鞘内注射脐带间充质干细胞治疗神经系统疾病的安全性和疗效研究（英文）

Safety and Therapeutic Effect of Intrathecal Administration of Umbilical Cord Mesenchymal Stem cells in Neurological Disorders

目的:探讨腰穿鞘内注射脐带间充质干细胞治疗神经系统疾病的安全性和疗效并且分析腰穿治疗过程中遇到的各种技术难题。方法:2008年12月至2011年5月88例伴有神经系统疾病的患者给予鞘内注射干细胞治疗,并且评价其在治疗过程中的技术难题,采用HAI评分系统对这些患者神经功能的恢复情况进行评估,定期观察患者的临床症状、生物学指标和影像学检查观察其治疗前后的变化。结果:88例患者中具有技术难题的患者有20例,主要表现为在鞘内注射脐带间充质干细胞的过程中需要行全麻补充、腰穿定位困难等,18例患者在治疗过程中出现副作用(头痛、低热、腰痛和下肢痛),但这些副作用在48小时内经过系统治疗后完全缓解。随访1年,50例患者神经功能得到一定改善,包括15例脊髓损伤患者、10例脑瘫患者、10例脑外伤后综合征患者、5例脑梗死后综合征患者、5例脊髓小脑共济失调患者和5例运动神经元病患者。结论:鞘内注射脐带间充质干细胞治疗神经系统疾病是安全和有效的,由于随访时间较短,有必要对这种治疗模式进行扩大的、双盲、安慰剂对照研究来进一步推广其临床应用。

Objective： Advances in stem cell biology have generated intense interest in the prospect of transplanting stem cells into the nervous system for the treatment of refractory neurological diseases. Here we investigate the safety of intrathecal administration of umbilical cord mesenchymal stem cells by lumbar puncture and analyze the technical difficulties and short and long-term effects of umbilical cord mesenchymal stem cells transplantation in various neurological conditions. Methods： Eighty-eight patients underwent subarachnoid placement of umbilical cord mesenchymal stem cells between December 2008 and May 2011 in the affiliated Ren Ming hospital of Hubei University of Medicine. Technical difficulties in the form of localization of subarachnoid space, number of attempts, and postprocedural complications were evaluated. Functional evaluation was done with Hanser Ambulation Index by the stem cell transplant team on a regular basis. All patients were followed up for more than one year after the treatment. Clinical symptoms and related biochem- ical index and photographic examinations were observed regularly. Results： Of 88 patients, we encountered technical difficulties in 20 patients（23%） in the form of general anesthesia supplementation and difficulty localizing the lumbar space. In 18（20.5%） patients, side ef- fects were observed （headache, low-grade fever, low back pain and lower limb pain）, which resolved with symptomatic treatment within 48 hours. On follow-up one year, functional indices improved in 50 （56.8%） patients, including 15 patients with spinal cord injury, 10 pa- tients with cerebral palsy, 10 patients with post-traumatic brain syndrome, 5 patients with post-brain infarction syndrome, 5 patients with spinocerebellar ataxias, and 5 patients with motor neuron disease. Conclusion： Intrathecal administration of umbilical cord mesenehymal stem cells is safe and effective with no long-term adverse effects in neurological disorders. The encouraging results provide compelling evidence to support the concept that in patients with profound neurological defects and inefficient conventional cure, there is a promise of restoration of lost tissue and improvement of function. These data support expanded double blind, placebo-controlled studies for this treatment modality.