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About this Publication
Title
Semiparametric isotonic regression analysis for risk assessment under nested case-control and case-cohort designs.
Pubmed ID
31868119 (View this publication on the PubMed website)
Digital Object Identifier
Publication
Stat Methods Med Res. 2020 Aug; Volume 29 (Issue 8): Pages 2328-2343
Authors
Li W, Li R, Feng Z, Ning J
Affiliations
  • Department of Biostatistics and Data Science, The University of Texas Health Science Center, Houston, TX, USA.
  • Fred Hutchinson Cancer Research Center, Seattle, Washington, DC, USA.
  • Department of Biostatistics, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
Abstract

Two-phase sampling designs, including nested case-control and case-cohort designs, are frequently utilized in large cohort studies involving expensive biomarkers. To analyze data from two-phase designs with a binary outcome, parametric models such as logistic regression are often adopted. However, when the model assumptions are not valid, parametric models may lead to biased estimation and risk evaluation. In this paper, we propose a robust semiparametric regression model for binary outcomes and an easy-to-implement computational procedure that combines the pool-adjacent violators algorithm with inverse probability weighting. The asymptotic properties are established, including consistency and the convergence rate. Simulation studies show that the proposed method performs well and is more robust than logistic regression methods. We demonstrate the application of the proposed method to real data from the Prostate, Lung, Colorectal, and Ovarian (PLCO) Cancer Screening Trial.

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