摘要
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：Many animal and clinical studies have reported that the safe and effective usage of umbilical cord blood-derived mesenchymal stem cells（UCB-MSCs） transplantation for treatment of neurological genetic diseases.OBJECTIVE：To investigate the feasibility and effect of UCB-MSCs transplantation in the treatment of spinal muscular atrophy（SMA）.METHODS：A child admitted at January 2010 had been confirmed as having SMA,and drug and rehabilitation therapies were invalid.Then,the child received UCB-MSCs transplantation via the first intravenous infusion and three times of subarachnoid injection,once a week,（4-6）×10^7 cells once and four times as a course.Neurological physical examination,biochemical test,muscle enzymes detection,FIM scoring and electromyography（EMG） examination were conducted.RESULTS AND CONCLUSION：Compared with prior to transplantation,the level of muscle enzymes decreased,FIM scores were increased from 68 to 93 points,EMG results showed that the motor units with re-contraction in each 10.0 ms were increased that the motor function was improved,the lower extremity muscle strength elevated,and the self-care ability was improved in the SMA child at 6 months after transplantation.During the 10-month follow-up,the child had no adverse effects.It is indicated that UCB-MSCs transplantation is effective to treat SMA,and the neurological function has a remarkable restoration.
出处
《中国组织工程研究与临床康复》
CAS
CSCD
北大核心
2011年第36期6837-6840,共4页
Journal of Clinical Rehabilitative Tissue Engineering Research
