Lifetime Data Analysis最新文献

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Modelling dependent censoring in time-to-event data using boosting copula regression. 基于增强联结回归的时间-事件数据相关滤波建模。
IF 1 3区 数学
Lifetime Data Analysis Pub Date : 2025-10-21 DOI: 10.1007/s10985-025-09674-x
Annika Strömer, Nadja Klein, Ingrid Van Keilegom, Andreas Mayr
{"title":"Modelling dependent censoring in time-to-event data using boosting copula regression.","authors":"Annika Strömer, Nadja Klein, Ingrid Van Keilegom, Andreas Mayr","doi":"10.1007/s10985-025-09674-x","DOIUrl":"https://doi.org/10.1007/s10985-025-09674-x","url":null,"abstract":"","PeriodicalId":49908,"journal":{"name":"Lifetime Data Analysis","volume":" ","pages":""},"PeriodicalIF":1.0,"publicationDate":"2025-10-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145338136","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}
引用次数: 0
Statistical methods for composite analysis of recurrent and terminal events in clinical trials. 临床试验中复发性和终末期事件综合分析的统计方法。
IF 1 3区 数学
Lifetime Data Analysis Pub Date : 2025-10-15 DOI: 10.1007/s10985-025-09672-z
Yiyuan Huang, Douglas Schaubel, Min Zhang
{"title":"Statistical methods for composite analysis of recurrent and terminal events in clinical trials.","authors":"Yiyuan Huang, Douglas Schaubel, Min Zhang","doi":"10.1007/s10985-025-09672-z","DOIUrl":"https://doi.org/10.1007/s10985-025-09672-z","url":null,"abstract":"<p><p>In many clinical trials, one is interested in evaluating the treatment effect based on different types of outcomes, including recurrent and terminal events. The most popular approach is the time-to-first-event analysis (TTFE), based on the composite outcome of the time to the first event among all events of interest. The motivation for the composite outcome approach is to increase the number of events and potentially increase power. Other composite outcome or composite analysis methods are also studied in the literature, but are less adopted in practice. In this article, we first review the mainstream composite analysis methods and classify them into three categories: (A) Composite-outcome Methods, which combine multiple events into a composite outcome before analysis, e.g., combining events into a time-to-event outcome in TTFE and into a single recurrent event process in the combined-recurrent-event analysis (CRE); (B) Joint-analysis Methods, which test for the recurrent event process and the terminal event jointly, e.g., Joint Frailty Model (JFM), Ghosh-Lin Method (GL), and Nelsen-Aalen Method (NA); (C) Win-ratio type Methods that account for the ordering of two types of events, e.g., Win-fraction Regression (WR). We conduct comprehensive simulation studies to evaluate the performance of various types of methods in terms of type I error control and power under a wide range of scenarios. We found that the non-parametric joint testing approach (GL/NA) and CRE have overall the best performance. However, TTFE and WR exhibit relatively low power. Also, adding events that have no or weak association with treatment usually decreases power.</p>","PeriodicalId":49908,"journal":{"name":"Lifetime Data Analysis","volume":" ","pages":""},"PeriodicalIF":1.0,"publicationDate":"2025-10-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145294258","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}
引用次数: 0
Assessing delayed treatment benefits of immunotherapy using long-term average hazard: a novel test/estimation approach. 使用长期平均危害评估免疫治疗的延迟治疗益处:一种新的测试/估计方法。
IF 1 3区 数学
Lifetime Data Analysis Pub Date : 2025-10-14 DOI: 10.1007/s10985-025-09671-0
Miki Horiguchi, Lu Tian, Kenneth L Kehl, Hajime Uno
{"title":"Assessing delayed treatment benefits of immunotherapy using long-term average hazard: a novel test/estimation approach.","authors":"Miki Horiguchi, Lu Tian, Kenneth L Kehl, Hajime Uno","doi":"10.1007/s10985-025-09671-0","DOIUrl":"https://doi.org/10.1007/s10985-025-09671-0","url":null,"abstract":"<p><p>Delayed treatment effects on time-to-event outcomes are commonly observed in randomized controlled trials of cancer immunotherapies. When the treatment effect has a delayed onset, the conventional test/estimation approach-using the log-rank test for between-group comparison and Cox's hazard ratio to quantify the treatment effect-can be suboptimal. The log-rank test may lack power in such scenarios, and the interpretation of the hazard ratio is often ambiguous. Recently, alternative test/estimation approaches have been proposed to address these limitations. One such approach is based on long-term restricted mean survival