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Estimation and inference for semi-competing risks based on data from a nested case-control study.

ABSTRACT: In semi-competing risks, the occurrence of some non-terminal event is subject to a terminal event, usually death. While existing methods for semi-competing risks data analysis assume complete information on all relevant covariates, data on at least one covariate are often not readily available in practice. In this setting, for standard univariate time-to-event analyses, researchers may choose from several strategies for sub-sampling patients on whom to collect complete data, including the nested case-control study design. Here, we consider a semi-competing risks analysis through the reuse of data from an existing nested case-control study for which risk sets were formed based on either the non-terminal or the terminal event. Additionally, we introduce the supplemented nested case-control design in which detailed data are collected on additional events of the other type. We propose estimation with respect to a frailty illness-death model through maximum weighted likelihood, specifying the baseline hazard functions either parametrically or semi-parametrically via B-splines. Two standard error estimators are proposed: (i) a computationally simple sandwich estimator and (ii) an estimator based on a perturbation resampling procedure. We derive the asymptotic properties of the proposed methods and evaluate their small-sample properties via simulation. The designs/methods are illustrated with an investigation of risk factors for acute graft-versus-host disease among N?=?8838 patients undergoing hematopoietic stem cell transplantation, for which death is a significant competing risk.

SUBMITTER: Jazic I

PROVIDER: S-EPMC7916743 | biostudies-literature | 2020 Nov

REPOSITORIES: biostudies-literature

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Estimation and inference for semi-competing risks based on data from a nested case-control study.

Jazić Ina I Lee Stephanie S Haneuse Sebastien S

Statistical methods in medical research 20200617 11

In semi-competing risks, the occurrence of some non-terminal event is subject to a terminal event, usually death. While existing methods for semi-competing risks data analysis assume complete information on all relevant covariates, data on at least one covariate are often not readily available in practice. In this setting, for standard univariate time-to-event analyses, researchers may choose from several strategies for sub-sampling patients on whom to collect complete data, including the nested ...[more]

PMID: 32552435

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Project description:In the analysis of semi-competing risks data interest lies in estimation and inference with respect to a so-called non-terminal event, the observation of which is subject to a terminal event. Multi-state models are commonly used to analyse such data, with covariate effects on the transition/intensity functions typically specified via the Cox model and dependence between the non-terminal and terminal events specified, in part, by a unit-specific shared frailty term. To ensure identifiability, the frailties are typically assumed to arise from a parametric distribution, specifically a Gamma distribution with mean 1.0 and variance, say, σ2. When the frailty distribution is misspecified, however, the resulting estimator is not guaranteed to be consistent, with the extent of asymptotic bias depending on the discrepancy between the assumed and true frailty distributions. In this paper, we propose a novel class of transformation models for semi-competing risks analysis that permit the non-parametric specification of the frailty distribution. To ensure identifiability, the class restricts to parametric specifications of the transformation and the error distribution; the latter are flexible, however, and cover a broad range of possible specifications. We also derive the semi-parametric efficient score under the complete data setting and propose a non-parametric score imputation method to handle right censoring; consistency and asymptotic normality of the resulting estimators is derived and small-sample operating characteristics evaluated via simulation. Although the proposed semi-parametric transformation model and non-parametric score imputation method are motivated by the analysis of semi-competing risks data, they are broadly applicable to any analysis of multivariate time-to-event outcomes in which a unit-specific shared frailty is used to account for correlation. Finally, the proposed model and estimation procedures are applied to a study of hospital readmission among patients diagnosed with pancreatic cancer.

Dataset Information

Estimation and inference for semi-competing risks based on data from a nested case-control study.

Publications

Estimation and inference for semi-competing risks based on data from a nested case-control study.

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