脐带间充质干细胞鞘内注射治疗脊髓小脑性共济失调

Intrathecal injection of umbilical cord mesenchymal stem cells for spinocerebellar ataxia

背景:脊髓小脑性共济失调是以小脑共济运动障碍为主要临床表现的遗传性变性疾病,迄今为止缺乏有效的药物治疗。目的:观察脐带间充质干细胞鞘内注射治疗脊髓小脑性共济失调的临床疗效。方法:38例脊髓小脑性共济失调患者给予脐带间充质干细胞鞘内注射治疗,1×106/(kg·次),1次/周,4次为1个疗程。38例患者共接受52个疗程治疗,其中27例接受1个疗程治疗,8例接受2个疗程,3例接受3个疗程。采用世界神经病联合会国际合作共济失调量表(ICARS)对患者治疗前后神经功能进行评定,分值越高表示神经功能缺损越严重,采用日常生活能力量表(ADL)对患者治疗前后的日常生活能力量进行评估,分值越低,日常生活能力越强。治疗后对患者进行随访。结果与结论:38例脊髓小脑性共济失调患者52个疗程治疗总有效率为84.6%。治疗结束1个月与治疗前比较ICARS及ADL评分均明显降低(P<0.01)。有效患者行走不稳、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(1例),腰痛(2例),头痛(1例),发热(2例),均在1-3 d内消失。在中位随访39个月(11-59个月)治疗过程中,无治疗相关的不良反应发生。有效患者疾病稳定时间在1-19个月,平均(5.95±4.84)个月。结果表明脐带间充质干细胞鞘内注射治疗是安全的,可在一定时间内一定程度上改善脊髓小脑性共济失调患者的临床症状,延缓疾病进展,多疗程治疗有助于多数患者神经功能的进一步改善。

BACKGROUND：Spinocerebel ar ataxia is a inherited neurodegenerative disease with progressive cerebel ar masonic movement disorders as the main clinical manifestation. So far, no drug is available to control the disease progression. OBJECTIVE：To observe the clinical effect of umbilical cord mesenchymal stem cells in treating spinocerebel ar ataxia by intrathecal injection. METHODS：Thirty-eight cases of spinocerebel ar ataxia were given umbilical cord mesenchymal stem cells by intrathecal injection, 1×10^6/kg once a week, four times as a course. These 38 cases received 52 courses. Among them, 27 cases received 1 course, 8 cases received 2 courses and 3 cases received 3 courses. International Cooperative Ataxia Rating Scale （ICARS） and Activity of Daily Living Scale （ADL） were used to evaluate patients’ neural functions （the greater scores, the more severe damage） and ability of daily living （the lower score, the stronger the ability of daily living）. After treatment, al patients were subjected to fol ow-up visit. RESULTS AND CONCLUSION：The total effective rate of 52 courses of treatment was 84.62%. ICARS and ADL scores were significantly decreased at 1 month after treatment （P〈0.01）. In most of effective patients, unstable walking and standing, slow movement, upper limb fine motor disorder, writing difficulties, dysarthria, eye movement disorders were improved. After treatment, common adverse effects were dizziness （1 case）, low back pain （2 cases）, headache （1 case）, and fever （2 cases）. Al these symptoms disappeared within 1-3 days. No treatment-related adverse events happened in the median fol ow-up of 39 months （11-59 months）. The il ness of effective patients had been stable for 1-19 months, average （5.95±4.84） months. Intrathecal injection of umbilical cord mesenchymal stem cells is safe to ameliorate clinical symptoms to some extent within a certain time. It may delay the progression of spinocerebel ar ataxia. Multiple courses of treatment can help to further improve neurological function in most patients.