临床疗效评价结局指标的理论框架概述

Summary of the theoretical framework of outcome for clinical efficacy evaluation

结局指标分类及术语表达尚未达成共识,阻碍了研究结局之间的比较和合并。本研究叙述性综合世界卫生组织对健康的定义、5Ds、国际功能、残疾和健康分类、患者报告结局测量信息系统、Porter’s结局层次、健康相关生活质量概念模型、风湿学中的结局测量Filter 2.0核心集、Cochrane结局框架、Williamson/Clarke结局等9个框架,介绍各框架内容并进行比较,认为Williamson/Clarke结局框架层次结构清晰,内容全面,适用于所有疾病结局分类,明确研究之间结局表达的一致性,应在临床试验、实践中被推荐。

No consensus has been reached on the taxonomy and terminology of outcome,which hinders the comparison and merger of outcomes between studies.This study narrates and synthesizes nine outcome frameworks,including World Health Organization’s definition of health,5Ds,international classification of functioning,patient-reported outcomes measurement information system,Porter’s outcome hierarchy,health-related quality of life conceptual model,outcome measurement in rheumatdogy Filter 2.0 core set,Cochrane outcome framework,and Williamson/Clarke outcome,introduces and compare framework’contents.It is concluded that the Williamson/Clarke outcome framework has a clear hierarchical structure and comprehensive content,which is suitable for the taxonomy of all disease outcomes,and clarify the consistency of outcome expression between studies.It should be recommended in clinical trials and practice.