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将随机临床试验的推断扩展至目标人群:可移植性方法的范围综述

Extending Inferences from Randomized Clinical Trials to Target Populations: A Scoping Review of Transportability Methods

Guanbo Wang, Ting-Wei Ernie Liao, David Furfaro, Leo Anthony Celi, Kevin Sheng-Kai Ma

AI总结:

本文通过范围综述评估将RCT结果外推至真实世界目标人群的可移植性方法,纳入15项研究,发现这些方法能有效连接试验与真实世界照护,为循证医学提供支持。

摘要:

目的:随机对照试验(RCT)的结果常常为临床决策提供依据,但试验中经过高度筛选的人群以及试验期间所提供的照护往往不能反映真实世界的实践。本范围综述的目的是识别将RCT的研究结果外推至目标人群的方法能力。研究设计:对关注RCT结果向观察性队列可移植性的文献进行了范围综述。每项研究均根据其用于可移植性的方法以及在观察性数据中估计目标人群治疗效应的程度进行评估。结果:共纳入15篇已发表论文。研究主题涵盖心血管疾病、传染病、精神病学、肿瘤学、骨科、麻醉学和血液学。这些研究表明,RCT的研究结果可以转化至真实世界环境,但效应量和精确度存在不同程度的变化。在某些情况下,为目标人群估计的治疗效应与RCT中的治疗效应在统计学上存在显著差异。结论:尽管RCT与真实世界研究之间的效应大小存在差异,可移植性方法在有效连接RCT与真实世界照护服务方面发挥着重要作用,为循证医学提供了有价值的见解。

英文摘要:

Objective: Randomized controlled trial (RCT) results often inform clinical decision-making, but the highly curated populations of trials and the care provided during the trial are often not reflective of real-world practice. The objective of this scoping review is to identify the ability of methods to transport findings from RCTs to target populations. Study design: A scoping review was conducted on the literature focusing on the transportability of the results from RCTs to observational cohorts. Each study was assessed based on the methodology used for transportability and the extent to which the treatment effect from the RCT was estimated in the target population in observational data. Results: A total of 15 published papers were included. The research topics include cardiovascular diseases, infectious diseases, psychiatry, oncology, orthopedics, anesthesiology, and hematology. These studies show that the findings from RCTs could be translated to real-world settings, with varying degrees of effect size and precision. In some cases, the estimated treatment effect for the target population were statistically significantly different from those in RCTs. Conclusion: Despite variations in the magnitude of effects between RCTs and real-world studies, transportability methods play an important role in effectively bridging the RCTs and real-world care delivery, offering valuable insights for evidence-based medicine.

机构、总结、中文摘要均由AI分析而来,可能存在错误,请以论文正文为准。

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