脐带间充质干细胞治疗橄榄桥脑小脑萎缩的安全性及有效性

目的:观察人脐带间充质干细胞(UC-MSC)治疗橄榄桥脑小脑萎缩(OPCA)的临床效果及不良反应。方法:2010年7月至2011年5月,对武汉大学中山医院神经内科及细胞治疗研究中心收治的OPCA患者25例,给予UC-MSC静脉滴注联合鞘内注射治疗,每次静脉滴注约3×107个细胞,鞘内注射约7×107个细胞,1次为1个疗程。采用世界神经病联合会国际合作共济失调评分量表(ICARS)、统一多系统萎缩评估量表(UMSARS)及Bar-thel指数对患者治疗前及疗程结束后1月、3个月时神经功能进行评定,对比治疗前及治疗后上述各项指标的变化。并对患者年龄、病程、神经损伤程度(UMSARS评分)与治疗后改善程度相关性进行分析。结果:本组患者治疗1月、3个月时与治疗前比较,ICARS、UMSARS评分指数均明显降低,Barthel指数升高(均P<0.05)。患者运动性共济失调、书写困难、吞咽困难、构音障碍等临床症状得到明显改善,改善程度与患者年龄和病程均没有明显关系性(P>0.05),与病情严重程度呈负相关(r=-0.356,P<0.05),病情越重改善越轻微。治疗后3月,患者疗效稳定,ICARS评分、UMSARS评分、Barthel指数较治疗后1月无明显改变,差异无统计学意义(P>0.05)。不良反应及并发症主要集中在治疗后3d内,主要为:发热、腰痛、头痛,经相应处理,症状很快缓解。极个别患者出现暂时的小便潴留;未见严重不良反应。结论:UC-MSC静脉滴注联合鞘内注射治疗OPCA可以一定程度地改善患者的临床症状,提高患者生活质量,在一定程度上延缓病情进展,未见严重不良反应。

Safety and Efficacy Evaluation on the Treatment of Olivopontocerebellar Atrophy with Umbilical Cord Mesenchymal Stem Cells

Objective: To observe the clinical effect and side effects of umbilical cord mesenchymal stem cells(UC-MSCs) in the treatment of olivopontocerebellar atrophy.Methods: From July 2010 to May 2011,the patients who were treated in our Cell Therapy and Research Center,25 cases with olivopontocerebellar atrophy were given UC-MSCs by intrathecal injection at a dose of 7×107 cells and intravenous injection at a dose of 3×107 cells in a course.The movement ability and the quality of daily life were evaluated with the International Cooperative Ataxia Rating Scale(ICARS),Unified Multiple System Atrophy Rating Scale(UMSARS) and Barthel Index before the treatment,1 and 3 month(s) after treatment.All the scores were compared before and after the treatment.The correlation analysis of age,the course of disease,the degree of nerve injury before treatment(scores of UMSARS) respectively to the degree of improvement were performed using statistical analysis method.Results: The scores were significantly decreased after treatment(after 1 month and 3 months respectively,both P<0.05).The symptoms,including unstable walking and standing,slow movement,upper limb fine motor disorders,writing difficulties and dysarthria,were greatly improved.The degree of improvement was not related to age and the course of disease(P>0.05 respectively),but it was negatively correlated with the severity of disease(r=-0.356,P<0.05).The efficacy was stable after treatment,the scores of ICARS,UMSARS,and Barthel Index in 3 months showed no significant difference as compared with that respectively in 1 month(P>0.05).The observed side effects including febrile,dizziness,back pain and headache were disappeared within 1-3 days.After expectant treatments,the symptoms were soon abated.Few patients had uroschesis,and no serious adverse reactions were found.Conclusion: UC-MSCs implantation by intrathecal injection and intravenous injection is safe,and able to ameliorate clinical symptoms,improve life quality and delay disease progression of olivopontocerebellar atrophy patients to some extent as well.There were no serious adverse reactions.