{"title":"Joint modeling of generalized scale-change models for recurrent event and failure time data.","authors":"Xiaoyu Wang, Liuquan Sun","doi":"10.1007/s10985-022-09573-5","DOIUrl":"https://doi.org/10.1007/s10985-022-09573-5","url":null,"abstract":"<p><p>Recurrent event and failure time data arise frequently in many clinical and observational studies. In this article, we propose a joint modeling of generalized scale-change models for the recurrent event process and the failure time, and allow the two processes to be correlated through a shared frailty. The proposed joint model is flexible in that it requires neither the Poisson assumption for the recurrent event process nor a parametric assumption on the frailty distribution. Estimating equation approaches are developed for parameter estimation, and the asymptotic properties of the resulting estimators are established. Simulation studies are conducted to evaluate the finite sample performances of the proposed method. An application to a medical cost study of chronic heart failure patients is provided.</p>","PeriodicalId":49908,"journal":{"name":"Lifetime Data Analysis","volume":"29 1","pages":"1-33"},"PeriodicalIF":1.3,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10698296","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}
Worku B Ewnetu, Irène Gijbels, Anneleen Verhasselt
{"title":"Flexible two-piece distributions for right censored survival data.","authors":"Worku B Ewnetu, Irène Gijbels, Anneleen Verhasselt","doi":"10.1007/s10985-022-09574-4","DOIUrl":"https://doi.org/10.1007/s10985-022-09574-4","url":null,"abstract":"<p><p>An important complexity in censored data is that only partial information on the variables of interest is observed. In recent years, a large family of asymmetric distributions and maximum likelihood estimation for the parameters in that family has been studied, in the complete data case. In this paper, we exploit the appealing family of quantile-based asymmetric distributions to obtain flexible distributions for modelling right censored survival data. The flexible distributions can be generated using a variety of symmetric distributions and monotonic link functions. The interesting feature of this family is that the location parameter coincides with an index-parameter quantile of the distribution. This family is also suitable to characterize different shapes of the hazard function (constant, increasing, decreasing, bathtub and upside-down bathtub or unimodal shapes). Statistical inference is done for the whole family of distributions. The parameter estimation is carried out by optimizing a non-differentiable likelihood function. The asymptotic properties of the estimators are established. The finite-sample performance of the proposed method and the impact of censorship are investigated via simulations. Finally, the methodology is illustrated on two real data examples (times to weaning in breast-fed data and German Breast Cancer data).</p>","PeriodicalId":49908,"journal":{"name":"Lifetime Data Analysis","volume":"29 1","pages":"34-65"},"PeriodicalIF":1.3,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9196524","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":"Special issue dedicated to David Oakes.","authors":"Jong H Jeong, Amita K Manatunga","doi":"10.1007/s10985-022-09572-6","DOIUrl":"https://doi.org/10.1007/s10985-022-09572-6","url":null,"abstract":"","PeriodicalId":49908,"journal":{"name":"Lifetime Data Analysis","volume":"28 4","pages":"543-545"},"PeriodicalIF":1.3,"publicationDate":"2022-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10042215/pdf/nihms-1881987.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9187942","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 : 2022-10-01Epub Date: 2022-08-15DOI: 10.1007/s10985-022-09569-1
Philip Hougaard
{"title":"Choice of time scale for analysis of recurrent events data.","authors":"Philip Hougaard","doi":"10.1007/s10985-022-09569-1","DOIUrl":"https://doi.org/10.1007/s10985-022-09569-1","url":null,"abstract":"<p><p>Recurrent events refer to events that over time can occur several times for each individual. Full use of such data in a clinical trial requires a method that addresses the dependence between events. For modelling this dependence, there are two time scales to consider, namely time since start of the study (running time) or time since most recent event (gap time). In the multi-state setup, it is possible to estimate parameters also in the case, where the hazard model allows for an effect of both time scales, making this an extremely flexible approach. However, for summarizing the effect of a treatment in a transparent and informative way, the choice of time scale and model requires much more care. This paper discusses these choices both from a theoretical and practical point of view. This is supported by a simulation study showing that in a frailty model with assumptions covered by both time scales, the gap time approach may give misleading results. A literature dataset is used for illustrating the issues.</p>","PeriodicalId":49908,"journal":{"name":"Lifetime Data Analysis","volume":" ","pages":"700-722"},"PeriodicalIF":1.3,"publicationDate":"2022-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"40615707","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 : 2022-10-01Epub Date: 2022-06-28DOI: 10.1007/s10985-022-09560-w
Per Kragh Andersen, Eva Nina Sparre Wandall, Maja Pohar Perme
