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背景:国内外已有实验动物和临床应用脐血间充质干细胞移植治疗神经系统遗传性疾病安全、有效的诸多报道。目的:探讨脐血间充质干细胞移植治疗儿童进行性脊髓性肌肉萎缩症的可行性及效果。方法:2010-01收治1例确诊为儿童进行性脊髓性肌肉萎缩症患儿,经药物及康复治疗无效,行脐血间充质干细胞移植治疗。移植途径采取首次静脉输注,后3次蛛网膜下腔注入,1次/周,每次细胞数量达(4～6)×107个,4次为1个疗程。治疗前和治疗后半年均需完善神经系统体检、实验室检查、肌酶、FIM评分、肌电图等。结果与结论:移植6个月与移植前比较,肌酶下降,FIM评分由68分提高到93分,肌电图检查重收缩每10.0ms所检肌运动单位增加两三个,双下肢肌力增加,生活自理能力改善。随访10个月,患者未出现明显的不良反应。提示脐血间充质干细胞移植治疗该例儿童进行性脊髓性肌肉萎缩症患儿有效,其神经功能恢复明显。
Background: There are many reports on the safe and effective treatment of genetic diseases of nervous system by experimental animals and clinical application of umbilical cord blood mesenchymal stem cells transplantation at home and abroad. Objective: To investigate the feasibility and effect of cord blood derived mesenchymal stem cell transplantation for treatment of progressive spinal muscular atrophy in children. Methods: One child diagnosed with progressive spinal muscular atrophy was treated in our hospital from January 2010 to January 2010. The drug and rehabilitation treatment were ineffective and treated with cord blood MSCs transplantation. Transplantation approach to take the first intravenous infusion, 3 times subarachnoid injection, 1 / week, each time the number of cells (4 ~ 6) 107, 4 times for a course of treatment. Neurological examination, laboratory tests, muscle enzymes, FIM scores, electromyography, etc. need to be completed before treatment and six months after treatment. RESULTS AND CONCLUSION: Compared with before transplantation, the muscle enzymes decreased and the FIM score increased from 68 to 93. The electromyographic examination showed a decrease of muscle movement of 10.0% , Self-care ability to improve. Follow-up of 10 months, patients did not appear obvious adverse reactions. Prompt cord blood derived mesenchymal stem cell transplantation in children with progressive spinal muscular atrophy in children with effective, and its neurological recovery was significant.