Semiparametric inference for a two-stage outcome-dependent sampling design with interval-censored failure time data.

TitleSemiparametric inference for a two-stage outcome-dependent sampling design with interval-censored failure time data.
Publication TypeJournal Article
Year of Publication2020
AuthorsZhou, Qingning, Jianwen Cai, and Haibo Zhou
JournalLifetime Data Anal
Volume26
Issue1
Pagination85-108
Date Published2020 01
ISSN1572-9249
KeywordsBias, Computer Simulation, Data Interpretation, Statistical, Humans, Likelihood Functions, Regression Analysis, Time
Abstract

We propose a two-stage outcome-dependent sampling design and inference procedure for studies that concern interval-censored failure time outcomes. This design enhances the study efficiency by allowing the selection probabilities of the second-stage sample, for which the expensive exposure variable is ascertained, to depend on the first-stage observed interval-censored failure time outcomes. In particular, the second-stage sample is enriched by selectively including subjects who are known or observed to experience the failure at an early or late time. We develop a sieve semiparametric maximum pseudo likelihood procedure that makes use of all available data from the proposed two-stage design. The resulting regression parameter estimator is shown to be consistent and asymptotically normal, and a consistent estimator for its asymptotic variance is derived. Simulation results demonstrate that the proposed design and inference procedure performs well in practical situations and is more efficient than the existing designs and methods. An application to a phase 3 HIV vaccine trial is provided.

DOI10.1007/s10985-019-09461-5
Alternate JournalLifetime Data Anal
Original PublicationSemiparametric inference for a two-stage outcome-dependent sampling design with interval-censored failure time data.
PubMed ID30617753
PubMed Central IDPMC6612481
Grant ListP30 ES010126 / ES / NIEHS NIH HHS / United States
P01 CA142538 / CA / NCI NIH HHS / United States
R01 ES021900 / ES / NIEHS NIH HHS / United States
Project: