自体骨髓移植治疗儿童1型糖尿病

Autologous bone marrow transplantation for type 1 diabetic mellitus in children

目的探讨自体骨髓移植治疗1型糖尿病的有效性及安全性。方法 16例1型糖尿病患者采集自体骨髓并予以冻存;采用环磷酰胺+兔抗人胸腺细胞球蛋白方案预处理后经静脉回输骨髓。观察移植前后胰岛素注射剂量、糖化血红蛋白(HbA1c)水平、C肽水平、血糖水平及不良反应。结果随访(62-365)d,中位随访时间265d,68.8%(11/16)患者术后停用胰岛素,开始停用胰岛素时间在骨髓移植后(5-114)d,中位时间为21d,最长停用胰岛素360d。11例停用胰岛素患者中2例于移植后2个月左右重新使用胰岛素,5例未停用者胰岛素用量均较移植前减少(P<0.05)。移植后患者C肽水平较移植前明显上升(P<0.05),所有患者未出现长期骨髓抑制等严重不良反应。结论自体骨髓移植治疗1型糖尿病,初步结果显示临床有效性及安全性,远期疗效有待进一步验证。

Objective To study the efficacy and safety of autologous bone marrow transiglantation（ABMT ） for type 1 diabetes mellitus（DM）. Methods Autologous bone marrow, collected from 16 type 1 DM patients, was stored in a refrigerator. The bone marrow preconditioned with cyclophosphamide and antithymocyte globulin was infused into type 1 DM patients through the veins. Dose of insulin, levels of HbAlc, C-peptide and blood glucose were measured, and adverse effects were recorded before and after ABMT. Results The patients were followed up for 62-365 days with a median follow-up time of 265 days. Insulin was withdrawn from 11（68.8%） of the patients 5-114 days after ABMT with a median time of 21 days and a longest time of 360 days. Two of the 11 patients after withdrawal of insulin received insulin again about 2 months after ABMT, the other 5 received a smaller dose of insulin after ABMT than before ABMT（P〈0.05）. The C-peptide level was significantly higher in the patients after ABMT than before ABMT（P〈0.05）. No severe adverse effects such as long-term myelosuppression occurred in any of the patients. Conclusion ABMT is effective and safe for type 1 DM patients and its long-term effect needs to be further confirmed.