骨髓间充质干细胞移植治疗小儿重度脑性瘫痪的疗效观察

摘要

目的 观察自体骨髓间充质干细胞移植治疗重度脑性瘫痪儿童的临床疗效.方法 对50例重症脑性瘫痪患儿进行骨髓间克质干细胞移植,移植前、后6个月进行自身对照,比较患儿粗大运动和综合功能的进步程度,并随访移植后0、1、3、6、12个月患儿一般情况.结果 50例患儿自体骨髓间充质干细胞培养均成功.移植后6个月失访1例,至12个月失访4例,余46例完成观察.移植后6例患儿在手术后3～5 d即出现显著临床改善,大部分患儿在移植后3个月左右运动能力进步,6个月之后进步幅度减缓.移植前6个月综合功能评估进步程度为(19.14±9.82)%,移植后6个月为(28.49±10.52)%,移植后进步显著大于移植前(P=0.001),其中以运动和认知改善明显.粗大运动功能评估总百分比从移植前的(23.80±10.64)%,上升到移植后(27.31±11.72)%,平均每月上升(1.05±0.49)%,显著大于移植前6个月进步幅度(P=0.001).结论 自体骨髓间充质干细胞移植对康复治疗效果不佳的重症脑瘫患儿有明显临床改善作用,能够有效促进患儿各功能区的发育.%Objective To observe the clinical outcome of bone marrow mesenchymal stem cells transplantation in children with severe cerebral palsy. Methods Fifty children with severe cerebral palsy performed transplantation of bone marrow mesenchymal stem cells. The gross motor function measure ( GMFM ) and comprehensive function assessment were compared at before and 6 months after transplantation. The patients had been followed up at 1, 3, 6 and 12 months after the transplantation. Results Bone marrow mesenchymal stem cells transplantation was successfully conducted in all patients. One patient lost follow-up at 6 months after transplantation. Four patients lost follow-up at 12 months after transplantation. Forty-six patients had completed the follow-up. Six patients experienced obvious clinical improvement in 3 - 5 days after transplantation. The motor function was improved in most of patients at 3 months after transplantation. The motor function improvement decreased after 6 months of transplantation. The rate of comprehensive function assessment was ( 28.49 ± 10.52)％ at 6 months after transplantation, which was significantly higher than ( 19.14 ± 9.82 )％ before transplantation ( P = 0.001 ). The rate of GMFM improved from ( 23.80 ± 10.64 )％ to ( 27.31 ± 11.72 )％ at before and 6 months after transplantation. The average monthly improvement was ( 1.05 ± 0.49 )％, which was significantly higher than ( 0.59 ± 0.38 )％ before transplantation ( P = 0.001 ). Conclusions Transplantation of bone marrow mesenchymal stem cells could improve clinical outcome in children with severe cerebral palsy who failed recuperative therapy, and efficiently promote the development.