者在APBHSCT前C肽水平均较低。未出现骨髓抑制等严重不良反应。

结论

APBHST治疗的患者取得了非常令人鼓舞的结果。短期内有确切的治疗效果和安全性。但需要进一步随访以确认胰岛素不依赖的持续时间和作用机制。

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