Two-stage penalized regression screening to detect biomarker-treatment interactions in randomized clinical trials

Biometrics. 2022 Mar;78(1):141-150. doi: 10.1111/biom.13424. Epub 2021 Jan 29.

Abstract

High-dimensional biomarkers such as genomics are increasingly being measured in randomized clinical trials. Consequently, there is a growing interest in developing methods that improve the power to detect biomarker-treatment interactions. We adapt recently proposed two-stage interaction detecting procedures in the setting of randomized clinical trials. We also propose a new stage 1 multivariate screening strategy using ridge regression to account for correlations among biomarkers. For this multivariate screening, we prove the asymptotic between-stage independence, required for familywise error rate control, under biomarker-treatment independence. Simulation results show that in various scenarios, the ridge regression screening procedure can provide substantially greater power than the traditional one-biomarker-at-a-time screening procedure in highly correlated data. We also exemplify our approach in two real clinical trial data applications.

Keywords: biomarker; clinical trial; interaction; randomization; ridge regression; screening.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Biomarkers
  • Computer Simulation
  • Genomics*
  • Randomized Controlled Trials as Topic

Substances

  • Biomarkers