中国组织工程研究

中国组织工程研究 ›› 2014, Vol. 18 ›› Issue (41): 6666-6670.doi: 10.3969/j.issn.2095-4344.2014.41.019

• 干细胞移植 stem cell transplantation • 上一篇    下一篇

脐带间充质干细胞鞘内注射治疗脊髓小脑性共济失调

刘 静1，韩冬梅1，丁 丽1，薛 梅1，阎洪敏1，王志东1，朱 玲1，郑晓丽1，董 磊1，郭子宽2，王恒湘1   

  1. 1解放军空军总医院血液科，北京市 100142；2北京放射与辐射医学研究所，北京市 100850
  • 修回日期:2014-09-16 出版日期:2014-10-01 发布日期:2014-10-01
  • 通讯作者: 王恒湘，硕士，主任医师，解放军空军总医院血液科，北京市 100142
  • 作者简介:刘静，女，1966年生，陕西省西安市人，汉族，2000年解放军第四军医大学毕业，硕士，副主任医师，主要从事血液肿瘤治疗及细胞治疗方面的研究。
  • 基金资助:

     首都发展基金项目(2007-2033)；国家高新技术研究发展计划(863项目)(2007AA 022454)；国家重大新药创制基金(20092x09503-019)；军队临床高新技术重大项目(2010gxjs049)

Intrathecal injection of umbilical cord mesenchymal stem cells for spinocerebellar ataxia

Liu Jing1, Han Dong-mei1, Ding Li1, Xue Mei1, Yan Hong-min1, Wang Zhi-dong1, Zhu Ling1, Zheng Xiao-li1, Dong Lei1, Guo Zi-kuan2, Wang Heng-xiang1   

  1. 1Department of Hematology, General Hospital of the Air Force, Beijing 100142, China; 2Department of Experimental Hematology, Beijing Institute of Radiation Medicine, Beijing 100850, China
  • Revised:2014-09-16 Online:2014-10-01 Published:2014-10-01
  • Contact: Wang Heng-xiang, Master, Chief physician, Department of Hematology, General Hospital of the Air Force, Beijing 100142, China
  • About author:Liu Jing, Master, Associate chief physician, Department of Hematology, General Hospital of the Air Force, Beijing 100142, China
  • Supported by:

    the Capital Development Fund, No. 2007-2033; the National High-Technology Research and Development Program of China (863 Program), No. 2007AA 022454; Project of National Great New Drug Research and Development, No. 20092x09503-019; the Military Clinical Advanced Technology Program, No. 2010gxjs049

PDF

847

被引次数

6

摘要：

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。


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：

关键词: 干细胞, 脐带脐血干细胞, 脊髓小脑性共济失调, 脐带间充质干细胞, 鞘内注射, 863项目

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

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