人神经干细胞侧脑室移植治疗40例重度脑性瘫痪的临床疗效

Forty cases of cerebral palsy treated with human neural stem cell transplantation

目的观察人神经干细胞（human neural stem cells，hNSCs）移植治疗重度脑性瘫痪（cerebral palsy，CP）的临床疗效。方法40例重度CP患儿进行hNSCs移植治疗，经侧脑室穿刺将hNSCs注入患儿脑室。术前1个月，术时及术后3，6个月采用GMFM、PDMS-FM量表评估患儿大运动、精细运动的发育情况。结果40例患儿中有26例在术后5d至6个月出现不同程度的临床症状的改善（有效率65％），量表评估示：GMFM总百分比术前为（4．52±2．50）％，术后3个月及6个月分别是（7．74±2．94）％、（13．01±6．71）％，术后与术前相比，差异有统计学意义（P〈0．05）。PDMS-FM总百分比术前为（15．01±12．00）％，术后3个月及6个月分别是（20．34±11．91）％、（30．02±12．50）％，术后3个月与术前相比，差异有统计学意义（P〈0．05）。且术后发育速率明显快于手术前，术后1～3个月内进步最为明显，术后6个月后发育有减缓趋势。无严重手术并发症及移植相关不良反应。结论hNSCs移植治疗重症CP是一种有一定疗效的、安全的治疗方法，长期疗效还需进一步观察。

Objective To assess the clinical efficacy of human neural stem cell （hNSC） transplantation in the treatment of severe cerebral palsy （CP） in children. Methods hNSCs were obtained from the forebrain of 10 to 12-week-fetus. Forty children with CP were voluntarily received hNSC transplantation that were injected into cerebral ventricle. The development of motor and fine motor functions were evaluated by GMFM and PDMS-FM 1 month before hNSC transplantation, as well as 3 and 6 months after hNSC transplantation. Results Twenty six （ 65% ） cases displayed improvement from day 5 to month 6 after hNSC transplantation. GMFM assessment showed that the percentage was （4.52±2.50） % 1 month before hNSC transplantation, （7.74±2.94） % 3 months after hNSC transplantation and （13.01±6.71）% 6 months after hNSC transplantation,indicating a significant improvement by the treatment of hNSC transplantation（P〈0.05）. The percentage in PDMS-FM evaluation was （15.01± 12.00）%, （ 20.34± 11.91 ） % and （ 30.02± 12.50） % one month before hNSC transplantation, 3 and 6 months after hNSC transplantation ,respectively, also suggesting a significant improvement induced by hNSC transplantation treatment （P〈0.05）. Moreover,the developmental improvement was the most prominent among 1-3 months post hNSC trans- plantation. Then the development slowed down. Significantly, patients received no hNSC transplantation experienced serious adverse events or complications. Conclusions hNSC transplantation is an effective and safer therapy for severe CP. Future observations are needed to evaluate long-term clinical efficacy of the therapy.