Semiparametric recurrent event vs time-to-first-event analyses in randomized trials: Estimands and model misspecification
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Date
2021-04-20
Authors
Zhong, Yujie
Cook, Richard
Advisor
Journal Title
Journal ISSN
Volume Title
Publisher
John Wiley & Sons Ltd.
Abstract
Insights regarding the merits of recurrent event and time-to-first-event analyses are needed to provide guidance on strategies for analyzing intervention effects in randomized trials involving recurrent event responses. Using established asymptotic results we introduce a framework for studying the large sample properties of estimators arising from semiparametric proportional rate function models and Cox regression undermodel misspecification. The asymptotic biases and power implications are investigated for different data generating models, and we study the impact of dependent censoring on these findings. Illustrative applications are given involving data from a cystic fibrosis trial and a carcinogenicity experiment, following which we summarize findings and discuss implications for clinical trial design.
Description
This is the peer reviewed version of the following article: “Zhong Y and Cook RJ (2021), Semiparametric recurrent event vs time-to-first-event analyses in randomized trials: Estimands and model misspecification, Statistics in Medicine, 40 (16): 3823–3842” which has been published in final form at https://doi.org/10.1002/sim.9002.
Keywords
Cox regression, estimands, multiplicative rate functions, power, recurrent events, robustness