目标试验模拟:运用随机试验原则提高炎症性肠病观察性研究的质量
Target Trial Emulation: Improving the Quality of Observational Studies in Inflammatory Bowel Disease Using the Principles of Randomized Trials.
作者信息
Honap Sailish, Danese Silvio, Peyrin-Biroulet Laurent
机构信息
INFINY Institute, Nancy University Hospital, F-54500 Vandœuvre-lès-Nancy, France.
School of Immunology and Microbial Sciences, King's College London, London, UK.
出版信息
Inflamm Bowel Dis. 2025 Mar 3;31(3):843-849. doi: 10.1093/ibd/izae131.
The past decade has seen a substantial increase in the number of randomized controlled trials (RCTs) conducted in inflammatory bowel disease (IBD). Randomized controlled trials are the gold standard method for generating robust evidence of drug safety and efficacy but are expensive, time-consuming, and may have ethical implications. Observational studies in IBD are often used to fill the gaps in evidence but are typically hindered by significant bias. There are several approaches for making statistical inferences from observational data with some that focus on study design and others on statistical techniques. Target trial emulation is an emerging methodological process that aims to bridge this gap and improve the quality of observational studies by applying the principles of an ideal, or "target," randomized trial to routinely collected clinical data. There has been a rapid expansion of observational studies that have emulated trials over the past 5 years in other medical fields, but this has yet to be adopted in gastroenterology and IBD. The wealth of nonrandomized clinical data available through electronic health records, patient registries, and administrative health databases afford innumerable hypothesis-generating opportunities for IBD research. This review outlines the principles of target trial emulation, discusses the merits to IBD observational studies in reducing the most common biases and improving confidence in causality, and details the caveats of using this approach.
在过去十年中,炎症性肠病(IBD)领域进行的随机对照试验(RCT)数量大幅增加。随机对照试验是获取药物安全性和有效性可靠证据的金标准方法,但成本高昂、耗时且可能存在伦理问题。IBD的观察性研究常被用于填补证据空白,但通常受到显著偏差的阻碍。从观察性数据进行统计推断有多种方法,有些侧重于研究设计,有些侧重于统计技术。目标试验模拟是一种新兴的方法学过程,旨在通过将理想的或“目标”随机试验的原则应用于常规收集的临床数据来弥合这一差距,并提高观察性研究的质量。在过去5年中,其他医学领域中模拟试验的观察性研究迅速增加,但胃肠病学和IBD领域尚未采用这种方法。通过电子健康记录、患者登记册和行政健康数据库可获得的大量非随机临床数据为IBD研究提供了无数产生假设的机会。本综述概述了目标试验模拟的原则,讨论了其对IBD观察性研究在减少最常见偏差和提高因果关系可信度方面的优点,并详细说明了使用这种方法的注意事项。