Lifetime Data AnalysisPub Date : 2024-07-01Epub Date: 2024-05-28DOI: 10.1007/s10985-024-09626-x
Jiayin Zheng, Li Hsu
{"title":"Risk projection for time-to-event outcome from population-based case-control studies leveraging summary statistics from the target population.","authors":"Jiayin Zheng, Li Hsu","doi":"10.1007/s10985-024-09626-x","DOIUrl":"10.1007/s10985-024-09626-x","url":null,"abstract":"<p><p>Risk stratification based on prediction models has become increasingly important in preventing and managing chronic diseases. However, due to cost- and time-limitations, not every population can have resources for collecting enough detailed individual-level information on a large number of people to develop risk prediction models. A more practical approach is to use prediction models developed from existing studies and calibrate them with relevant summary-level information of the target population. Many existing studies were conducted under the population-based case-control design. Gail et al. (J Natl Cancer Inst 81:1879-1886, 1989) proposed to combine the odds ratio estimates obtained from case-control data and the disease incidence rates from the target population to obtain the baseline hazard function, and thereby the pure risk for developing diseases. However, the approach requires the risk factor distribution of cases from the case-control studies be same as the target population, which, if violated, may yield biased risk estimation. In this article, we propose two novel weighted estimating equation approaches to calibrate the baseline risk by leveraging the summary information of (some) risk factors in addition to disease-free probabilities from the targeted population. We establish the consistency and asymptotic normality of the proposed estimators. Extensive simulation studies and an application to colorectal cancer studies demonstrate the proposed estimators perform well for bias reduction in finite samples.</p>","PeriodicalId":49908,"journal":{"name":"Lifetime Data Analysis","volume":" ","pages":"549-571"},"PeriodicalIF":1.2,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11283322/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141158740","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"数学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Lifetime Data AnalysisPub Date : 2024-07-01Epub Date: 2024-05-28DOI: 10.1007/s10985-024-09627-w
Anindya Bhadra, Rubin Wei, Ruth Keogh, Victor Kipnis, Douglas Midthune, Dennis W Buckman, Ya Su, Ananya Roy Chowdhury, Raymond J Carroll
{"title":"Measurement error models with zero inflation and multiple sources of zeros, with applications to hard zeros.","authors":"Anindya Bhadra, Rubin Wei, Ruth Keogh, Victor Kipnis, Douglas Midthune, Dennis W Buckman, Ya Su, Ananya Roy Chowdhury, Raymond J Carroll","doi":"10.1007/s10985-024-09627-w","DOIUrl":"10.1007/s10985-024-09627-w","url":null,"abstract":"<p><p>We consider measurement error models for two variables observed repeatedly and subject to measurement error. One variable is continuous, while the other variable is a mixture of continuous and zero measurements. This second variable has two sources of zeros. The first source is episodic zeros, wherein some of the measurements for an individual may be zero and others positive. The second source is hard zeros, i.e., some individuals will always report zero. An example is the consumption of alcohol from alcoholic beverages: some individuals consume alcoholic beverages episodically, while others never consume alcoholic beverages. However, with a small number of repeat measurements from individuals, it is not possible to determine those who are episodic zeros and those who are hard zeros. We develop a new measurement error model for this problem, and use Bayesian methods to fit it. Simulations and data analyses are used to illustrate our methods. Extensions to parametric models and survival analysis are discussed briefly.</p>","PeriodicalId":49908,"journal":{"name":"Lifetime Data Analysis","volume":" ","pages":"600-623"},"PeriodicalIF":1.2,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141162786","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"数学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Lifetime Data AnalysisPub Date : 2024-07-01Epub Date: 2024-03-21DOI: 10.1007/s10985-024-09623-0
Robert L Strawderman, Benjamin R Baer
{"title":"On the role of Volterra integral equations in self-consistent, product-limit, inverse probability of censoring weighted, and redistribution-to-the-right estimators for the survival function.","authors":"Robert L Strawderman, Benjamin R Baer","doi":"10.1007/s10985-024-09623-0","DOIUrl":"10.1007/s10985-024-09623-0","url":null,"abstract":"<p><p>This paper reconsiders several results of historical and current importance to nonparametric estimation of the survival distribution for failure in the presence of right-censored observation times, demonstrating in particular how Volterra integral equations help inter-connect the resulting estimators. The paper begins by considering Efron's self-consistency equation, introduced in a seminal 1967 Berkeley symposium paper. Novel insights provided in the current work include the observations that (i) the self-consistency equation leads directly to an anticipating Volterra integral equation whose solution is given by a product-limit estimator for the censoring survival function; (ii) a definition used in this argument immediately establishes the familiar product-limit estimator for the failure survival function; (iii) the usual Volterra integral equation for the product-limit estimator of the failure survival function leads to an immediate and simple proof that it can be represented as an inverse probability of censoring weighted estimator; (iv) a simple identity characterizes the relationship between natural inverse probability of censoring weighted estimators for the survival and distribution functions of failure; (v) the resulting inverse probability of censoring weighted estimators, attributed to a highly influential 1992 paper of Robins and Rotnitzky, were implicitly introduced in Efron's 1967 paper in its development of the redistribution-to-the-right algorithm. All results developed herein allow for ties between failure and/or censored observations.