骨髓间充质干细胞移植治疗Becker型肌营养不良症58例临床分析

Clinical Analysis of 58 Cases with Becker Muscular Dystrophy Treated with Mesenchymal Stem Cells Transplantation

目的：探讨骨髓间充质干细胞（MSCs）移植治疗Becker型肌营养不良症的临床效果。方法：2007年6月-2009年4月对58例Becker型肌营养不良症患者，进行自身治疗前后对照研究，患者按照3～10μg·kg^-1·d^-1。剂量，皮下注射粒细胞集落刺激因子，动员4d后采集骨髓．利用Ficoll密度梯度离心方法，体外培养每例患者的骨髓干细胞7～10d，然后配制成浓度为2．46×10^10·L^-1的骨髓间充质干细胞悬液，采用经静脉及四肢肌肉局部双移植途径，其一部分经局部肌内注射移植入患者四肢近端肌肉群中，另一部分经静脉移植入患者体内，单次移植提取的单个核细胞总量为（6．25±2．19）×10^8，间充质细胞含量为（1．96±0．92）×10^8。观察移植后3个月，6个月，9个月，12个月患者的肌力、行走间距以及心肌酶谱变化。结果：MSCs移植后75．9％的患者肌力有所改善，行走间距延长，心肌酶谱好转。结论：MSCs双途径移植治疗Becker型进行性肌营养不良症是一种新的有效治疗方法。

Objective：To explore the clinical effect of the mesenchymal stem cells （MSCs） transplantation for the patients with the Becker type muscular dystrophy. Methods： Separated and cultivated the MSCsin vitro for 58 cases with Becker type muscu- lar dystrophy from June 2007 to April 2009, Granulocyte-colony stimulating factor （G-CSF） was infused into patients, and bone marrow was collected following 4 days of mobilization. Bone marrow mononuclear cells were isolated and cultured for 7-10 days by Ficoll gradient centrifugation. Cell suspension （2.46×10^10/L） was prepared. The amount of mononuclear cells was （6.25 ± 2.19） ×11^8 and the amount of mesenchymal ceils was （1.96 ± 0.92） × 10^8. Then adopted two types of transplantation, portion cells were transplanted in the proximal muscles of four limbs by intromuseular injection, the other portion cells were transplanted into the bodies by intravenous injection. The muscular force, walking distance and laboratory data after 3,6,9, 12months were analyzed. Results： Among 58 patients, 75.9% patients＇ muscular force were increased, walking distance were extended and myocardial enzymology became better. Conclusions： MSCs transplantation with two types is one new effective method for Becker type muscular dystrophy.