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目的:采用粗大运动功能评定(GMFM)量表评价骨髓间充质干细胞移植(BM-MSCs)对小儿脑瘫患儿粗大运动功能的影响。方法:采用开放性自身对照法。对24例脑瘫患儿实施试验研究,其中男13例,女11例,年龄0.5～12岁;痉挛型13例,徐动型8例,混合型3例;术前GMFCS评分IV级18例、V级6例。入院后查体无禁忌证者行自体BM-MSCs移植,方法为经头立体定向干细胞移植和腰穿蛛网膜下腔注射干细胞移植,分别于移植前及移植后12个月内评估粗大运动功能。结果:24例受试者均顺利完成临床研究。其术后1个月、6个月及12个月的GMFM总分及A、B、C功能区得分均较移植前显著提高(P<0.01);在移植后1个月时D、E功能区得分较移植前略有提高,这两个区的得分在移植后半年和1年时与移植前相比均显著提高(均P<0.05);移植后半年的GMFM总分较术后1个月时显著提高(P<0.05)。患儿的年龄、性别对移植的疗效无显著性影响,发病类型、手术方式对疗效影响显著。结论:BM-MSCs移植治疗小儿脑瘫是安全、有效、可行的,可改善脑瘫患者的大运动功能,疗效以术后半年最明显。
OBJECTIVE: To evaluate the effect of bone marrow mesenchymal stem cell transplantation (BM-MSCs) on gross motor function in children with cerebral palsy using the gross motor function assessment (GMFM) scale. Methods: Open self-control method. A total of 24 children with cerebral palsy were enrolled in this study. There were 13 males and 11 females, aged from 0.5 to 12 years. Thirteen were spastic, 8 were Xu and 8 were mixed. The preoperative GMFCS score was 18, V grade in 6 cases. Autologus BM-MSCs were transplanted without contraindications after admission. The methods were stereotactic stem cell transplantation and lumbar subarachnoid injection of stem cell transplantation. The function of coarse motor was evaluated before transplantation and 12 months after transplantation. Results: All the 24 subjects successfully completed the clinical study. The scores of GMFM, A, B and C functional areas at 1 month, 6 months and 12 months after operation were significantly higher than those before transplantation (P <0.01). At 1 month after transplantation, D and E functions The scores of the two districts increased slightly compared with that before transplantation (P <0.05). The total score of GMFM at six months after transplantation was significantly higher than that at one month after transplantation Was significantly increased (P <0.05). The age and sex of children had no significant effect on the curative effect of transplantation. The type of disease and operation method had significant effect on curative effect. Conclusion: BM-MSCs transplantation for children with cerebral palsy is safe, effective and feasible, can improve the large motor function in patients with cerebral palsy, the most obvious effect after six months.