</p>","PeriodicalId":49908,"journal":{"name":"Lifetime Data Analysis","volume":" ","pages":"649-666"},"PeriodicalIF":1.2,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140186140","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"数学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Regression analysis of doubly censored failure time data with ancillary information","authors":"Mingyue Du, Xiyuan Gao, Ling Chen","doi":"10.1007/s10985-024-09625-y","DOIUrl":"https://doi.org/10.1007/s10985-024-09625-y","url":null,"abstract":"<p>Doubly censored failure time data occur in many areas and for the situation, the failure time of interest usually represents the elapsed time between two related events such as an infection and the resulting disease onset. Although many methods have been proposed for regression analysis of such data, most of them are conditional on the occurrence time of the initial event and ignore the relationship between the two events or the ancillary information contained in the initial event. Corresponding to this, a new sieve maximum likelihood approach is proposed that makes use of the ancillary information, and in the method, the logistic model and Cox proportional hazards model are employed to model the initial event and the failure time of interest, respectively. A simulation study is conducted and suggests that the proposed method works well in practice and is more efficient than the existing methods as expected. The approach is applied to an AIDS study that motivated this investigation.</p>","PeriodicalId":49908,"journal":{"name":"Lifetime Data Analysis","volume":"224 1","pages":""},"PeriodicalIF":1.3,"publicationDate":"2024-04-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140625569","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"数学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Partial-linear single-index transformation models with censored data","authors":"Myeonggyun Lee, Andrea B. Troxel, Mengling Liu","doi":"10.1007/s10985-024-09624-z","DOIUrl":"https://doi.org/10.1007/s10985-024-09624-z","url":null,"abstract":"<p>In studies with time-to-event outcomes, multiple, inter-correlated, and time-varying covariates are commonly observed. It is of great interest to model their joint effects by allowing a flexible functional form and to delineate their relative contributions to survival risk. A class of semiparametric transformation (ST) models offers flexible specifications of the intensity function and can be a general framework to accommodate nonlinear covariate effects. In this paper, we propose a partial-linear single-index (PLSI) transformation model that reduces the dimensionality of multiple covariates into a single index and provides interpretable estimates of the covariate effects. We develop an iterative algorithm using the regression spline technique to model the nonparametric single-index function for possibly nonlinear joint effects, followed by nonparametric maximum likelihood estimation. We also propose a nonparametric testing procedure to formally examine the linearity of covariate effects. We conduct Monte Carlo simulation studies to compare the PLSI transformation model with the standard ST model and apply it to NYU Langone Health de-identified electronic health record data on COVID-19 hospitalized patients’ mortality and a Veteran’s Administration lung cancer trial.</p>","PeriodicalId":49908,"journal":{"name":"Lifetime Data Analysis","volume":"19 1","pages":""},"PeriodicalIF":1.3,"publicationDate":"2024-04-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140574258","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"数学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Cox model inference for relative hazard and pure risk from stratified weight-calibrated case-cohort data","authors":"","doi":"10.1007/s10985-024-09621-2","DOIUrl":"https://doi.org/10.1007/s10985-024-09621-2","url":null,"abstract":"<h3>Abstract</h3> <p>The case-cohort design obtains complete covariate data only on cases and on a random sample (the subcohort) of the entire cohort. Subsequent publications described the use of stratification and weight calibration to increase efficiency of estimates of Cox model log-relative hazards, and there has been some work estimating pure risk. Yet there are few examples of these options in the medical literature, and we could not find programs currently online to analyze these various options. We therefore present a unified approach and R software to facilitate such analyses. We used influence functions adapted to the various design and analysis options together with variance calculations that take the two-phase sampling into account. This work clarifies when the widely used “robust” variance estimate of Barlow (Biometrics 50:1064–1072, 1994) is appropriate. The corresponding R software, CaseCohortCoxSurvival, facilitates analysis with and without stratification and/or weight calibration, for subcohort sampling with or without replacement. We also allow for phase-two data to be missing at random for stratified designs. We provide inference not only for log-relative hazards in the Cox model, but also for cumulative baseline hazards and covariate-specific pure risks. We hope these calculations and software will promote wider use of more efficient and principled design and analysis options for case-cohort studies.</p>","PeriodicalId":49908,"journal":{"name":"Lifetime Data Analysis","volume":"45 1","pages":""},"PeriodicalIF":1.3,"publicationDate":"2024-04-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140574106","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"数学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Lifetime Data AnalysisPub Date : 2024-04-01Epub Date: 2023-11-26DOI: 10.1007/s10985-023-09611-w
Theresa P Devasia, Alexander Tsodikov
