Intrathecal injection of umbilical cord mesenchymal stem cells for spinocerebellar ataxia

doi:10.3969/j.issn.2095-4344.2014.41.019

Abstract

Abstract:

BACKGROUND: Spinocerebellar ataxia is a inherited neurodegenerative disease with progressive cerebellar 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 spinocerebellar ataxia by intrathecal injection.

METHODS: Thirty-eight cases of spinocerebellar ataxia were given umbilical cord mesenchymal stem cells by intrathecal injection, 1×10⁶/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, all patients were subjected to follow-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). All these symptoms disappeared within 1-3 days. No treatment-related adverse events happened in the median follow-up of 39 months (11-59 months). The illness 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 spinocerebellar ataxia. Multiple courses of treatment can help to further improve neurological function in most patients.

中国组织工程研究杂志出版内容重点：干细胞；骨髓干细胞；造血干细胞；脂肪干细胞；肿瘤干细胞；胚胎干细胞；脐带脐血干细胞；干细胞诱导；干细胞分化；组织工程

全文链接：

Key words: umbilical cord, mesenchymal stem cell transplantation, injections, spinal, cerebellar ataxia

CLC Number:

R394.2

Liu Jing, Han Dong-mei, Ding Li, Xue Mei, Yan Hong-min, Wang Zhi-dong, Zhu Ling, Zheng Xiao-li, Dong Lei, Guo Zi-kuan, Wang Heng-xiang. Intrathecal injection of umbilical cord mesenchymal stem cells for spinocerebellar ataxia[J]. Chinese Journal of Tissue Engineering Research, 2014, 18(41): 6666-6670.

Figures/Tables 1

2.1 参与者数量分析 38例脊髓小脑性共济失调患者共接受52个疗程(4次为1疗程)脐带间充质干细胞鞘内注射治疗，每个疗程治疗后均进行观察及随访，所有病例均进入结果分析。 2.2 临床疗效 38例脊髓小脑性共济失调患者52个疗程治疗中，44个疗程有效，有效率为84.6%，无效的8个疗程中6个疗程发生于接受第1个疗程治疗的患者，2个疗程发生于接受第2个疗程治疗的患者。11例接受2个或3个疗程治疗的患者中，9例在后续疗程治疗中神经系统功能获得进一步改善。 38例脊髓小脑性共济失调患者52个疗程治疗结束1个月与治疗前比较，ICARS、ADL评分差异均有显著性意义(表1)。脊髓小脑性共济失调患者52个疗程治疗结束1个月与治疗前ICARS各单项：姿势和步态障碍、动态功能(共济运动、描图)障碍、语言障碍、眼球运动障碍评分比较差异有显著性意义(表2)。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。有效的44个疗程中，姿势和步态改善37例次，动态功能(共济运动、描图)改善33例次，语言障碍改善13例次，眼球运动障碍改善5例次。 2.3 不良反应及并发症脐带间充质干细胞治疗中及治疗后，患者的生命体征均平稳。1例腰椎穿刺后去枕平卧时感头晕，起来后症状逐渐消失，而后经相关检查表明与其患颈椎病有关。2例治疗后感腰痛，1例头痛，2例低至中度发热，未经特殊处理，1-3 d后症状消失，无其他不良反应。 2.4 随访中位随访时间39个月(11-59个月)，随访期间无治疗相关不良事件发生，有效患者治疗后疾病稳定时间在1-19个月，平均(5.95±4.84)个月，之后疾病出现进展。"

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