首页|基于真实世界数据的观察性研究因果推断——目标试验模拟实施要点及案例分析

基于真实世界数据的观察性研究因果推断——目标试验模拟实施要点及案例分析

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随机对照试验(randomized controlled trial,RCT)是评估干预与结局指标因果效应的"金标准".然而,由于研究成本高昂和伦理限制,在临床实际中,尤其是在外科领域中开展RCT面临受试者招募困难、盲法实施困难和干预标准化困难等诸多挑战.在此情况下,基于目标试验模拟(target trial emulation,TTE)框架、利用真实世界数据按照RCT研究设计原则进行因果推断,有助于识别并减少传统观察性研究因设计缺陷产生的永恒时间偏倚、混杂偏倚、选择偏倚或碰撞偏倚,以得到接近RCT的高质量证据,提高真实世界数据研究的临床指导价值.但TTE存在无法完全消除混杂、源数据质量要求高和暂缺报告规范等局限,故研究者应充分认识以免做出错误的因果推断.本文拟就TTE的框架简介、实施要点、应用范围、应用案例及框架优缺点进行概述.
Causal inference in observational studies based on real-world data:Key points and case studies for target trial emulation
Randomized controlled trial(RCT)are considered the"gold standard"for evaluating the causal effects of interventions on outcome measures.However,due to high research costs and ethical constraints,conducting RCT in clinical practice,especially in the surgical field,faces numerous challenges such as difficulties in subject recruitment,implementation of blinding,and standardization of interventions.In such cases,using real-world data to perform causal inference under the framework of target trial emulation(TTE),based on the principles of RCT design,helps to identify and reduce biases arising from design flaws in traditional observational studies,such as immortal time bias,confounding,selection bias,or collider bias.This approach can produce high-quality evidence comparable to that of RCT,thereby enhancing the clinical guidance value of real-world data studies.However,TTE has limitations,such as the inability to completely eliminate confounding,high quality requirements for source data,and the current lack of reporting standards.Therefore,researchers should be fully aware of these limitations to avoid making incorrect causal inferences.This article intends to provide an overview of the TTE framework,implementation points,application scope,application cases,and advantages and disadvantages of the framework.

Target trial emulationreal-world datacausal inferenceobservational study

袁驰、周祎灵、曹雨滋、张豪杰、王依倩、李舍予

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四川大学华西医院小儿外科(成都 610041)

四川大学华西医院内分泌代谢科(成都 610041)

四川大学华西医院中国循证医学中心MAGIC中心(成都 610041)

格罗宁根大学流行病学系(格罗宁根荷兰)

麦吉尔大学数学与统计系(蒙特利尔加拿大)

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目标试验模拟 真实世界数据 因果推断 观察性研究

2024

中国胸心血管外科临床杂志
四川大学华西医院

中国胸心血管外科临床杂志

CSTPCD北大核心
影响因子:0.846
ISSN:1007-4848
年,卷(期):2024.31(12)