Semiparametric regression analysis of case-cohort studies with multiple interval-censored disease outcomes.

Pubmed ID: 33783001

Pubmed Central ID: PMC8691208

Journal: Statistics in medicine

Publication Date: June 15, 2021

MeSH Terms: Humans, Cohort Studies, Proportional Hazards Models, Regression Analysis, Computer Simulation, Likelihood Functions

Grants: P01 CA142538, P01CA142538, P30 ES010126, P42ES031007, P30ES010126, P42 ES031007

Authors: Zhou Q, Zhou H, Cai J

Cite As: Zhou Q, Cai J, Zhou H. Semiparametric regression analysis of case-cohort studies with multiple interval-censored disease outcomes. Stat Med 2021 Jun 15;40(13):3106-3123. Epub 2021 Mar 29.

Studies:

Abstract

Interval-censored failure time data commonly arise in epidemiological and biomedical studies where the occurrence of an event or a disease is determined via periodic examinations. Subject to interval-censoring, available information on the failure time can be quite limited. Cost-effective sampling designs are desirable to enhance the study power, especially when the disease rate is low and the covariates are expensive to obtain. In this work, we formulate the case-cohort design with multiple interval-censored disease outcomes and also generalize it to nonrare diseases where only a portion of diseased subjects are sampled. We develop a marginal sieve weighted likelihood approach, which assumes that the failure times marginally follow the proportional hazards model. We consider two types of weights to account for the sampling bias, and adopt a sieve method with Bernstein polynomials to handle the unknown baseline functions. We employ a weighted bootstrap procedure to obtain a variance estimate that is robust to the dependence structure between failure times. The proposed method is examined via simulation studies and illustrated with a dataset on incident diabetes and hypertension from the Atherosclerosis Risk in Communities study.