time (LT-RMST), while another is based on average hazard with survival weight (AH-SW). This paper integrates these two concepts and introduces a novel long-term average hazard (LT-AH) approach with survival weight for both hypothesis testing and estimation. Numerical studies highlight specific scenarios where the proposed LT-AH method achieves higher power than the existing alternatives. The LT-AH for each group can be estimated nonparametrically, and the proposed between-group comparison maintains test/estimation coherency. Because the difference and ratio of LT-AH do not rely on model assumptions about the relationship between two groups, the LT-AH approach provides a robust framework for estimating the magnitude of between-group differences. Furthermore, LT-AH allows for treatment effect quantification in both absolute (difference in LT-AH) and relative (ratio of LT-AH) terms, aligning with guideline recommendations and addressing practical needs. Given its interpretability and improved power in certain settings, the proposed LT-AH approach offers a useful alternative to conventional hazard-based methods, particularly when delayed treatment effects are expected.</p>","PeriodicalId":49908,"journal":{"name":"Lifetime Data Analysis","volume":" ","pages":""},"PeriodicalIF":1.0,"publicationDate":"2025-10-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145287554","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}
引用次数: 0
Bayesian joint models for longitudinal, recurrent, and terminal event data. 纵向、循环和终端事件数据的贝叶斯联合模型。
IF 1 3区 数学
Lifetime Data Analysis Pub Date : 2025-10-09 DOI: 10.1007/s10985-025-09673-y
Emily M Damone, Matthew A Psioda, Joseph G Ibrahim
{"title":"Bayesian joint models for longitudinal, recurrent, and terminal event data.","authors":"Emily M Damone, Matthew A Psioda, Joseph G Ibrahim","doi":"10.1007/s10985-025-09673-y","DOIUrl":"https://doi.org/10.1007/s10985-025-09673-y","url":null,"abstract":"<p><p>Many methods exist to jointly model either recurrent and related terminal survival events or longitudinal outcome measures and related terminal survival event. However, few methods exist which can account for the dependency between all three outcomes of interest, and none allow for the modeling of all three outcomes without strong correlation assumptions. We propose a joint model which uses subject-specific random effects to connect the survival model (terminal and recurrent events) with a longitudinal outcome model. In the proposed method, proportional hazards models with shared frailties are used to model dependence between the recurrent and terminal events, while a separate (but correlated) set of random effects are utilized in a generalized linear mixed model to model dependence with longitudinal outcome measures. All random effects are related based on an assumed multivariate normal distribution. The proposed joint modeling approach allows for flexible models, particularly for unique longitudinal trajectories, that can be utilized in a wide range of health applications. We evaluate the model through simulation studies as well as through an application to data from the Atherosclerosis Risk in Communities (ARIC) study.</p>","PeriodicalId":49908,"journal":{"name":"Lifetime Data Analysis","volume":" ","pages":""},"PeriodicalIF":1.0,"publicationDate":"2025-10-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145253405","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}
引用次数: 0
Bayesian generalized method of moments applied to pseudo-observations in survival analysis. 贝叶斯广义矩法在生存分析伪观测中的应用。
IF 1 3区 数学
Lifetime Data Analysis Pub Date : 2025-09-22 DOI: 10.1007/s10985-025-09670-1
Léa Orsini, Caroline Brard, Emmanuel Lesaffre, Guosheng Yin, David Dejardin, Gwénaël Le Teuff
{"title":"Bayesian generalized method of moments applied to pseudo-observations in survival analysis.","authors":"Léa Orsini, Caroline Brard, Emmanuel Lesaffre, Guosheng Yin, David Dejardin, Gwénaël Le Teuff","doi":"10.1007/s10985-025-09670-1","DOIUrl":"https://doi.org/10.1007/s10985-025-09670-1","url":null,"abstract":"<p><p>Bayesian inference for survival regression modeling offers numerous advantages, especially for decision-making and external data borrowing, but demands the specification of the baseline hazard function, which may be a challenging task. We propose an alternative approach that does not need the specification of this function. Our approach combines pseudo-observations to convert censored data into longitudinal data with the generalized method of moments (GMM) to estimate the parameters of