自体骨髓间充质干细胞移植治疗假肥大型肌营养不良症疗效分析

目的探讨自体骨髓间充质干细胞（MSCs）移植治疗假肥大型肌营养不良症临床效果。方法对316例假肥大型肌营养不良症患者皮下注射粒细胞集落刺激因子，动员4天后采集骨髓，提取的单个核细胞（MNC），总量为（5．72±1．89）×10^8，培养骨髓单个核细胞7—10天，最终得到间充质干细胞含量为（1．90±0．96）×10^8。应用四肢肌肉内注射的方式进行干细胞移植。结果干细胞移植治疗1年后肌力增加者占总患者数的81．3％。临床升级人数占27．5％。血清肌酸激酶（CK）降低率81．0％，血清乳酸脱氢酶（LDH）降低率86．1％。结论自体MSCs移植可使部分假肥大型肌营养不良症患者肌力改善，生活自理能力增加，血清CK、LDH水平下降，其安全性高，无不良反应，是治疗假肥大型肌营养不良症的一种新方法。

Curative effect analysis of transplantation of autologous mesenchymal stem cells in patients with psudohypertrophic muscular dystrophy

Objective To discuss the clinical effect of transplantation of autologous mesenchymal stem cells in patients with psudohypertrophic muscular dystrophy. Methods Totally 316 patients who were diagnosed with psudohypertrophic muscular dystrophy received a whole year treatment with autologous MSCs transplantation in our center. Parameters before and after treatment were compared. Granulocyte-colany stimulating factor（ G-CSF ） was infused into all patients, and bone marrow was collected following 4 days of mobilization. Bone marrow mononuclear cells （ MNCs ） were cultured for 7 - 10 days and MSCs were got at last. The amount of MSCs was（ 1.90 ×0.96） ×10^8. MSCs were transplanted into all the limbs by intramuscular injection. Results One year after transplantation, increased rate of muscle force of the patients was 81.3% and 27.5% of the patients were upgraded by the transplantation. The decreased rates of serum creatine kinase（CK）and lactic acid dehydrogenase（LDH） were respectively 81.0% and 86. 1%. Conclusion Transplantation of autologous MSCs can make many of the patients with psudohypertrophic muscular dystrophy increase muscle force and living ability. And it can also decrease the level of CK and LDH in blood serum. Because of safety and no bad effect, transplantation of autologous MSCs is a new way to treat with psudohypertrophic muscular dystrophy.