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自体骨髓来源干细胞治疗脑瘫患者:概念验证

Administration of Autologous Bone Marrow-Derived Stem Cells for Treatment of Cerebral Palsy Patients: A Proof of Concept.

作者信息

Bansal Himanshu, Singh Lipi, Verma Poonam, Agrawal Anupama, Leon Jerry, Sundell I Birgitta, Koka Prasad S

机构信息

Mother Cell, Anupam Hospital, Rudrapur, Uttarakhand, India.

RegennMed Research and Therapeutics, New Delhi, India.

出版信息

J Stem Cells. 2016;11(1):37-49.

PMID:28296863
Abstract

BACKGROUND

Stem cell therapy is a promising treatment for cerebral palsy, which refers to a category of brain diseases that are associated with chronic motor disability in children. Autologous bone marrow stem cells may be a better cell source and have been studied for the treatment of cerebral palsy because of their functions in tissue repair and the regulation of immunological processes.

METHODS

To assess autologous marrow stem cells as a novel treatment for patients with moderate-to-severe cerebral palsy, a total of 10 cerebral palsy patients were enrolled in this clinical study with 24 months follow-up. A total of 10 cerebral palsy patients received autologous bone marrow cells transplantation (4.5 × 10 mononuclear cells; 90% viability) into the subarachnoid cavity and rehabilitation.

RESULTS

We recorded the gross motor function measurement scores, manual ability function measurement score, and adverse events up to 24 months post-treatment. The gross motor function measurement scores were significantly higher at month 6 post-treatment compared with the baseline scores and were stable up to 24 months follow-up. The increase in manual ability and communication function measurement scores at 6 months were not significant when compared to the baseline score. All the 10 patients survived and none of the patients experienced any serious adverse events or complications.

CONCLUSION

Our results indicated that bone marrow derived MNCs are safe and effective for the treatment of motor deficits related to cerebral palsy. Further randomized clinical trials are necessary to establish the efficacy of this procedure.

摘要

背景

干细胞疗法是治疗脑瘫的一种有前景的方法,脑瘫是一类与儿童慢性运动障碍相关的脑部疾病。自体骨髓干细胞可能是一种更好的细胞来源,因其在组织修复和免疫过程调节中的作用,已被研究用于治疗脑瘫。

方法

为评估自体骨髓干细胞作为中重度脑瘫患者的一种新治疗方法,本临床研究共纳入10例脑瘫患者,并进行了24个月的随访。10例脑瘫患者接受了自体骨髓细胞移植(4.5×10个单核细胞;活力90%)至蛛网膜下腔并接受康复治疗。

结果

我们记录了治疗后24个月内的粗大运动功能测量评分、手动能力功能测量评分及不良事件。治疗后6个月时粗大运动功能测量评分显著高于基线评分,且在24个月随访期内保持稳定。与基线评分相比,6个月时手动能力和沟通功能测量评分的增加不显著。10例患者均存活,且无一例患者发生任何严重不良事件或并发症。

结论

我们的结果表明,骨髓来源的单核细胞治疗与脑瘫相关的运动功能障碍安全有效。需要进一步进行随机临床试验以确定该治疗方法的疗效。

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