中国神经再生研究(英文版)

中国神经再生研究(英文版) ›› 2017, Vol. 12 ›› Issue (3): 493-498.doi: 10.4103/1673-5374.202918

• 原著:脊髓损伤修复保护与再生 • 上一篇    下一篇

自体外周血单个核细胞蛛网膜下腔移植治疗肌萎缩性侧索硬化：14例安全性分析

  

  • 收稿日期:2017-02-13 出版日期:2017-03-15 发布日期:2017-03-15
  • 基金资助:

    中国国家自然科学基金项目（81471308）；大连市科技计划项目（2015F11GH094）

Transplantation of autologous peripheral blood mononuclear cells in the subarachnoid space for amyotrophic lateral sclerosis: a safety analysis of 14 patient

Xiao-yan Li1, Zhan-hua Liang2, Chao Han1, Wen-juan Wei1, Chun-li Song3, Li-na Zhou3, Yang Liu1, Ying Li1, Xiao-fei Ji2, Jing Liu1   

  1. 1 Regenerative Medicine Center, First Affiliated Hospital of Dalian Medical University, Dalian, Liaoning Province, China; 2 Neurological Department, First Affiliated Hospital of Dalian Medical University, Dalian, Liaoning Province, China; 3 Electromyography Department, First Affiliated Hospital of Dalian Medical University, Dalian, Liaoning Province, China
  • Received:2017-02-13 Online:2017-03-15 Published:2017-03-15
  • Contact: Jing Liu, M.D.,liujing.dlrmc@hotmail.com.
  • Supported by:

    This study was supported by the National Natural Science Foundation of China, No. 81471308; a grant from the Science and Technology
    Plan Project of Dalian City in China, No. 2015F11GH094.

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摘要：

自体外周血单个核细胞通过蛛网膜下腔移植用于肌萎缩性侧索硬化患者的治疗，已有安全性和可行性的少量临床数据。文章回顾性分析了14例自体外周血单个核细胞移植治疗肌萎缩性侧索硬化的安全性和可行性，旨在为今后临床试验提供更多的客观数据。试验首先进行干细胞动员、干细胞采集以及肌萎缩性侧索硬化患者自体外周血单个核细胞(1×109)分离，未经培养直接通过蛛网膜下腔移植回患者体内。试验主要观察指标为移植不同阶段患者的白细胞数量、血清丙氨酸转氨酶及肌酸酐水平、体温等指标，次要观察指标为移植后1，4周时评价14例肌萎缩性侧索硬化患者的肌电图，移植前及移植后1，2，4，12周时评价患者功能独立性评定量表、Berg平衡量表以及构音障碍评价量表评分。结果显示，14例患者在干细胞动员、干细胞采集以及干细胞移植后1周时均未发现与移植相关的细胞毒性反应，且患者白细胞数量、血清丙氨酸转氨酶及肌酸酐水平、体温均处于正常范围，同时患者影像学检测也未见严重的不良反应。但是患者治疗前后肌力等级以及功能独立性评定量表、Berg平衡量表以及构音障碍评价量表评分均没有明显变化。结果表明自体外周血单个核细胞蛛网膜下腔移植治疗肌萎缩性侧索硬化是安全的，但外周血单个核细胞治疗肌萎缩性侧索硬化的效果并不显著，仍需大样本研究进一步确定其临床效果。

ORCID:0000-0001-5687-4004(Xian-yan Li)

关键词: 神经再生, 肌萎缩性侧索硬化, 外周血单个核细胞, 蛛网膜下腔移植, 自体, 临床研究, 安全性, 不良反应

Abstract:

There is a small amount of clinical data regarding the safety and feasibility of autologous peripheral blood mononuclear cell transplantation into the subarachnoid space for the treatment of amyotrophic lateral sclerosis. The objectives of this retrospective study were to assess the safety and efficacy of peripheral blood mononuclear cell transplantation in 14 amyotrophic lateral sclerosis patients to provide more objective data for future clinical trials. After stem cell mobilization and collection, autologous peripheral blood mononuclear cells (1 × 109) were isolated and directly transplanted into the subarachnoid space of amyotrophic lateral sclerosis patients. The primary outcome measure was incidence of adverse events. Secondary outcome measures were electromyography 1 week before operation and 4 weeks after operation, Functional Independence Measurement, Berg Balance Scale, and Dysarthria Assessment Scale 1 week preoperatively and 1, 2, 4 and 12 weeks postoperatively. There was no immediate or delayed transplant-related cytotoxicity. The number of leukocytes, serum alanine aminotransferase and creatinine levels, and body temperature were within the normal ranges. Radiographic evaluation showed no serious transplant-related adverse events. Muscle strength grade, results of Functional Independence Measurement, Berg Balance Scale, and Dysarthria Assessment Scale were not significantly different before and after treatment. These findings suggest that peripheral blood mononuclear
cell transplantation into the subarachnoid space for the treatment of amyotrophic lateral sclerosis is safe, but its therapeutic effect is not remarkable. Thus, a large-sample investigation is needed to assess its efficacy further.

Key words: nerve regeneration, amyotrophic lateral sclerosis, peripheral blood mononuclear cells, subarachnoid space transplantation, autologous, clinical research, safety, adverse events, neural regeneration