Powers John H, O'Connell Robert J
Clinical Research Directorate, Frederick National Laboratory for Cancer Research, Frederick, Maryland, USA.
Department of Medicine, George Washington University School of Medicine, Washington, District of Columbia, USA.
Clin Infect Dis. 2025 Aug 6;81(Supplement_1):S4-S10. doi: 10.1093/cid/ciaf319.
Evidence from clinical research studies, in addition to patient values and clinical experience, helps practitioners best apply medical interventions to clinical practice. Part of evaluating the evidence is understanding the outcomes used as endpoints in research studies. The most direct evidence of patient benefit comes from endpoints that evaluate the effects of interventions on patient survival, symptoms, and function, and the impact of symptoms and function on health-related quality of life. In this article, we discuss how to develop endpoints for clinical research studies focusing on patient-reported outcome measures, including those measuring patient symptoms, function, and health-related quality of life. We discuss the process for determining (1) which concepts to measure, including which concepts are most relevant to patients (content validity); (2) who performs the measurement and which measurement properties to use (construct validity); and (3) how to analyze and interpret the results obtained using the chosen measurements.
除了患者价值观和临床经验外，临床研究的证据有助于从业者将医学干预措施最佳地应用于临床实践。评估证据的一部分是理解在研究中用作终点的结果。患者受益的最直接证据来自于评估干预措施对患者生存、症状和功能的影响，以及症状和功能对健康相关生活质量的影响的终点。在本文中，我们讨论如何为专注于患者报告结局测量的临床研究制定终点，包括那些测量患者症状、功能和健康相关生活质量的测量。我们讨论确定以下内容的过程：（1）测量哪些概念，包括哪些概念与患者最相关（内容效度）；（2）谁进行测量以及使用哪些测量属性（结构效度）；以及（3）如何分析和解释使用所选测量获得的结果。
