自体骨髓间充质干细胞移植治疗脑瘫2年随访

 目的 观察自体骨髓间充质干细胞(human mesenchymal stem cells,hMSCs)转化神经干细胞治疗脑瘫的临床效果。方法 选择2010-06至2011-06期间, GMFCS级别III～Ⅴ级,自愿接受干细胞移植的脑瘫患儿11例为移植组,同时选择年龄、性别及疾病严重程度相仿的11例脑瘫患儿为对照组。移植组采用hMSCs转化神经干细胞移植治疗和运动功能锻炼为主的康复治疗,对照组仅仅给予运动锻炼为主的康复治疗。采用脑瘫患儿粗大运动功能评定(GMFM)量表评价移植组和对照组在治疗前和治疗后1、3、6、12、18、24个月的GMFM得分。结果 (1)移植组治疗后1个月、3个月、6个月的GMFM较GMFM0上升率均明显高于对照组(P=0.025);移植组治疗后3个月GMFM3较1个月GMFM1的上升率比较有明显差异,而治疗后6个月GMFM6较3个月GMFM3的上升率比较无统计学差异,提示移植组运动能力在移植后2~3个月时快速显示明显的康复效果。对照组GMFM上升率无此特征;(2)随访12个月、18个月及24个月发现移植组和对照组的GMFM12、GMFM18、GMFM24较之前6个月的GMFM的上升率无差异,而较治疗之前的GMFM0的上升率有显著差异(P<0.01)。(3)两组治疗后1个月、3个月的语言商与治疗前语言商比较,上升率差异均无统计学意义,两组治疗后6个月、12个月移植组语言商较治疗前上升率明显高于对照组(P<0.01)而18个月及24个月随访提示语言商上升率较对照组无增快显现,但较治疗前语言商上升率有明显差异(P<0.05)。结论 hMSCs转化神经干细胞移植治疗脑瘫安全,有效,对脑瘫患儿的运动功能改善明显,在移植后3个月明显显效。6个月以后运动康复效果逐渐消失,但患儿运动能力无倒退现象。语言功能的改善显效较晚,在移植治疗后6个月后显效,12个月后语言快速改善效果消失,但没有语言能力的倒退。

Transplantation of autologous bone marrow mesenchymal stem cells into neural stem cells for treatment of cerebral palsy: 2-year follow-up of 11 cases

Objective To study the efficacy of autologous bone marrow mesenchymal stem cells (human mesenchymal stem cells, hMSCs) transplanted into neural stem cells for the treatment of cerebral palsy. Methods The transplantation group included 11 children with cerebral palsy; another 11 children with cerebral palsy and of the same age, gender and disease severity was as control group. The transplantation group received transplantation of autologous bone marrow mesenchymal stem cells into neural stem cells for the treatment of motor function and exercise-based rehabilitation, where as the control group were only given exercise-based rehabilitation. Both groups underwent gross motor function assessment (GMFM) before treatment and 1 month, 3 months, 6 months, 12 months, 18 months, and 24 months after treatment. Results (1) In the transplantation group 1 month, 3 months, 6 months after treatment,GMFM0 was significantly higher than that in the control group (P<0.025). In transplantation group 3 months after therapy, the rising rate of GMFM3 over GMFM1 had significant difference; and 6 months after treatment the rising rate of GMFM3 over GMFM6 after three months had no statistically significant difference. (2) Follow-up of 12 months, 18 months and 24 months found that between the transplantation group and the control group GMFM12, GMFM18, and GMFM24 had no siqnificant difference in the rate of rise, whereas compared the GMFM the rising rate in with the six preceding months, had significant difference ( P<0.01). (3) 1 month,3 months after the treatment in the two groups, language quotients rising rate compared with pre-treatment language quotients rising rate had no significant difference. 6 months and 12 months after treatment, in transplantation group, rise rate of language quotients was significantly higher than pre-treatment (P<0.01) and 18 months and 24-month follow-up showed that the rise rates of language guofients were not apparently faster than the control, but faster than pre-treatment with a significant difference (P<0.05). Conclusions Transplantation of autologous bone marrow mesenchymal stem cells into neural stem cells for the treatment of children with cerebral palsy is effective and safe, markedly improves motor function, 3 months after transplantation. The exercise rehabilitation effects gradually disappear after 6 months, but the motor ability in children had no retrogradation. Marked improvement of language function is late in the 6 months after transplantation and marked rapid improvement of language disappears after 12 months, but there is no language skills setback.