Article type
Abstract
Background: One approach frequently adopted by RCT is the use of composite outcomes, a type of outcome that combines two or more components to increase the computation of events within a trial. Three key criteria have been proposed to help in deciding whether a composite outcome is adequate for decision-making: (i) components with balanced importance to patients; (ii) components with similar frequency of occurrence; and (iii) components with the same susceptibility to treatment effect.
Objective: to estimate the frequency and critically appraise the use and reporting of composite outcomes in RCT on pharmacological interventions for coronary artery disease (CAD).
Methods: meta-research considering all articles from MEDLINE, published from 1st January 2020, to December 31, 2021, reporting results of clinical primary outcomes from RCT which assessed pharmacological interventions, used alone or in combination, for the treatment or secondary prevention of CAD.
Results: from the 34 included RCTs, 28 (82.35%) had a primary composite outcome and 13 unique composite primary outcomes were used. The most frequent was ""cardiovascular death, myocardial infarction, stroke"" (12/28, 42.86%). The term MACE (major adverse cardiac event) was used for five distinct composite primary outcomes. A combination of 12 different components resulted in the 28 primary composite outcomes, with stroke being the most frequent component present in 96.43% (27/28) of the primary composite outcomes. 60.71% (17/28) reported the estimates for each individual component and none discussed potential limitations and/or related advantages of the composite outcomes.
Conclusions: In RCT on pharmacological interventions for CAD, composite outcomes are frequently used, but the definition of its components is very heterogeneous. The estimate for individual components is often not fully reported, which prevents a complete analysis of their adequacy for clinical practice. The term MACE was used inconsistently to refer to different sets of components, which can also be misleading and confusing.
Relevance to patients: to properly support health care, we need RCT with trustworthy results, so they need to address clinically relevant outcomes. The choice for a composite outcome should be conscious, consider the criteria recommended by the literature and discuss the limitations inherent to this approach.
Objective: to estimate the frequency and critically appraise the use and reporting of composite outcomes in RCT on pharmacological interventions for coronary artery disease (CAD).
Methods: meta-research considering all articles from MEDLINE, published from 1st January 2020, to December 31, 2021, reporting results of clinical primary outcomes from RCT which assessed pharmacological interventions, used alone or in combination, for the treatment or secondary prevention of CAD.
Results: from the 34 included RCTs, 28 (82.35%) had a primary composite outcome and 13 unique composite primary outcomes were used. The most frequent was ""cardiovascular death, myocardial infarction, stroke"" (12/28, 42.86%). The term MACE (major adverse cardiac event) was used for five distinct composite primary outcomes. A combination of 12 different components resulted in the 28 primary composite outcomes, with stroke being the most frequent component present in 96.43% (27/28) of the primary composite outcomes. 60.71% (17/28) reported the estimates for each individual component and none discussed potential limitations and/or related advantages of the composite outcomes.
Conclusions: In RCT on pharmacological interventions for CAD, composite outcomes are frequently used, but the definition of its components is very heterogeneous. The estimate for individual components is often not fully reported, which prevents a complete analysis of their adequacy for clinical practice. The term MACE was used inconsistently to refer to different sets of components, which can also be misleading and confusing.
Relevance to patients: to properly support health care, we need RCT with trustworthy results, so they need to address clinically relevant outcomes. The choice for a composite outcome should be conscious, consider the criteria recommended by the literature and discuss the limitations inherent to this approach.