【摘要】 患者报告结局（PRO）的最小重要性差值（MID）的估计基于多种选择原则和统计方法，研究的可信度参差不齐，应用时应考虑其评价结果。就广为接受的基于锚定法的MID研究而言，由于涉及锚的选择与估计的统计方法，不同研究可因临床试验设计的差异、测量对象和情境的差异、偏倚控制等因素而导致PRO的MID不同。为此，加拿大McMaster大学研制了基于锚定法的PRO的MID可信度评价工具。该工具包括了5个核心评价问题和4个附加问题。5个核心评价问题包括以下评估框架：（1）患者或代诉人是否直接对PRO和锚进行评估？（2）锚是否易于理解且与患者或代诉人相关？（3）锚是否与PRO表现出良好的相关性？（4）MID的精确性如何？（5）用于估计MID的锚的阈值或组间差异是否反映了小但重要的差异？此外，涉及转换条目的4个附加评价问题包括：（1）MID估计的基线和后续测量之间的时间是否最佳？（2）转换条目与随访时的PRO分数是否具有令人满意的相关性？（3）转换条目与基线时的PRO分数是否相关？（4）转换条目与PRO变化分数的相关性是否明显大于转换条目与随访时PRO分数的相关性？因工具中各条目的相对权重不确定，且取决于研究中报告的情况，所以没有对条目评分，而是采用定性评级的方法进行总体判断。本文对这一工具的条目进行介绍，并通过实例对评价过程加以解读，以便读者更加清晰地理解和应用，以优化临床试验、系统评价、卫生技术评估和临床实践指南中PRO结果的呈现和解释。更多还原

【Abstract】 The estimation of the minimal important difference(MID) in patient-reported outcomes(PRO) relies on various selection principles and statistical methods, resulting in varying degrees of credibility among studies. When applying these findings, it is crucial to consider their evaluation outcomes. In the context of widely accepted MID studies based on the anchoring method, the credibility of the MID of PRO is influenced by the selection of anchors and the statistical methods employed for estimation. Variations in the anchors utilized, differences in clinical trial designs,disparities in the characteristics of measurement subjects and environment, as well as the control of biases in studies, can all contribute to inconsistencies in the MID of PRO. In response to this, McMaster University in Canada has developed a credibility evaluation tool specifically for MID studies in PRO. The tool comprises five core items and four additional items. The five core items encompass an evaluation framework that assesses:(1) Is the patient or necessary proxy responding directly to both the PRO and the anchor?(2) Is the anchor easily understandable and relevant for patients or necessary proxy?(3)Has the anchor shown good correlation with the PRO?(4) Is the MID precise?(5) Does the threshold or difference between groups on the anchor used to estimate the MID reflect a small but important difference? The four additional items concerning transition-rated anchors assess:(1) Is the amount of elapsed time between baseline and follow-up measurement for MID estimation optimal?(2) Does the transition item have a satisfactory correlation with the PRO score at follow-up?(3) Does the transition item correlate with the PRO score at baseline?(4) Is the correlation of the transition item with the PRO change score appreciably greater than the correlation of the transition item with the PRO score at follow-up? Given the relative weights of each item in the tool are uncertain and environment-dependent, items are not scored; instead, an overall judgment is made using a qualitative rating approach. This article introduces the specific items of this tool and illustrates the evaluation process through a case study to improve its use in optimizing PRO results presentation and interpretation in clinical trials, reviews, assessments, and guidelines.更多还原