脐带间充质干细胞治疗多系统萎缩小脑型的安全性及疗效研究

目的观察脐带间充质干细胞(UC-MSCs)鞘内注射,治疗多系统萎缩小脑型(MSA-C)的安全性及临床疗效。方法自2009年12月至2012年4月,对40例MSA-C患者给予UC-MSCs鞘内注射治疗,每次每Kg体重给予1×106个细胞,1次/周,4次为1个疗程。本组中29例治疗1个疗程,9例患者治疗2个疗程,2例患者治疗3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)及日常生活量表(ADL)对患者治疗前后的神经功能进行评定。治疗后对患者进行随访。结果 40例MSA-C患者共53个疗程,其中47疗程有效(有效率88.68%)。11例接受多疗程治疗的患者中,9例在后续疗程中疗效进一步提高。治疗结束1个月后ICARS及ADL均比治疗前明显降低(P<0.01)。治疗有效患者多表现为行走、站立不稳,运动迟缓,上肢精细动作障碍,书写困难,构音障碍,眼球运动障碍等临床症状得到改善。治疗后常见的不良反应有头晕(4例)、腰痛(1例)、头痛(2例)、发热(2例),均在1~3 d内消失。随访27~55个月(中位随访时间40个月),无治疗相关的副作用发生,有效患者疾病稳定时间为2~13个月(平均5.88±2.86个月),之后疾病再次出现进展。结论 UC-MSC鞘内注射治疗是安全的,可在一定程度上改善MSA-C患者的临床症状,多疗程治疗有助于多数患者神经功能的进一步改善,延缓疾病进展。

Effectiveness of Umbilical Cord Mesenchymal Stem Cells in Treating Multiple System Atrophy-Cerebellar Type

Objective To observe the effectiveness of intrathecal injection with umbilical cord mesenchymal stem cells （UC-MSCs） in treating multiple system atrophy-cerebellar type （MSA-C）. Methods From December 2009 to April 2012, 40 patients with MSA-C were given UC-MSCs intrathecal injection with the dosage of 1 ×10^6 cells/Kg for once a week, 4 times as a course. Twenty-nine patients were treated with 1 course, 9 patients with 2 courses and 2 patients with 3 courses. International Cooperative Ataxia Rating Scale （ICARS） and Activity of Daily Living Scale （ADL） were used to evaluate patients＇ neural function and ability of daily living. Patients were followed up after treatment. Results In 53 courses, 47 courses are effective （the effective rate was 88.68%）. Compared with the ICARS and ADL scores before treatment, patients’ scores were significantly decreased 1 month after treatment （P〈0.01）. In most effective cases, clinical symptoms such as unstable walking and standing, slow movement, upper limb fine motor disorder, writing difficulties, dysarthria, eye movement disorders were improved. During treatment, common adverse effects were observed, as dizziness （4 cases）, low back pain （1 cases）, headache （2 case） and fever （2 cases）, all these symptoms disappeared within 1-3 days. No treatment-related adverse events were observed during 27-55 months （median 40 months） of follow-up. The stable effective period of patients ws 2-13 months （average 5.88±2.86 months）, then progressed. Conclusion UC-MSCs intrathecal injection is safe, and can ameliorate clinical symptoms to some extent. Multiple courses of treatment can further improve neurological function and delay illness progression in most patients.