调整功率计算以纳入优势边际:有什么影响?

Samuel Bishara
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摘要

本文探讨了超优边际在研究功率计算中的应用。传统的研究功率计算主要是为了拒绝任何差值的零假设,而超优差值则不同,它设定了一个具有临床意义的阈值。这种方法与非劣效性计算类似,但方向相反,尽管有潜在的好处,但很少使用。采用超优差将两组之间可能存在的差异(效应大小)与最小临床显著性差异的概念分开,如果没有超优差,就会出现立场不一致的情况。然而,这两个概念经常被交替使用。在对最近 30 项随机对照试验的功率计算进行的审计中,4 项研究使用了最小可接受差异,9 项研究使用了预期差异。其他研究则没有明确说明。在事后分析的情况下,这种方法可以揭示开展进一步研究的价值,而这在标准功率计算中并不明显。比较了超优、非劣效、等效和标准优效研究对替代假设的接受和拒绝情况。当应用固定的最小可接受差异时,研究结果将处于同时应用这些假设的七个逻辑位置之一。试验规模扩大的趋势和非劣效性研究的镜像方法意味着,较新的干预措施可能会变得不那么有效。增强优效性研究可以解决这一问题,并确保在试验前对临床意义进行评估,这是确认干预措施是否有益的必要条件。
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Adapting power calculations to include a superiority margin: what are the implications?

This paper examines the application of super-superiority margins in study power calculations. Unlike traditional power calculations, which primarily aim to reject the null hypothesis by any margin, a super-superiority margin establishes a clinically significant threshold. Despite potential benefits, this approach, akin to a non-inferiority calculation but in an opposing direction, is rarely used. Implementing a super-superiority margin separates the notion of the likely difference between two groups (the effect size) from the minimum clinically significant difference, without which inconsistent positions could be held. However, these are often used interchangeably. In an audit of 30 recent randomized controlled trial power calculations, four studies utilized the minimal acceptable difference, and nine utilized the expected difference. In the other studies, this was unclarified. In the post hoc scenario, this approach can shed light on the value of undertaking further studies, which is not apparent from the standard power calculation. The acceptance and rejection of the alternate hypothesis for super-superiority, non-inferiority, equivalence, and standard superiority studies have been compared. When a fixed minimal acceptable difference is applied, a study result will be in one of seven logical positions with regards to the simultaneous application of these hypotheses. The trend for increased trial size and the mirror approach of non-inferiority studies implies that newer interventions may be becoming less effective. Powering for superiority could counter this and ensure that a pre-trial evaluation of clinical significance has taken place, which is necessary to confirm that interventions are beneficial.

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