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目的:探讨脐血干细胞移植治疗中间型脊髓性肌萎缩症的临床治疗可行性及效果。方法:已确诊的中间型脊髓性肌萎缩症患儿,采用脐血干细胞移植治疗,4次为一疗程,移植途径采用静脉输注(1次)加蛛网膜下腔注射(3次)的方法,治疗前和治疗后半年均需完善神经系统体检、实验室检查、FIM评分、肌电图等。结果:移植治疗后患儿神经系统症状明显改善,FIM评分提高,实验室检查肌酶下降,肌电图提示重收缩每10.0ms所检肌运动单位较前增加。随访10月患儿未出现副反应。结论:应用脐血干细胞移植治疗中间型脊髓性肌萎缩症是有效安全的,可以改善患儿神经功能。
Objective: To investigate the feasibility and effect of clinical treatment of cord spinal cord atrophy with cord blood stem cell transplantation. Methods: The children with intermediate spinal muscular atrophy who had been diagnosed were treated with umbilical cord blood stem cell transplantation for 4 times. The method of transplantation was intravenous infusion (1 time) and subarachnoid injection (3 times) , Before treatment and six months after treatment need to improve the nervous system examination, laboratory tests, FIM score, EMG and so on. Results: After the transplantation, the neurological symptoms were significantly improved, the FIM score was increased, and the muscle enzymes in the laboratory were decreased. The electromyogram showed that the contractile activity increased every ten milliseconds. Follow-up in October did not appear in children with side effects. Conclusion: The application of cord blood stem cell transplantation in the treatment of middle spinal muscular atrophy is effective and safe, can improve children’s neurological function.