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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 autologous bone marrow mesenchymal stem cell transplantation can improve the motor function in patients with progressive muscular dystrophy. Methods: With the approval of the Ethics Committee of the 463 Hospital of PLA, 332 patients with Duchenne muscular dystrophy hospitalized with complete follow-up data from December 2007 to May 2009 in the cell therapy center were enrolled, all of whom were male and 6 ~ 18 years old, mean (9.4 ± 6.1) years; duration of 2 ~ 18 years, mean (6.9 ± 5.7) years. Patients were injected subcutaneously with granulocyte colony-stimulating factor. After 4 days of mobilization, bone marrow was collected and bone marrow mononuclear cells were cultured with Percoll gradient centrifugation for 10-14 days to prepare cell suspension with concentration of (2 ~ 3) × 1010 / L, In the case of partial isograft transplantation, the total amount of mononuclear cells extracted from a single transplant was (1.99 ± 1.18) × 108, and that of mesenchymal stem cells was (1.77 ± 0.84) × 108. Results: Twelve months after transplantation, 332 patients improved the hand muscle strength improvement rate of 46.1%, daily living activity improvement rate of 81.8%, motor function improvement rate of 70.8%. CONCLUSION: Autologous bone marrow mesenchymal stem cell transplantation for the treatment of Duchenne muscular dystrophy can increase the patient’s muscle strength, improve the living ability and improve the motor function in the near future.