{"title":"Efficiency of the Breslow estimator in semiparametric transformation models.","authors":"Theresa P Devasia, Alexander Tsodikov","doi":"10.1007/s10985-023-09611-w","DOIUrl":"10.1007/s10985-023-09611-w","url":null,"abstract":"<p><p>Semiparametric transformation models for failure time data consist of a parametric regression component and an unspecified cumulative baseline hazard. The nonparametric maximum likelihood estimator (NPMLE) of the cumulative baseline hazard can be summarized in terms of weights introduced into a Breslow-type estimator (Weighted Breslow). At any given time point, the weights invoke an integral over the future of the cumulative baseline hazard, which presents theoretical and computational challenges. A simpler non-MLE Breslow-type estimator (Breslow) was derived earlier from a martingale estimating equation (MEE) setting observed and expected counts of failures equal, conditional on the past history. Despite much successful theoretical and computational development, the simpler Breslow estimator continues to be commonly used as a compromise between simplicity and perceived loss of full efficiency. In this paper we derive the relative efficiency of the Breslow estimator and consider the properties of the two estimators using simulations and real data on prostate cancer survival.</p>","PeriodicalId":49908,"journal":{"name":"Lifetime Data Analysis","volume":" ","pages":"291-309"},"PeriodicalIF":1.2,"publicationDate":"2024-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11237962/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138441550","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"数学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Lifetime Data AnalysisPub Date : 2024-04-01Epub Date: 2023-11-28DOI: 10.1007/s10985-023-09613-8
Chun Pan, Bo Cai, Xuemei Sui
{"title":"A Bayesian proportional hazards mixture cure model for interval-censored data.","authors":"Chun Pan, Bo Cai, Xuemei Sui","doi":"10.1007/s10985-023-09613-8","DOIUrl":"10.1007/s10985-023-09613-8","url":null,"abstract":"<p><p>The proportional hazards mixture cure model is a popular analysis method for survival data where a subgroup of patients are cured. When the data are interval-censored, the estimation of this model is challenging due to its complex data structure. In this article, we propose a computationally efficient semiparametric Bayesian approach, facilitated by spline approximation and Poisson data augmentation, for model estimation and inference with interval-censored data and a cure rate. The spline approximation and Poisson data augmentation greatly simplify the MCMC algorithm and enhance the convergence of the MCMC chains. The empirical properties of the proposed method are examined through extensive simulation studies and also compared with the R package \"GORCure\". The use of the proposed method is illustrated through analyzing a data set from the Aerobics Center Longitudinal Study.</p>","PeriodicalId":49908,"journal":{"name":"Lifetime Data Analysis","volume":" ","pages":"327-344"},"PeriodicalIF":1.2,"publicationDate":"2024-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138446796","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"数学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Lifetime Data AnalysisPub Date : 2024-04-01Epub Date: 2024-01-18DOI: 10.1007/s10985-023-09614-7
Cui-Juan Kong, Han-Ying Liang
{"title":"Quantile difference estimation with censoring indicators missing at random.","authors":"Cui-Juan Kong, Han-Ying Liang","doi":"10.1007/s10985-023-09614-7","DOIUrl":"10.1007/s10985-023-09614-7","url":null,"abstract":"<p><p>In this paper, we define estimators of distribution functions when the data are right-censored and the censoring indicators are missing at random, and establish their strong representations and asymptotic normality. Besides, based on empirical likelihood method, we define maximum empirical likelihood estimators and smoothed log-empirical likelihood ratios of two-sample quantile difference in the presence and absence of auxiliary information, respectively, and prove their asymptotic distributions. Simulation study and real data analysis are conducted to investigate the finite sample behavior of the proposed methods.</p>","PeriodicalId":49908,"journal":{"name":"Lifetime Data Analysis","volume":" ","pages":"345-382"},"PeriodicalIF":1.2,"publicationDate":"2024-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139492096","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"数学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Lifetime Data AnalysisPub Date : 2024-04-01Epub Date: 2024-03-04DOI: 10.1007/s10985-024-09619-w
Motahareh Parsa, Seyed Mahmood Taghavi-Shahri, Ingrid Van Keilegom
{"title":"On variable selection in a semiparametric AFT mixture cure model.","authors":"Motahareh Parsa, Seyed Mahmood Taghavi-Shahri, Ingrid Van Keilegom","doi":"10.1007/s10985-024-09619-w","DOIUrl":"10.1007/s10985-024-09619-w","url":null,"abstract":"<p><p>In clinical studies, one often encounters time-to-event data that are subject to right censoring and for which a fraction of the patients under study never experience the event of interest. Such data can be modeled using cure models in survival analysis. In the presence of cure fraction, the mixture cure model is popular, since it allows to model probability to be cured (called the incidence) and the survival function of the uncured individuals (called the latency). In this paper, we develop a variable selection procedure for the incidence and latency parts of a mixture cure model, consisting of a logistic model for the incidence and a semiparametric accelerated failure time model for the latency. We use a penalized likelihood approach, based on adaptive LASSO penalties for each part of the model, and we consider two algorithms for optimizing the criterion function. Extensive simulations are carried out to assess the accuracy of the proposed selection procedure. Finally, we employ the proposed method to a real dataset regarding heart failure patients with left ventricular systolic dysfunction.</p>","PeriodicalId":49908,"journal":{"name":"Lifetime Data Analysis","volume":" ","pages":"472-500"},"PeriodicalIF":1.2,"publicationDate":"2024-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140023093","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"数学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}