骨髓和脐带间充质干细胞联合移植治疗杜氏型肌营养不良1例并文献复习

Bone marrow and umbilical cord mesenchymal stem cells transplantation for duchenne muscular dystrophy:Report of one case and review of the Literature

目的:研究自体骨髓和脐带间充质干细胞联合移植治疗杜氏型肌营养不良(DMD)的疗效和安全性方法对经临床表现、血清酶学、肌电图、基因分析、肌肉活检、肌肉核磁共振成像(MRI)确诊的1例DMD患者采用自体骨髓和脐带间充质干细胞联合移植的方案进行治疗。术后第3、6、9、12个月定期观察患者临床症状及各项疗效指标的变化并结合国内外文献进行综合分析。结果随访12个月,该患者肌力提高,动作灵活。血清肌酸激酶和乳酸脱氢酶较前明显下降,活动能力评分和徒手肌力较前增加,肌电图运动单位电位波幅减低及时限缩短较前改善,肌肉MRI复查显示肌肉病变程度较前减轻、肌肉轮廓较前清晰。结论自体骨髓和脐带间充质干细胞联合移植是一种治疗DMD的有效治疗方法。

Objective：To investigate the clinical effect and safety of combining autologous bone marrow mesenchymal stem cells（BM-MSCs） and umbilical cord mesenchymal stem cells（UC-MSCs） tansplantation in the treatment of Duchenne muscular dystrophy（DMD）.Methods：One case with DMD diagnosed by clinical manifestation,creatine kinase（CK）,lactate dehydrogenase（LDH）,genetic analysis,electromyography,MRI and pathologic examination of biopsied muscle specimens was treated by the concurrent transplantations of BM-MSCs and UC-MSCs.Clinical symptoms and various therapeutic effect index after 3,6,9,12 months were detected.Results：After 12 months of combining tansplantation,muscle strength and agility of action were enhanced,CK and LDH decreased,Activities of daily living（ADL） scale and bare-handed muscle strength increased,amplitude and time limit of motor unit potentials of electromyography ameliorated,and outline of muscle was distinct compared pre-transplantation.Conclusions：The combining transplantation of BM-MSCs and UC-MSCs is convenient,safe and effective in the treatment of DMD.