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Marginal structural models and other analyses allow multiple estimates of treatment effects in randomized clinical trials: Meta-epidemiological analysis

Ewald, Hannah and Speich, Benjamin and Ladanie, Aviv and Bucher, Heiner C. and Ioannidis, John P. A. and Hemkens, Lars G.. (2019) Marginal structural models and other analyses allow multiple estimates of treatment effects in randomized clinical trials: Meta-epidemiological analysis. Journal of clinical epidemiology, 107. pp. 12-26.

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Official URL: https://edoc.unibas.ch/69819/

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Abstract

To determine how marginal structural models (MSMs), which are increasingly used to estimate causal effects, are used in randomized clinical trials (RCTs) and compare their results with those from intention-to-treat (ITT) or other analyses.; We searched PubMed, Scopus, citations of key references, and Clinicaltrials.gov. Eligible RCTs reported clinical effects based on MSMs and at least one other analysis.; We included 12 RCTs reporting 138 analyses for 24 clinical questions. In 19/24 (79%), MSM-based and other effect estimates were all in the same direction, 22/22 had overlapping 95% confidence intervals (CIs), and in 19/22 (86%), the MSM effect estimate lay within all 95% CIs of all other effects (in two cases no CIs were reported). For the same clinical question, the largest effect estimate from any analysis was 1.19-fold (median; interquartile range 1.13-1.34) larger than the smallest. All MSM and ITT effect estimates were in the same direction and had overlapping 95% CIs. In 71% (12/17), they also agreed on the presence of statistical significance. MSM-based effect estimates deviated more from the null than those based on ITT (P = 0.18). The effect estimates of both approaches differed 1.12-fold (median; interquartile range 1.02-1.22).; MSMs provided largely similar effect estimates as other available analyses. Nevertheless, some of the differences in effect estimates or statistical significance may become important in clinical decision-making, and the multiple estimates require utmost attention of possible selective reporting bias.
Faculties and Departments:09 Associated Institutions > Swiss Tropical and Public Health Institute (Swiss TPH)
09 Associated Institutions > Swiss Tropical and Public Health Institute (Swiss TPH) > Department of Epidemiology and Public Health (EPH) > Environmental Exposures and Health > Physical Hazards and Health (Röösli)
UniBasel Contributors:Ladanie, Aviv
Item Type:Article, refereed
Article Subtype:Research Article
Publisher:Pergamon Press
ISSN:0895-4356
Note:Publication type according to Uni Basel Research Database: Journal article
Identification Number:
Last Modified:18 Mar 2019 13:01
Deposited On:18 Mar 2019 13:01

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