Use of propensity score and disease risk score for multiple treatments with time-to-event outcome: a simulation study.

JOURNAL OF BIOPHARMACEUTICAL STATISTICS(2019)

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摘要
Propensity score (PS) and disease risk score (DRS) are often used in pharmacoepidemiologic safety studies. Methods of applying these two balancing scores are extensively studied in binary treatment settings. However, the use of PS and DRS is not well understood in the case of non-ordinal multiple treatments. Some PS methods of multiple treatments have been implemented since the theoretical establishment. Nevertheless, most of the work applies to continuous or binary outcomes. Little work has been done for time-to-event outcomes. In this study, we extend the application of the PS and DRS methods to time-to-event outcomes in multiple treatment settings. The analytical approaches include weighing, matching, stratification, and regression. Simulation studies with rare event rates are conducted to evaluate the performances of different methods. Different treatment-covariates and outcome-covariates strength of associations are considered. Additionally, the impacts of imbalanced designs and large or limited PS overlaps are investigated on various analytical approaches. We found that the inverse probability treatment weighting with bootstrap variance estimator, the generalized PS matching, and the Cox regression estimated DRS in full cohort generally performed well in multiple treatment settings. This study aims to provide additional guidance for researchers on PS and DRS analyses in pharmacoepidemiologic observational studies.
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关键词
Multinomial logit model,inverse probability,treatment weight,matching,bootstrap,observational study
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