自体骨髓间充质干细胞移植改善进行性肌营养不良症患者运动功能的研究

Clinical observation of ameliorating motor function in 332 cases with Duchenne muscular dystrophies by autologous bone marrow mesenchymal stem cells transplantation

目的:探讨自体骨髓间充质干细胞移植临床治疗进行性肌营养不良症能否改善患者运动功能。方法:经解放军第四六三医院伦理委员会同意,纳入2007年12月至2009年5月在细胞治疗中心住院的具有完整随访资料的杜氏型肌营养不良症患者332例,均为男性,年龄6～18岁,平均(9.4±6.1)岁;病程2～18年,平均(6.9±5.7)年。患者皮下注射粒细胞集落刺激因子,动员4d后采集骨髓,Percoll梯度离心培养骨髓单个核细胞10～14d,配制浓度为(2～3)×1010/L的细胞混悬液,经静脉及四肢肌肉局部等量移植,单次移植提取的单个核细胞总量为(1.99±1.18)×108,间充质干细胞含量为(1.77±0.84)×108。结果:移植后12个月,332例患者的徒手肌力好转率46.1%,日常生活活动能力好转率81.8%,运动功能好转率70.8%。结论:自体骨髓间充质干细胞移植治疗杜氏型肌营养不良症可在近期增加患者肌力,提高生活能力,改善运动功能。

Objective To investigate whether treatment with autologous bone marrow mesenchymal stem cells transplantation can ameliorate the motor function in patients with Duchenne muscular dystrophies.Method Following the agreement of Ethics Committee of the 463 Hospital of Chinese PLA,a total of 332 cases with Duchenne muscular dystrophy（DMD）（all were male）,aged 6-18 years,with the course of disease of 2-18 years were enrolled from the Department of Cell treatment Center of the 463 Hospital of Chinese PLA from December 2007 to May 2009.Granulocyte-colony stimulating factor（G-CSF）was injected subcutaneously,and bone marrow was collected 4 days later.Bone marrow mononuclear cells were isolated and cultured for 10-14 days by Percoll gradient centrifugation.Cell suspension [（2-3）× 1010/L] was prepared and transplanted into all over the body by intravenous injection and intramuscular injection in the four extremities.The amount of mononuclear cells was（1.99 ± 1.18）× 108 and the amount of mesenchymal cells was（1.77 ± 0.84）× 108.Result Twelve months after transplantation,the improvement rates of bare-handed muscle force and activities of daily living（ADL）were 46.1% and 81.8% respectively.Motor function was ameliorated by 70.8%.Conclusion Transplantation of autologous bone marrow mesenchymal stem cells treated for DMD can enhance the bare-handed muscle force and activities of daily living and ameliorate motor function.