Jones Linda A T, Bryden Anne, Wheeler Tracey L, Tansey Keith E, Anderson Kim D, Beattie Michael S, Blight Andrew, Curt Armin, Field-Fote Edelle, Guest James D, Hseih Jane, Jakeman Lyn B, Kalsi-Ryan Sukhvinder, Krisa Laura, Lammertse Daniel P, Leiby Benjamin, Marino Ralph, Schwab Jan M, Scivoletto Giorgio, Tulsky David S, Wirth Ed, Zariffa José, Kleitman Naomi, Mulcahey Mary Jane, Steeves John D
Craig H. Neilsen Foundation, Encino, CA, USA.
Case Western Reserve University, Cleveland, OH, USA.
Spinal Cord. 2018 May;56(5):414-425. doi: 10.1038/s41393-017-0015-5. Epub 2017 Dec 28.
This is a focused review article.
This review presents important features of clinical outcomes assessments (COAs) in human spinal cord injury research. Considerations for COAs by trial phase and International Classification of Functioning, Disability and Health are presented as well as strengths and recommendations for upper extremity COAs for research. Clinical trial tools and designs to address recruitment challenges are identified.
The methods include a summary of topics discussed during a two-day workshop, conceptual discussion of upper extremity COAs and additional focused literature review.
COAs must be appropriate to trial phase and particularly in mid-late-phase trials, should reflect recovery vs. compensation, as well as being clinically meaningful. The impact and extent of upper vs. lower motoneuron disease should be considered, as this may affect how an individual may respond to a given therapeutic. For trials with broad inclusion criteria, the content of COAs should cover all severities and levels of SCI. Specific measures to assess upper extremity function as well as more comprehensive COAs are under development. In addition to appropriate use of COAs, methods to increase recruitment, such as adaptive trial designs and prognostic modeling to prospectively stratify heterogeneous populations into appropriate cohorts should be considered.
With an increasing number of clinical trials focusing on improving upper extremity function, it is essential to consider a range of factors when choosing a COA.
Craig H. Neilsen Foundation, Spinal Cord Outcomes Partnership Endeavor.
这是一篇重点综述文章。
本综述介绍了人类脊髓损伤研究中临床结局评估（COA）的重要特征。阐述了按试验阶段以及《国际功能、残疾和健康分类》对COA的考量，以及上肢COA在研究方面的优势和建议。确定了应对招募挑战的临床试验工具和设计。
方法包括总结为期两天的研讨会中讨论的主题、上肢COA的概念性讨论以及额外的重点文献综述。
COA必须适合试验阶段，特别是在中晚期试验中，应反映恢复与代偿情况，且具有临床意义。应考虑上运动神经元病与下运动神经元病的影响和程度，因为这可能会影响个体对特定治疗的反应。对于纳入标准宽泛的试验，COA的内容应涵盖脊髓损伤的所有严重程度和水平。评估上肢功能的具体措施以及更全面的COA正在开发中。除了合理使用COA外，还应考虑增加招募的方法，如适应性试验设计和预后建模，以便将异质人群前瞻性地分层到合适的队列中。
随着越来越多的临床试验聚焦于改善上肢功能，在选择COA时考虑一系列因素至关重要。
克雷格·H·尼尔森基金会、脊髓结局合作项目。
