患者报告结局在疗效评价中重要结局值（CIOVs）的确定与解读

摘要/Abstract

摘要： 随着患者报告结局测量（patient-reported outcome measures， PROMs）的广泛使用，越来越多的研究提出为特定的PROMs定义临床重要结局值（clinical important outcom evalues， CIOVs），用以评估解读临床意义或患者可感知的差异。当前国际上常见的CIOVs如最小临床意义重要差值（minimal clinically important difference，MCID）、实质临床获益（substantial clinical benefit， SCB）、患者可接受的症状状态（patient acceptable symptomatic state，PASS）以及最大结局改善（maximal outcome improvement， MOI）。MCID、PASS和SCB值的确定有助于解释群体的治疗结果，而MOI值更适用于个人层面的解读。合理选择CIOVs并将其应用于临床试验以及患者为中心的疗效评价中，可以为疾病、治疗相关的症状、功能、总体健康状况以及生命质量评估提供切实可行的治疗目标。

关键词: font-size:medium, ">患者报告结局；临床重要结局值；最小临床意义重要差值；患者可接受的症状状态；最大结局改善

Abstract: A growing number of studies have proposed to define clinical important outcome values (CIOVs) for patient-reported outcomes to evaluate and interpret clinically meaningness of patient-perceived differences. CIOVs as minimal clinically important difference (MCID)， substantial clinical benefit (SCB)， patient acceptable symptomatic state (PASS)， and maximal outcome improvement (MOI) are currently accepted internationally. The determination of MCID， PASS， and SCB values can be used to explain treatment results in groups， whereas MOI values is appropriate for individual interpretations. CIOVs are capable of providing realistic therapeutic targets for disease， treatment-related symptoms， function， general health status， and quality of life assessment， when selected and applied appropriately in clinical trials and patient-centred effectiveness evaluations.

Key words: font-size:medium, ">Patient-reported outcome； Clinical important outcome values； Minimal clinically important difference； Patient acceptable symptomatic state； Maximal outcome improvement

中图分类号:

申晨, 刘建平. 患者报告结局在疗效评价中重要结局值（CIOVs）的确定与解读[J]. 中国药物评价, 2023, 40(1): 1-6.

SHEN Chen, LIU Jian-ping. Determination and Interpretation of Clinical Important Outcome Values for Patient-reported Outcomes[J]. CHINESE JOURNAL OF DRUG EVALUATION, 2023, 40(1): 1-6.

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