Comparing effect estimates in randomized trials and observational studies from the same population: an application to percutaneous coronary intervention

medRxiv(2021)

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摘要
Background The ability for real world data to deliver similar results as a trial that asks the same question about the risks or benefits of a clinical intervention can be restricted not only by lack of randomization, but also limited information on eligibility criteria and outcomes. To understand when results from observational studies and randomized trials are comparable, we carried out an observational emulation of a target trial designed to ask similar questions as the VALIDATE randomized trial. VALIDATE compared the effect of bivalirudin and heparin during percutaneous coronary intervention on the risk of death, myocardial infarction, and bleeding across Sweden. Methods We specified the protocol of a target trial similar to the VALIDATE trial protocol, then emulated the target trial in the period before the trial took place using data from the SWEDEHEART registry; the same registry in which the trial was undertaken. Results The target trial emulation and the VALIDATE trial both estimated no difference in the effect of bivalirudin and heparin on the risk of death or myocardial infarction by 180 days: emulation risk ratio for death 1.21 (0.88, 1.54); VALIDATE hazard ratio for death 1.05 (0.78, 1.41). The observational data, however, could not capture less severe cases of bleeding, resulting in an inability to define a bleeding outcome like the trial, and could not account for intractable confounding early in follow-up (risk ratio for death by 14 days 1.85 (0.95, 3.63)). Conclusion Using real world data to emulate a target trial can deliver accurate long-term effect estimates. Yet, even with rich observational data, it is not always possible to estimate the short-term effect of interventions, or the effect on outcomes for which data are not routinely collected. If registries included information on reasons for treatment decisions, researchers may be better positioned to identify important confounders. ### Competing Interest Statement Dr. Matthews, Dr. Szummer, Dr. Lindahl, and Dr. Jernberg have nothing to disclose; Dr. Dahabreh reports grants from PCORI, during the conduct of the study; Dr. Erlinge reports personal fees from AstraZeneca, outside the submitted work; Dr. Feychting, Dr. Berglund, and Dr. Hernan report grants from Swedish Research Council, during the conduct of the study. ### Funding Statement This work was supported by grant from the Swedish Research Council (registration number 2018-03028). Issa J Dahabreh is supported by the Patient-Centered Outcomes Research Institute (PCORI, award ME-1502-27794) ### Author Declarations I confirm all relevant ethical guidelines have been followed, and any necessary IRB and/or ethics committee approvals have been obtained. Yes The details of the IRB/oversight body that provided approval or exemption for the research described are given below: Ethical approval was gained from the Swedish Ethical Review Authority All necessary patient/participant consent has been obtained and the appropriate institutional forms have been archived. Yes I understand that all clinical trials and any other prospective interventional studies must be registered with an ICMJE-approved registry, such as ClinicalTrials.gov. I confirm that any such study reported in the manuscript has been registered and the trial registration ID is provided (note: if posting a prospective study registered retrospectively, please provide a statement in the trial ID field explaining why the study was not registered in advance). Yes I have followed all appropriate research reporting guidelines and uploaded the relevant EQUATOR Network research reporting checklist(s) and other pertinent material as supplementary files, if applicable. Yes Pseudonymized personal data were obtained from national Swedish Registry holders after ethical approval and secrecy assessment. According to Swedish laws and regulations, personal sensitive data can only be made available for researchers who fulfil legal requirements for access to personal sensitive data.
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关键词
percutaneous coronary intervention,effect estimates,trials,observational studies
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