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

Jazić, Ina; Haneuse, Sebastien; French, Benjamin; MacGrogan, Gaëtan; Rondeau, Virginie

doi:10.1002/sim.8302

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

Journal Article · Tue Jul 09 00:00:00 EDT 2019 · Statistics in Medicine

DOI:https://doi.org/10.1002/sim.8302· OSTI ID:1532770

^[1]; Haneuse, Sebastien ^[1]; French, Benjamin ^[2]; MacGrogan, Gaëtan ^[3]; Rondeau, Virginie ^[4]

Department of Biostatistics Harvard T.H. Chan School of Public Health Boston Massachusetts
Department of Statistics Radiation Effects Research Foundation Hiroshima Japan
Department of Biopathology Institut Bergonié Bordeaux France
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.

View Accepted Manuscript (Publisher)

Sponsoring Organization:: USDOE

Grant/Contract Number:: HS0000031

OSTI ID:: 1532770

Alternate ID(s):: OSTI ID: 1613761

Journal Information:: Statistics in Medicine, Journal Name: Statistics in Medicine Journal Issue: 22 Vol. 38; ISSN 0277-6715

Publisher:: Wiley Blackwell (John Wiley & Sons)Copyright Statement

Country of Publication:: United Kingdom

Language:: English

References (36)

