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In cancer research, leveraging patient-derived xenografts (PDXs) in pre-clinical experiments is a crucial approach for assessing innovative therapeutic strategies. Addressing the inherent variability in treatment response among and within individual PDX lines is essential. However, the current literature lacks a user-friendly statistical power analysis tool capable of concurrently determining the required number of PDX lines and animals per line per treatment group in this context. In this paper, we present a simulation-based R package for sample size determination, named `PDXpower', which is publicly available at The Comprehensive R Archive Network https: //CRAN. R-project. org/package=PDXpower. The package is designed to estimate the necessary number of both PDX lines and animals per line per treatment group for the design of a PDX experiment, whether for an uncensored outcome, or a censored time-to-event outcome. Our sample size considerations rely on two widely used analytical frameworks: the mixed effects ANOVA model for uncensored outcomes and Cox's frailty model for censored data outcomes, which effectively account for both inter-PDX variability and intra-PDX correlation in treatment response. Step-by-step illustrations for utilizing the developed package are provided, catering to scenarios with or without preliminary data.
Li et al. (Sat,) studied this question.