{"title":"Inference for transition probabilities in non-Markov multi-state models.","authors":"Per Kragh Andersen, Eva Nina Sparre Wandall, Maja Pohar Perme","doi":"10.1007/s10985-022-09560-w","DOIUrl":"https://doi.org/10.1007/s10985-022-09560-w","url":null,"abstract":"<p><p>Multi-state models are frequently used when data come from subjects observed over time and where focus is on the occurrence of events that the subjects may experience. A convenient modeling assumption is that the multi-state stochastic process is Markovian, in which case a number of methods are available when doing inference for both transition intensities and transition probabilities. The Markov assumption, however, is quite strict and may not fit actual data in a satisfactory way. Therefore, inference methods for non-Markov models are needed. In this paper, we review methods for estimating transition probabilities in such models and suggest ways of doing regression analysis based on pseudo observations. In particular, we will compare methods using land-marking with methods using plug-in. The methods are illustrated using simulations and practical examples from medical research.</p>","PeriodicalId":49908,"journal":{"name":"Lifetime Data Analysis","volume":" ","pages":"585-604"},"PeriodicalIF":1.3,"publicationDate":"2022-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"40407181","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 : 2022-10-01Epub Date: 2022-06-21DOI: 10.1007/s10985-022-09558-4
Ross L Prentice
{"title":"On the targets of inference with multivariate failure time data.","authors":"Ross L Prentice","doi":"10.1007/s10985-022-09558-4","DOIUrl":"https://doi.org/10.1007/s10985-022-09558-4","url":null,"abstract":"<p><p>There are several different topics that can be addressed with multivariate failure time regression data. Data analysis methods are needed that are suited to each such topic. Specifically, marginal hazard rate models are well suited to the analysis of exposures or treatments in relation to individual failure time outcomes, when failure time dependencies are themselves of little or no interest. On the other hand semiparametric copula models are well suited to analyses where interest focuses primarily on the magnitude of dependencies between failure times. These models overlap with frailty models, that seem best suited to exploring the details of failure time clustering. Recently proposed multivariate marginal hazard methods, on the other hand, are well suited to the exploration of exposures or treatments in relation to single, pairwise, and higher dimensional hazard rates. Here these methods will be briefly described, and the final method will be illustrated using the Women's Health Initiative hormone therapy trial data.</p>","PeriodicalId":49908,"journal":{"name":"Lifetime Data Analysis","volume":" ","pages":"546-559"},"PeriodicalIF":1.3,"publicationDate":"2022-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"40141664","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":"Semiparametric single-index models for optimal treatment regimens with censored outcomes.","authors":"Jin Wang, Donglin Zeng, D Y Lin","doi":"10.1007/s10985-022-09566-4","DOIUrl":"https://doi.org/10.1007/s10985-022-09566-4","url":null,"abstract":"<p><p>There is a growing interest in precision medicine, where a potentially censored survival time is often the most important outcome of interest. To discover optimal treatment regimens for such an outcome, we propose a semiparametric proportional hazards model by incorporating the interaction between treatment and a single index of covariates through an unknown monotone link function. This model is flexible enough to allow non-linear treatment-covariate interactions and yet provides a clinically interpretable linear rule for treatment decision. We propose a sieve maximum likelihood estimation approach, under which the baseline hazard function is estimated nonparametrically and the unknown link function is estimated via monotone quadratic B-splines. We show that the resulting estimators are consistent and asymptotically normal with a covariance matrix that attains the semiparametric efficiency bound. The optimal treatment rule follows naturally as a linear combination of the maximum likelihood estimators of the model parameters. Through extensive simulation studies and an application to an AIDS clinical trial, we demonstrate that the treatment rule derived from the single-index model outperforms the treatment rule under the standard Cox proportional hazards model.</p>","PeriodicalId":49908,"journal":{"name":"Lifetime Data Analysis","volume":"28 4","pages":"744-763"},"PeriodicalIF":1.3,"publicationDate":"2022-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10349573/pdf/nihms-1913037.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9775484","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 : 2022-10-01Epub Date: 2022-08-07DOI: 10.1007/s10985-022-09570-8
Debajyoti Sinha, Piyali Basak, Stuart R Lipsitz
