自体骨髓干细胞移植治疗肢带型肌营养不良症33例

Transplantation of autologous bone marrow stem cells for limb-girdle muscular dystrophies in 33 cases

经解放军第四六三医院伦理委员会同意,纳入2007-12/2008-06在血液内分泌科住院的具有完整随访资料的肢带型肌营养不良症患者33例,男27例,女6例,年龄6～46岁,病程2～20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7～10d,配制浓度为2.48×1010L-1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(6.13±2.39)×108,间充质细胞含量为(1.92±0.98)×108。移植后6个月,33例患者的徒手肌力好转率54.5%,日常生活活动能力好转率81.8%,血清肌酸激酶降低率78.8%,乳酸脱氢酶降低率81.8%,肌酐升高率72.7%。提示自体骨髓干细胞移植治疗肢带型肌营养不良症可在近期增加患者肌力,提高生活能力,改善血清肌酸肌酶、乳酸脱氢酶活性及肌酐含量等指标。

Following the agreement of Ethics Committee of the 463 Hospital of Chinese PLA, a total of 33 limb-girdle muscular dystrophy （LGMD） patients, including 27 males and 6 females, aged 6 to 46 years with the course of disease of 2 to 20 years were enrolled from the Department of Hematology and Endocrinology of the 463 Hospital of Chinese PLA from December 2007 to June 2008. 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 Percoll gradient centrifugation. Cell suspension （2.48×10^10/L） was prepared. Stem ceils were transplanted into all over the body by intravenous infusion and extremities by intramuscular injection. The amount of mononuclear cells was （6.13±2.39）×10^8 and the amount of mesenchymal ceils was （1.92±0.98）×10^8. Six months after transplantation, increased rates of bare-handed muscle force and activities of daily living （ADL） were respectively 54.5% and 81.8%. The decreased rates of serum creatine kinase and lactic acid dehydrogenase were respectively 78.8% and 81.8%. The increased rate of creatinine was 72.7%. It was indicated that transplantation of autologous bone marrow stem cells for LGMD can enhance the bare-handed muscle force and activities of daily living and improve values of serum creatine kinase, lactic acid dehydrogenase and creatinine.