中国神经再生研究(英文版) ›› 2014, Vol. 9 ›› Issue (16): 1493-1497.doi: 10.4103/1673-5374.139470

• 观点:脊髓损伤修复保护与再生 •    下一篇

颈脊髓损伤:调整临床试验终端使其适合反映有意义的功能改进的重要性

  

  • 收稿日期:2014-07-18 出版日期:2014-08-22 发布日期:2014-08-22

Cervical spinal cord injury: tailoring clinical trial endpoints to reflect meaningful functional improvements

Lisa M. Bond 2, Lisa McKerracher 1, 2   

  1. 1 McGill University, Department of Neurology and Neurosurgery, Montreal, Quebec, Canada
    2 BioAxone BioSciences, Inc., Cambridge, MA, USA
  • Received:2014-07-18 Online:2014-08-22 Published:2014-08-22
  • Contact: Lisa M Bond, Ph.D., Director of Scientific and Regulatory Affairs, BioAxone BioSciences, Inc., One Kendall Square, Building 200, Suite 2203, Cambridge, MA 02139, USA, lisa.bond@bioaxonebio.com.

摘要:

颈脊髓损伤,最具破坏性的和最常见的一种脊髓损伤类型,这种类型的脊髓损伤除了会给患者造成下半身胸/腰/骶瘫痪外,也会导致上肢(手臂和双手)损伤和可见的功能缺失。尽管有许多临床前研究显示再生药物可带来一定效果,但目前仍没有已批准的药物可用于促进脊髓损伤后的修复。与其他神经外伤不同,脊髓损伤几乎没有临床试验,最近,只有一项临床试验完全集中研究颈脊髓损伤(NCT01828203,NCT01502631,NCT01597518)。传统意义上,临床试验用于追踪脊髓损伤受试者的运动/感觉变化。从神经系统评估中得出的最常见主要终端是由美国脊髓损伤协会(ASIA)提出的损伤标度变换和总运动得分。这些广泛的终端对颈椎损伤后恢复的评估价值十分有限,因为颈脊髓损伤后,在手臂没有明显变化时,级数或总运动得分可能会有改变之间的转换,这点对四肢瘫痪病人的生活和康复来说至关重要。

Abstract:

Cervical spinal cord injury (SCI) results in partial to full paralysis of the upper and lower extremities. Traditional primary endpoints for acute SCI clinical trials are too broad to assess functional recovery in cervical subjects, raising the possibility of false positive outcomes in trials for cervical SCI. Endpoints focused on the recovery of hand and arm control (e.g., upper extremity motor score, motor level change) show the most potential for use as primary outcomes in upcoming trials of cervical SCI. As the field moves forward, the most reliable way to ensure meaningful clinical testing in cervical subjects may be the development of a composite primary endpoint that measures both neurological recovery and functional improvement.

Key words: spinal cord injury, SCI, cervical, clinical trial, endpoint, Cethrin, UEMS