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患者报告结局评分的多个有意义变化阈值的三角剖分。

Triangulation of multiple meaningful change thresholds for patient-reported outcome scores.

机构信息

Adelphi Values, Adelphi Mill, Bollington, Cheshire, SK10 5JB, UK.

IQVIA, Reading, Berkshire, UK.

出版信息

Qual Life Res. 2021 Oct;30(10):2755-2764. doi: 10.1007/s11136-021-02957-4. Epub 2021 Jul 28.


DOI:10.1007/s11136-021-02957-4
PMID:34319532
Abstract

PURPOSE: The notion of what constitutes meaningful differences or changes in patient-reported outcome scores is represented by meaningful change thresholds (MCTs). Applying multiple methods to estimate MCTs inevitably results in a range of estimates; however, a single estimate or small range is sought in practice to enable consistent interpretation of scores. While current recommendations for triangulation are appropriate in principle, the vital step of moving from all estimates to a value or small range lacks clarity and is subjective in nature. This article aims to review current triangulation approaches and provide more robust recommendations than what is currently available. METHODS: Current approaches to perform triangulation are described and discussed. Anchor-based estimates are focussed upon due to their recognition as the most valid and developed approach. Recommendations for triangulation are provided. RESULTS: A correlation-weighted average of MCT estimates is recommended to triangulate multiple MCT estimates derived from a single study into a single value, where increased weighting is given to stronger anchor measures. The choice of method to triangulate estimates from several published studies is highly dependent on the availability of information within the publications. MCTs designed for between-group differences, within-group changes, and within-individual changes should be considered separately. CONCLUSION: The recommendations within this article provide a reliable and transparent approach to triangulation when a single value is sought, based on meta-analytic approaches. This approach is preferable to a simple mean of estimates where all are weighted equally, or through 'eyeballing' plotted estimates which is unreliable. We encourage researchers to adopt these methods, but to remain aware of the limitations within each method and further nuances in study design that result in heterogeneity. Sensitivity analyses with a range of plausible values are encouraged; however, the recommendations provide a suitable starting value for inferences. Unresolved issues in triangulation, requiring further exploration, are highlighted.

摘要

目的:患者报告结局评分中具有意义的差异或变化的概念由有意义的变化阈值(MCT)表示。应用多种方法来估计 MCT 不可避免地会导致一系列估计值;然而,在实践中,需要寻求单一的估计值或小范围,以实现对评分的一致解释。虽然目前的三角测量建议原则上是合适的,但从所有估计值到一个值或小范围的关键步骤缺乏明确性,本质上是主观的。本文旨在回顾当前的三角测量方法,并提供比当前可用方法更可靠的建议。

方法:描述和讨论了当前执行三角测量的方法。由于锚定估计值被认为是最有效和最发达的方法,因此重点介绍了它们。提供了三角测量的建议。

结果:建议对从单个研究中得出的多个 MCT 估计值进行加权平均,以将多个 MCT 估计值三角化为单个值,其中对更强的锚定测量值给予更高的权重。从几个已发表的研究中进行三角测量估计值的方法选择高度依赖于出版物中的信息可用性。应分别考虑用于组间差异、组内变化和个体内变化的 MCT。

结论:本文中的建议提供了一种基于荟萃分析方法的可靠且透明的三角测量方法,当寻求单个值时。这种方法优于简单的平均值方法,其中所有值都平等加权,或者通过不可靠的“目测”绘制估计值。我们鼓励研究人员采用这些方法,但要意识到每种方法的局限性以及导致异质性的研究设计中的进一步细微差别。鼓励进行一系列合理值的敏感性分析;然而,建议为推断提供了合适的起始值。突出了三角测量中需要进一步探索的未解决问题。

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本文引用的文献

[1]
The minimal perceived change: a formal model of the responder definition according to the patient's meaning of change for patient-reported outcome data analysis and interpretation.

BMC Med Res Methodol. 2021-6-21

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BMJ. 2020-6-4

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Med Care. 2019-5

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J Patient Rep Outcomes. 2019-4-3

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Eur J Cancer. 2018-10-22

[10]
Minimum clinically important differences in chronic pain vary considerably by baseline pain and methodological factors: systematic review of empirical studies.

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