interest from the survival function directly. GMM may be viewed as an extension of the generalized estimating equations (GEE) currently used for frequentist pseudo-observations analysis and can be extended to the Bayesian framework using a pseudo-likelihood function. We assessed the behavior of the frequentist and Bayesian GMM in the new context of analyzing pseudo-observations. We compared their performances to the Cox, GEE, and Bayesian piecewise exponential models through a simulation study of two-arm randomized clinical trials. Frequentist and Bayesian GMMs gave valid inferences with similar performances compared to the three benchmark methods, except for small sample sizes and high censoring rates. For illustration, three post-hoc efficacy analyses were performed on randomized clinical trials involving patients with Ewing Sarcoma, producing results similar to those of the benchmark methods. Through a simple application of estimating hazard ratios, these findings confirm the effectiveness of this new Bayesian approach based on pseudo-observations and the generalized method of moments. This offers new insights on using pseudo-observations for Bayesian survival analysis.</p>","PeriodicalId":49908,"journal":{"name":"Lifetime Data Analysis","volume":" ","pages":""},"PeriodicalIF":1.0,"publicationDate":"2025-09-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145114927","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}
引用次数: 0
Pseudo-observations and super learner for the estimation of the restricted mean survival time. 估计有限平均生存时间的伪观察和超级学习器。
IF 1 3区 数学
Lifetime Data Analysis Pub Date : 2025-09-22 DOI: 10.1007/s10985-025-09668-9
Ariane Cwiling, Vittorio Perduca, Olivier Bouaziz
{"title":"Pseudo-observations and super learner for the estimation of the restricted mean survival time.","authors":"Ariane Cwiling, Vittorio Perduca, Olivier Bouaziz","doi":"10.1007/s10985-025-09668-9","DOIUrl":"https://doi.org/10.1007/s10985-025-09668-9","url":null,"abstract":"<p><p>In the context of right-censored data, we study the problem of predicting the restricted time to event based on a set of covariates. Under a quadratic loss, this problem is equivalent to estimating the conditional restricted mean survival time (RMST). To that aim, we propose a flexible and easy-to-use ensemble algorithm that combines pseudo-observations and super learner. The classical theoretical results of the super learner are extended to right-censored data, using a new definition of pseudo-observations, the so-called split pseudo-observations. Simulation studies indicate that the split pseudo-observations and the standard pseudo-observations are similar even for small sample sizes. The method is applied to maintenance and colon cancer datasets, showing the interest of the method in practice, as compared to other prediction methods. We complement the predictions obtained from our method with our RMST-adapted risk measure, prediction intervals and variable importance measures developed in a previous work.</p>","PeriodicalId":49908,"journal":{"name":"Lifetime Data Analysis","volume":" ","pages":""},"PeriodicalIF":1.0,"publicationDate":"2025-09-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145115022","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}
引用次数: 0
Bayesian joint analysis of longitudinal data and interval-censored failure time data. 纵向数据和间隔截尾失效时间数据的贝叶斯联合分析。
IF 1 3区 数学
Lifetime Data Analysis Pub Date : 2025-08-27 DOI: 10.1007/s10985-025-09666-x
Yuchen Mao, Lianming Wang, Xuemei Sui
{"title":"Bayesian joint analysis of longitudinal data and interval-censored failure time data.","authors":"Yuchen Mao, Lianming Wang, Xuemei Sui","doi":"10.1007/s10985-025-09666-x","DOIUrl":"10.1007/s10985-025-09666-x","url":null,"abstract":"<p><p>Joint modeling of longitudinal responses and survival time has gained great attention in statistics literature over the last few decades. Most existing works focus on joint analysis of longitudinal data and right-censored data. In this article, we propose a new frailty model for joint analysis of a longitudinal response and interval-censored survival time. Such data commonly arise in real-life studies where participants are examined at periodical or irregular follow-up times. The proposed joint model contains a nonlinear mixed effects submodel for the longitudinal response and a semiparametric probit submodel for the survival time given a shared normal frailty. The proposed joint model allows the regression coefficients to be interpreted as the marginal effects up to a multiplicative constant on both the longitudinal and survival responses. Adopting splines allows us to approximate the unknown baseline functions in both submodels with only a finite number of unknown coefficients while providing great modeling flexibility. An efficient Gibbs sampler is developed for posterior computation, in which all parameters and latent variables can be sampled easily from their full conditional distributions. The proposed method shows a good estimation performance in simulation studies and is further illustrated by a real-life application to the patient data from the Aerobics Center Longitudinal Study. The R code for the proposed methodology is made available for public use.