论文标题
随机对照试验中顺序结果的统计分析:范围审查的协议
Statistical analyses of ordinal outcomes in randomised controlled trials: protocol for a scoping review
论文作者
论文摘要
随机对照试验旨在评估相对于其他标准干预措施的一种(或更多)健康干预措施的影响。 RCT有时会使用顺序结果,这是一个由多个单调有序类别组成的终点,不一定会被可量化的距离隔开。在临床环境中,序列结果具有吸引力,因为疾病状态可以代表可能具有临床重要性的有意义的类别。与二分法结果相比,它们还可以保留信息并增加统计能力。 RCT中有序结果的目标参数可能会因研究问题的性质,建模假设和数据分析师的专业知识而有所不同。这项范围审查的目的是系统地描述当代RCT中序数结果的使用。具体而言,我们的目标是(i)确定哪些目标参数在使用序数结果的试验中具有感兴趣; (ii)描述如何在RCT中分析序数结局以估计治疗效果; (iii)描述使用顺序结局的RCT是否充分报告了分析结果分析的关键方法论方面。 本综述的结果将概述RCT中使用顺序结果的当前实践状态。将讨论改善RCT中顺序结果的分析和报告的方法。我们将在2012年1月1日至2022年7月31日之间发表在前四个医学期刊(BMJ,NEJM,柳叶刀和JAMA)中发表的RCT。该评论将使用PubMed进行。我们的评论将遵守Prisma-SCR清单中所述的范围审查指南。研究特征和设计,包括目标参数和统计方法,将从合格的研究中提取。数据将使用描述性统计数据汇总。
Randomised controlled trials aim to assess the impact of one (or more) health interventions relative to other standard interventions. RCTs sometimes use an ordinal outcome, which is an endpoint that comprises of multiple, monotonically ordered categories that are not necessarily separated by a quantifiable distance. Ordinal outcomes are appealing in clinical settings as disease states can represent meaningful categories that may be of clinical importance. They can also retain information and increase statistical power compared to dichotomised outcomes. Target parameters for ordinal outcomes in RCTs may vary depending on the nature of the research question, the modelling assumptions, and the expertise of the data analyst. The aim of this scoping review is to systematically describe the use of ordinal outcomes in contemporary RCTs. Specifically, we aim to (i) identify which target parameters are of interest in trials that use an ordinal outcome; (ii) describe how ordinal outcomes are analysed in RCTs to estimate a treatment effect; and (iii) describe whether RCTs that use an ordinal outcome adequately report key methodological aspects specific to the analysis of the outcome. Results from this review will outline the current state of practice of the use of ordinal outcomes in RCTs. Ways to improve the analysis and reporting of ordinal outcomes in RCTs will be discussed. We will review RCTs that are published in the top four medical journals (BMJ, NEJM, The Lancet and JAMA) between 1 January 2012 and 31 July 2022 that use an ordinal outcome. The review will be conducted using PubMed. Our review will adhere to guidelines for scoping reviews as described in the PRISMA-ScR checklist. The study characteristics and design, including the target parameter(s) and statistical methods will be extracted from eligible studies. The data will be summarised using descriptive statistics.