Clinical Effectiveness of Influenza Vaccination in Persons Younger Than 65 Years With High-Risk Medical Conditions: The PRISMA Study Hak, Eelko Archives of Internal Medicine, Vol. 165, Issue 3 https://doi.org/10.1001/archinte.165.3.274	journal	February 2005
Multivariate frailty models for two types of recurrent events with a dependent terminal event: Application to breast cancer data: Multivariate frailty models Mazroui, Yassin; Mathoulin-Pélissier, Simone; MacGrogan, Gaetan Biometrical Journal, Vol. 55, Issue 6 https://doi.org/10.1002/bimj.201200196	journal	August 2013
Nested case-control data utilized for multiple outcomes: a likelihood approach and alternatives Saarela, Olli; Kulathinal, Sangita; Arjas, Elja Statistics in Medicine, Vol. 27, Issue 28 https://doi.org/10.1002/sim.3416	journal	December 2008
General joint frailty model for recurrent event data with a dependent terminal event: Application to follicular lymphoma data Mazroui, Yassin; Mathoulin-Pelissier, Simone; Soubeyran, Pierre Statistics in Medicine, Vol. 31, Issue 11-12 https://doi.org/10.1002/sim.4479	journal	February 2012
The value of reusing prior nested case-control data in new studies with different outcome Salim, Agus; Yang, Qian; Reilly, Marie Statistics in Medicine, Vol. 31, Issue 11-12 https://doi.org/10.1002/sim.4494	journal	February 2012
Dynamic prediction of risk of death using history of cancer recurrences in joint frailty models Mauguen, Audrey; Rachet, Bernard; Mathoulin-Pélissier, Simone Statistics in Medicine, Vol. 32, Issue 30 https://doi.org/10.1002/sim.5980	journal	September 2013
Inverse probability weighting in nested case-control studies with additional matching-a simulation study Støer, Nathalie C.; Samuelsen, Sven Ove Statistics in Medicine, Vol. 32, Issue 30 https://doi.org/10.1002/sim.6019	journal	October 2013
Analysis of secondary outcomes in nested case-control study designs Kim, Ryung S.; Kaplan, Robert C. Statistics in Medicine, Vol. 33, Issue 24 https://doi.org/10.1002/sim.6231	journal	June 2014
Risk of cardiovascular disease: The effects of diabetes and anti-diabetic drugs — A nested case–control study Gejl, Michael; Starup-Linde, Jakob; Scheel-Thomsen, Jan International Journal of Cardiology, Vol. 178 https://doi.org/10.1016/j.ijcard.2014.11.096	journal	January 2015
Postmenopausal Estrogen Therapy and Cardiovascular Disease: Ten-Year Follow-up from the Nurses' Health Study Stampfer, Meir J.; Colditz, Graham A.; Willett, Walter C. New England Journal of Medicine, Vol. 325, Issue 11 https://doi.org/10.1056/NEJM199109123251102	journal	September 1991
Panel Data with Survival: Hospitalization of HIV-Positive Patients Lancaster, Tony; Intrator, Orna Journal of the American Statistical Association, Vol. 93, Issue 441 https://doi.org/10.1080/01621459.1998.10474086	journal	March 1998
Resampling Procedures for Making Inference Under Nested Case–Control Studies Cai, Tianxi; Zheng, Yingye Journal of the American Statistical Association, Vol. 108, Issue 504 https://doi.org/10.1080/01621459.2013.856715	journal	December 2013
Counter-matching: A stratified nested case-control sampling method Langholz, B.; Borgan, O. R. Biometrika, Vol. 82, Issue 1 https://doi.org/10.1093/biomet/82.1.69	journal	March 1995
A pseudolikelihood approach to analysis of nested case-control studies Samuelsen, S. Biometrika, Vol. 84, Issue 2 https://doi.org/10.1093/biomet/84.2.379	journal	June 1997
Joint frailty models for recurring events and death using maximum penalized likelihood estimation: application on cancer events Rondeau, V.; Mathoulin-Pelissier, S.; Jacqmin-Gadda, H. Biostatistics, Vol. 8, Issue 4 https://doi.org/10.1093/biostatistics/kxl043	journal	December 2006
Combining data from 2 nested case-control studies of overlapping cohorts to improve efficiency Salim, A.; Hultman, C.; Sparen, P. Biostatistics, Vol. 10, Issue 1 https://doi.org/10.1093/biostatistics/kxn016	journal	May 2008
Nonparametric Analysis of Recurrent Events and Death Ghosh, Debashis; Lin, D. Y. Biometrics, Vol. 56, Issue 2 https://doi.org/10.1111/j.0006-341X.2000.00554.x	journal	June 2000
Semiparametric Analysis of Recurrent Events Data in the Presence of Dependent Censoring Ghosh, Debashis; Lin, D. Y. Biometrics, Vol. 59, Issue 4 https://doi.org/10.1111/j.0006-341X.2003.00102.x	journal	December 2003
Shared Frailty Models for Recurrent Events and a Terminal Event Liu, Lei; Wolfe, Robert A.; Huang, Xuelin Biometrics, Vol. 60, Issue 3 https://doi.org/10.1111/j.0006-341X.2004.00225.x	journal	August 2004
A Joint Frailty Model for Survival and Gap Times Between Recurrent Events Huang, Xuelin; Liu, Lei Biometrics, Vol. 63, Issue 2 https://doi.org/10.1111/j.1541-0420.2006.00719.x	journal	January 2007
Marginal Mark Regression Analysis of Recurrent Marked Point Process Data French, Benjamin; Heagerty, Patrick J. Biometrics, Vol. 65, Issue 2 https://doi.org/10.1111/j.1541-0420.2008.01076.x	journal	June 2008
Regression Analysis for Recurrent Events Data under Dependent Censoring Hsieh, Jin-Jian; Ding, A. Adam; Wang, Weijing Biometrics, Vol. 67, Issue 3 https://doi.org/10.1111/j.1541-0420.2010.01497.x	journal	October 2010
Semi-supervised approaches to efficient evaluation of model prediction performance Gronsbell, Jessica L.; Cai, Tianxi Journal of the Royal Statistical Society: Series B (Statistical Methodology), Vol. 80, Issue 3 https://doi.org/10.1111/rssb.12264	journal	December 2017
An Algorithm for Least-Squares Estimation of Nonlinear Parameters Marquardt, Donald W. Journal of the Society for Industrial and Applied Mathematics, Vol. 11, Issue 2 https://doi.org/10.1137/0111030	journal	June 1963
Elevation of Tumor Necrosis Factor-α and Increased Risk of Recurrent Coronary Events After Myocardial Infarction Ridker, Paul M.; Rifai, Nader; Pfeffer, Marc Circulation, Vol. 101, Issue 18 https://doi.org/10.1161/01.CIR.101.18.2149	journal	May 2000
Multiple Biomarkers for Risk Prediction in Chronic Heart Failure Ky, Bonnie; French, Benjamin; Levy, Wayne C. Circulation: Heart Failure, Vol. 5, Issue 2 https://doi.org/10.1161/CIRCHEARTFAILURE.111.965020	journal	March 2012
Heart Failure With Recovered Ejection Fraction: Clinical Description, Biomarkers, and Outcomes Basuray, Anupam; French, Benjamin; Ky, Bonnie Circulation, Vol. 129, Issue 23 https://doi.org/10.1161/CIRCULATIONAHA.113.006855	journal	June 2014
Comprehensive prognostic analysis in breast cancer integrating clinical, tumoral, micro-environmental and immunohistochemical criteria de Mascarel, Isabelle; Debled, Marc; Brouste, Véronique SpringerPlus, Vol. 4, Issue 1 https://doi.org/10.1186/s40064-015-1297-8	journal	September 2015
Analyzing Recurrent Event Data With Informative Censoring Wang, Mei-Cheng; Qin, Jing; Chiang, Chin-Tsang Journal of the American Statistical Association, Vol. 96, Issue 455 https://doi.org/10.1198/016214501753209031	journal	September 2001
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 Cook, Richard J.; Lawless, Jerald F.; Lakhal-Chaieb, Lajmi Journal of the American Statistical Association, Vol. 104, Issue 485 https://doi.org/10.1198/jasa.2009.0004	journal	March 2009
Asymptotic Theory for Nested Case-Control Sampling in the Cox Regression Model Goldstein, Larry; Langholz, Bryan The Annals of Statistics, Vol. 20, Issue 4 https://doi.org/10.1214/aos/1176348895	journal	December 1992
Monotone Regression Splines in Action Ramsay, J. O. Statistical Science, Vol. 3, Issue 4 https://doi.org/10.1214/ss/1177012761	journal	November 1988
Sampling strategies in nested case-control studies. Langholz, B.; Clayton, D. Environmental Health Perspectives, Vol. 102, Issue suppl 8 https://doi.org/10.1289/ehp.94102s847	journal	November 1994
Tutorial in Joint Modeling and Prediction: A Statistical Software for Correlated Longitudinal Outcomes, Recurrent Events and a Terminal Event Król, Agnieszka; Mauguen, Audrey; Mazroui, Yassin Journal of Statistical Software, Vol. 81, Issue 3 https://doi.org/10.18637/jss.v081.i03	journal	January 2017
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
A Penalized Likelihood Approach for Arbitrarily Censored and Truncated Data: Application to Age-Specific Incidence of Dementia Joly, Pierre; Commenges, Daniel; Letenneur, Luc Biometrics, Vol. 54, Issue 1 https://doi.org/10.2307/2534006	journal	March 1998

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