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[方法学漫谈。二十五。结局指标、替代结局和中间指标]

[Roaming through methodology. XXV. Outcome measures, surrogate outcomes, and intermediate measures].

作者信息

Boers M

机构信息

Academisch Ziekenhuis Vrije Universiteit, afd. Klinische Epidemiologie en Biostatistiek, Amsterdam.

出版信息

Ned Tijdschr Geneeskd. 2000 Oct 7;144(41):1949-52.

Abstract

Clinical medicine is aimed at decreasing the current or future burden of disease. Disease outcome is best expressed as burden of disease or change thereof, and as such the only true 'outcome measure' in clinical research. However, outcome research is expensive in costs and time expenditure, and sometimes impossible. Therefore study results are often expressed in intermediate measures. These measures tell us something about the disease process and the pathophysiological consequences of the disease and should have a relation with outcome. If this relation is strong, the measure is called 'surrogate outcome'. Intermediate measures and surrogate outcomes have advantages and disadvantages. The reader of clinical trial results first has to decide whether the answer to the study question is relevant in his personal situation. If so, the applicability of a measure can be simply appraised by answering 3 questions: 'Is the measure truthful (relevant, unbiased)?'; 'Does it discriminate between situations that are of interest?'; 'Is the measure feasible in my setting?'

摘要

临床医学旨在减轻当前或未来的疾病负担。疾病结局最好用疾病负担或其变化来表示,因此它是临床研究中唯一真正的“结局指标”。然而,结局研究在成本和时间投入方面都很昂贵,有时甚至是不可能的。因此,研究结果通常用中间指标来表示。这些指标能让我们了解疾病过程以及疾病的病理生理后果,并且应该与结局相关。如果这种关系紧密,该指标就被称为“替代结局”。中间指标和替代结局都有其优缺点。临床试验结果的读者首先要确定该研究问题的答案在其个人情况下是否相关。如果是,那么可以通过回答三个问题来简单评估一个指标的适用性:“该指标是否真实(相关、无偏倚)?”;“它能否区分感兴趣的不同情况?”;“该指标在我的环境中是否可行?”

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