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

  • 摘要: 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。

     

    Abstract: Objective To study the efficacy and safety of autologous bone marrow transplantation(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 HbA1c,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 Results The patients were followed up for 62-365 days with a median follow-up time of 265 days.Insulin was withdrawn from 11(68.8%) of the patients 5-114 days after ABMT with a median time of 21 days and a longest time of 360 days.Two of the 11 patients after withdrawal of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.

     

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