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Title: Design and analysis of nested case–control studies for recurrent events subject to a terminal event

Journal Article · · Statistics in Medicine
DOI: https://doi.org/10.1002/sim.8302 · OSTI ID:1532770
ORCiD logo [1];  [1];  [2];  [3];  [4]
  1. Department of Biostatistics Harvard T.H. Chan School of Public Health Boston Massachusetts
  2. Department of Statistics Radiation Effects Research Foundation Hiroshima Japan
  3. Department of Biopathology Institut Bergonié Bordeaux France
  4. Centre de recherche INSERM U1219 Université de Bordeaux–ISPED Bordeaux France

The process by which patients experience a series of recurrent events, such as hospitalizations, may be subject to death. In cohort studies, one strategy for analyzing such data is to fit a joint frailty model for the intensities of the recurrent event and death, which estimates covariate effects on the two event types while accounting for their dependence. When certain covariates are difficult to obtain, however, researchers may only have the resources to subsample patients on whom to collect complete data: one way is using the nested case–control (NCC) design, in which risk set sampling is performed based on a single outcome. We develop a general framework for the design of NCC studies in the presence of recurrent and terminal events and propose estimation and inference for a joint frailty model for recurrence and death using data arising from such studies. We propose a maximum weighted penalized likelihood approach using flexible spline models for the baseline intensity functions. Two standard error estimators are proposed: a sandwich estimator and a perturbation resampling procedure. We investigate operating characteristics of our estimators as well as design considerations via a simulation study and illustrate our methods using two studies: one on recurrent cardiac hospitalizations in patients with heart failure and the other on local recurrence and metastasis in patients with breast cancer.

Sponsoring Organization:
USDOE
Grant/Contract Number:
DE‐HS0000031
OSTI ID:
1532770
Journal Information:
Statistics in Medicine, Journal Name: Statistics in Medicine Vol. 38 Journal Issue: 22; ISSN 0277-6715
Publisher:
Wiley Blackwell (John Wiley & Sons)Copyright Statement
Country of Publication:
United Kingdom
Language:
English

References (36)

An Algorithm for Least-Squares Estimation of Nonlinear Parameters journal June 1963
Marginal Mark Regression Analysis of Recurrent Marked Point Process Data journal June 2008
Sampling strategies in nested case-control studies. journal November 1994
Heart Failure With Recovered Ejection Fraction: Clinical Description, Biomarkers, and Outcomes journal June 2014
Regression Analysis for Recurrent Events Data under Dependent Censoring journal October 2010
Nested case-control data utilized for multiple outcomes: a likelihood approach and alternatives journal December 2008
Monotone Regression Splines in Action journal November 1988
Multivariate frailty models for two types of recurrent events with a dependent terminal event: Application to breast cancer data: Multivariate frailty models journal August 2013
Joint frailty models for recurring events and death using maximum penalized likelihood estimation: application on cancer events journal December 2006
Asymptotic Theory for Nested Case-Control Sampling in the Cox Regression Model journal December 1992
Risk of cardiovascular disease: The effects of diabetes and anti-diabetic drugs — A nested case–control study journal January 2015
Multiple Biomarkers for Risk Prediction in Chronic Heart Failure journal March 2012
A Penalized Likelihood Approach for Arbitrarily Censored and Truncated Data: Application to Age-Specific Incidence of Dementia journal March 1998
Tutorial in Joint Modeling and Prediction: A Statistical Software for Correlated Longitudinal Outcomes, Recurrent Events and a Terminal Event journal January 2017
Comprehensive prognostic analysis in breast cancer integrating clinical, tumoral, micro-environmental and immunohistochemical criteria journal September 2015
Semiparametric Analysis of Recurrent Events Data in the Presence of Dependent Censoring journal December 2003
A Joint Frailty Model for Survival and Gap Times Between Recurrent Events journal January 2007
Elevation of Tumor Necrosis Factor-α and Increased Risk of Recurrent Coronary Events After Myocardial Infarction journal May 2000
Inverse probability weighting in nested case-control studies with additional matching-a simulation study journal October 2013
General joint frailty model for recurrent event data with a dependent terminal event: Application to follicular lymphoma data journal February 2012
Nonparametric Analysis of Recurrent Events and Death journal June 2000
Shared Frailty Models for Recurrent Events and a Terminal Event journal August 2004
Panel Data with Survival: Hospitalization of HIV-Positive Patients journal March 1998
A pseudolikelihood approach to analysis of nested case-control studies journal June 1997
Methods of Cohort Analysis: Appraisal by Application to Asbestos Mining
  • Liddell, F. D. K.; McDonald, J. C.; Thomas, D. C.
  • Journal of the Royal Statistical Society. Series A (General), Vol. 140, Issue 4 https://doi.org/10.2307/2345280
journal January 1977
Semi-supervised approaches to efficient evaluation of model prediction performance journal December 2017
Analysis of secondary outcomes in nested case-control study designs journal June 2014
Resampling Procedures for Making Inference Under Nested Case–Control Studies journal December 2013
Analyzing Recurrent Event Data With Informative Censoring journal September 2001
Dynamic prediction of risk of death using history of cancer recurrences in joint frailty models journal September 2013
The value of reusing prior nested case-control data in new studies with different outcome journal February 2012
Postmenopausal Estrogen Therapy and Cardiovascular Disease: Ten-Year Follow-up from the Nurses' Health Study journal September 1991
Combining data from 2 nested case-control studies of overlapping cohorts to improve efficiency journal May 2008
Counter-matching: A stratified nested case-control sampling method journal March 1995
Clinical Effectiveness of Influenza Vaccination in Persons Younger Than 65 Years With High-Risk Medical Conditions: The PRISMA Study journal February 2005
Robust Estimation of Mean Functions and Treatment Effects for Recurrent Events Under Event-Dependent Censoring and Termination: Application to Skeletal Complications in Cancer Metastatic to Bone journal March 2009