{"title":"Median regression models for clustered, interval-censored survival data - An application to prostate surgery study.","authors":"Debajyoti Sinha, Piyali Basak, Stuart R Lipsitz","doi":"10.1007/s10985-022-09570-8","DOIUrl":"https://doi.org/10.1007/s10985-022-09570-8","url":null,"abstract":"<p><p>Genitourinary surgeons and oncologists are particularly interested in whether a robotic surgery improves times to Prostate Specific Antigen (PSA) recurrence compared to a non-robotic surgery for removing the cancerous prostate. Time to PSA recurrence is an example of a survival time that is typically interval-censored between two consecutive clinical inspections with opposite test results. In addition, success of medical devices and technologies often depends on factors such as experience and skill level of the medical service providers, thus leading to clustering of these survival times. For analyzing the effects of surgery types and other covariates on median of clustered interval-censored time to post-surgery PSA recurrence, we present three competing novel models and associated frequentist and Bayesian analyses. The first model is based on a transform-both-sides of survival time with Gaussian random effects to account for the within-cluster association. Our second model assumes an approximate marginal Laplace distribution for the transformed log-survival times with a Gaussian copula to accommodate clustering. Our third model is a special case of the second model with Laplace distribution for the marginal log-survival times and Gaussian copula for the within-cluster association. Simulation studies establish the second model to be highly robust against extreme observations while estimating median regression coefficients. We provide a comprehensive comparison among these three competing models based on the model properties and the computational ease of their Frequentist and Bayesian analysis. We also illustrate the practical implementations and uses of these methods via analysis of a simulated clustered interval-censored data-set similar in design to a post-surgery PSA recurrence study.</p>","PeriodicalId":49908,"journal":{"name":"Lifetime Data Analysis","volume":" ","pages":"723-743"},"PeriodicalIF":1.3,"publicationDate":"2022-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"40605557","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 : 2022-10-01Epub Date: 2022-06-20DOI: 10.1007/s10985-022-09561-9
Richard J Cook, Jerald F Lawless, Bingfeng Xie
{"title":"Marker-dependent observation and carry-forward of internal covariates in Cox regression.","authors":"Richard J Cook, Jerald F Lawless, Bingfeng Xie","doi":"10.1007/s10985-022-09561-9","DOIUrl":"https://doi.org/10.1007/s10985-022-09561-9","url":null,"abstract":"<p><p>Studies of chronic disease often involve modeling the relationship between marker processes and disease onset or progression. The Cox regression model is perhaps the most common and convenient approach to analysis in this setting. In most cohort studies, however, biospecimens and biomarker values are only measured intermittently (e.g. at clinic visits) so Cox models often treat biomarker values as fixed at their most recently observed values, until they are updated at the next visit. We consider the implications of this convention on the limiting values of regression coefficient estimators when the marker values themselves impact the intensity for clinic visits. A joint multistate model is described for the marker-failure-visit process which can be fitted to mitigate this bias and an expectation-maximization algorithm is developed. An application to data from a registry of patients with psoriatic arthritis is given for illustration.</p>","PeriodicalId":49908,"journal":{"name":"Lifetime Data Analysis","volume":" ","pages":"560-584"},"PeriodicalIF":1.3,"publicationDate":"2022-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"40104617","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 : 2022-10-01Epub Date: 2022-06-24DOI: 10.1007/s10985-022-09559-3
Mei-Cheng Wang, Yuxin Zhu
{"title":"Bias correction via outcome reassignment for cross-sectional data with binary disease outcome.","authors":"Mei-Cheng Wang, Yuxin Zhu","doi":"10.1007/s10985-022-09559-3","DOIUrl":"https://doi.org/10.1007/s10985-022-09559-3","url":null,"abstract":"<p><p>Cross-sectionally sampled data with binary disease outcome are commonly analyzed in observational studies to identify the relationship between covariates and disease outcome. A cross-sectional population is defined as a population of living individuals at the sampling or observational time. It is generally understood that binary disease outcome from cross-sectional data contains less information than longitudinally collected time-to-event data, but there is insufficient understanding as to whether bias can possibly exist in cross-sectional data and how the bias is related to the population risk of interest. Wang and Yang (2021) presented the complexity and bias in cross-sectional data with binary disease outcome with detailed analytical explorations into the data structure. As the distribution of the cross-sectional binary outcome is quite different from the population risk distribution, bias can arise when using cross-sectional data analysis to draw inference for population risk. In this paper we argue that the commonly adopted age-specific risk probability is biased for the estimation of population risk and propose an outcome reassignment approach which reassigns a portion of the observed binary outcome, 0 or 1, to the other disease category. A sign test and a semiparametric pseudo-likelihood method are developed for analyzing cross-sectional data using the OR approach. Simulations and an analysis based on Alzheimer's Disease data are presented to illustrate the proposed methods.</p>","PeriodicalId":49908,"journal":{"name":"Lifetime Data Analysis","volume":" ","pages":"659-674"},"PeriodicalIF":1.3,"publicationDate":"2022-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"40395608","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}