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综述文章:面向临床研究人员的急诊医学创新试验设计入门指南

Review article: Primer for clinical researchers on innovative trial designs for emergency medicine.

作者信息

Lee Katherine J, Middleton Melissa, Mahar Robert K

机构信息

Clinical Epidemiology and Biostatistics Unit, Murdoch Children's Research Institute, Melbourne, Victoria, Australia.

Department of Paediatrics, University of Melbourne, Melbourne, Victoria, Australia.

出版信息

Emerg Med Australas. 2025 Feb;37(1):e14532. doi: 10.1111/1742-6723.14532. Epub 2024 Nov 18.

Abstract

Randomised trials have long been recognised as the gold standard research tool for evidence-based medicine. The past decade has seen the emergence of several innovative trial designs that are revolutionising how trials are conducted. These innovative designs enable more efficient, pragmatic trials that can address complex research questions which were previously not possible. In this paper, we provide an overview of the key innovative designs that are likely to be useful in the emergency medicine context, namely cluster crossover and stepped wedge designs, sequential multiple assignment randomised trial (SMART) designs, and platform trials. We describe the main features of each design, outline their pros and cons, and describe when they may or may not be useful. We also provide examples of these innovative designs in contexts that are relevant to emergency medicine.

摘要

长期以来,随机试验一直被视为循证医学的金标准研究工具。在过去十年中,出现了几种创新的试验设计,正在彻底改变试验的进行方式。这些创新设计能够开展更高效、务实的试验,从而解决以前无法解决的复杂研究问题。在本文中,我们概述了可能在急诊医学背景下有用的关键创新设计,即整群交叉和阶梯楔形设计、序贯多重分配随机试验(SMART)设计以及平台试验。我们描述了每种设计的主要特点,概述了它们的优缺点,并说明了它们何时可能有用或无用。我们还提供了这些创新设计在与急诊医学相关背景下的实例。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/93b0/11744427/d0926fc24e58/EMM-37-0-g002.jpg

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