自体骨髓和脐血间充质干细胞联合移植治疗杜氏型肌营养不良症疗效分析

Curative effect analysis on transplantation of autologous bone marrow and cord blood mesenchymal stem cells in patients with Duchenne muscular dystrophy

目的探讨自体骨髓间充质干细胞（BMSC）和脐血间充质干细胞（CB-MSC）联合移植治疗杜氏型肌营养不良症临床效果。方法 2007年6月至2009年10月我中心应用自体BMSC和UMSC联合移植治疗杜氏型肌营养不良症患者,并对132例移植满1年的患者采用自身治疗前后对照的方法进行研究。全部患者皮下注射粒细胞集落刺激因子,动员4天后采集骨髓,Percoll梯度离心,提取的单个核细胞（MNC）,总量为（5.81±1.82）×10^8,培养骨髓单个核细胞7-14天,最终得到间充质干细胞含量为（1.88±0.92）×10^8。应用四肢肌肉内注射的方式进行干细胞移植。采集人脐带血,制备CB-MSC方法同BMSC,应用静脉滴注方式将CB-MSC移植至患者体内。结果干细胞移植治疗1年后肌力增加者占总患者数的82.6%（109/132）。临床升级人数占24.2%（32/132）。血清肌酸激酶（CK）降低率81.8%,血清乳酸脱氢酶（LDH）降低率85.6%。结论自体BMSC和UMSC联合移植可使部分杜氏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,是治疗杜氏型肌营养不良症的一种新方法 。

Objective To discuss the clinical effect of transplantation of autologous bone marrow mesenchymal stem cells（BMSC） and umbilical cord blood mesenchymal stem cells（CB-MSC） in patients with Duchenne muscular dystrophy.Methods Totally 132 patients who were diagnosed with Duchenne muscular dystrophy received a whole year treatment with autologous BMSC and UMSC transplantation from March 2008 to September 2009 in our center.Parameters before and after treatment were compared.Granulocyte-colony stimulating factor（G-CSF） was infused into all patients,and bone marrow was collected following 4 days of mobilization.Bone marrow mononuclear cells（MNC） were isolated by Percoll gradient centrifugation.The amount of MNC was（5.81±1.82）×10^8.The MNC were cultured for 7-14 days and BMSC were obtained at last.The amount of BMSC was （1.88±0.92）×10^8.BMSC were transplanted into all the limbs by intramuscular injection.We collected human umbilical cord blood and got CB-MSC by the same way as BMSC.CB-MSC were transplanted into the body of the patients by intravenous injection.Results One year after transplantation,increased rate of muscle force of the patients was 82.6%（109/132）,and 24.2%（32/132） of the patients were upgraded by the transplantation.The decreased rates of serum creatine kinase（CK） and lactic acid dehydrogenase（LDH） were respectively 81.8% and 85.6%.Conclusion Transplantation of autologous BMSC and CB-MSC can make many of the patients with Duchenne muscular dystrophy increase muscle force and living ability.And it can also decrease the level of CK and LDH in blood serum.Transplantation of autologous BMSC and CB-MSC is a new way to treat Duchenne muscular dystrophy because of safety and no bad effect.