</p>","PeriodicalId":49908,"journal":{"name":"Lifetime Data Analysis","volume":" ","pages":""},"PeriodicalIF":1.0,"publicationDate":"2025-08-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144976304","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}
引用次数: 0
Causal effect estimation on restricted mean survival time under case-cohort design via propensity score stratification. 通过倾向评分分层对病例队列设计下受限平均生存时间的因果效应估计。
IF 1 3区 数学
Lifetime Data Analysis Pub Date : 2025-08-18 DOI: 10.1007/s10985-025-09667-w
Wei-En Lu, Ai Ni
{"title":"Causal effect estimation on restricted mean survival time under case-cohort design via propensity score stratification.","authors":"Wei-En Lu, Ai Ni","doi":"10.1007/s10985-025-09667-w","DOIUrl":"https://doi.org/10.1007/s10985-025-09667-w","url":null,"abstract":"<p><p>In large observational studies with survival outcome and low event rates, the case-cohort design is commonly used to reduce the cost associated with covariate measurement. The restricted mean survival time (RMST) difference has been increasingly used as an alternative to hazard ratio when estimating the causal effect on survival outcomes. We investigate the estimation of marginal causal effect on RMST under the stratified case-cohort design while adjusting for measured confounders through propensity score stratification. The asymptotic normality of the estimator is established, and its variance formula is derived. Simulation studies are performed to evaluate the finite sample performance of the proposed method compared to several alternative methods. Finally, we apply the proposed method to the Atherosclerosis Risk in Communities study to estimate the marginal causal effect of high-sensitivity C-reactive protein level on coronary heart disease-free survival.</p>","PeriodicalId":49908,"journal":{"name":"Lifetime Data Analysis","volume":" ","pages":""},"PeriodicalIF":1.0,"publicationDate":"2025-08-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144876417","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}
引用次数: 0
Simultaneous clustering and joint modeling of multivariate binary longitudinal and time-to-event data. 多元二元纵向和时间-事件数据的同时聚类和联合建模。
IF 1.2 3区 数学
Lifetime Data Analysis Pub Date : 2025-07-12 DOI: 10.1007/s10985-025-09664-z
Srijan Chattopadhyay, Sevantee Basu, Swapnaneel Bhattacharyya, Manash Pratim Gogoi, Kiranmoy Das
{"title":"Simultaneous clustering and joint modeling of multivariate binary longitudinal and time-to-event data.","authors":"Srijan Chattopadhyay, Sevantee Basu, Swapnaneel Bhattacharyya, Manash Pratim Gogoi, Kiranmoy Das","doi":"10.1007/s10985-025-09664-z","DOIUrl":"https://doi.org/10.1007/s10985-025-09664-z","url":null,"abstract":"<p><p>Joint modeling of longitudinal outcomes and time-to-event data has been extensively used in medical studies because it can simultaneously model the longitudinal trajectories and assess their effects on the event-time. However, in many applications we come across heterogeneous populations, and therefore the subjects need to be clustered for a powerful statistical inference. We consider multivariate binary longitudinal outcomes for which we use Bayesian data-augmentation and get the corresponding latent continuous outcomes. These latent outcomes are clustered using Bayesian consensus clustering, and then we perform a cluster-specific joint analysis. Longitudinal outcomes are modeled by generalized linear mixed models, and we use the proportional hazards model for modeling time-to-event data. Our work is motivated by a clinical trial conducted by Tata Translational Cancer Research Center, Kolkata, where 184 cancer patients were treated for the first two years, and then were followed for the next three years. Three biomarkers (lymphocyte count, neutrophil count and platelet count), categorized as normal/abnormal, were measured during the treatment, and the relapse time (if any) was recorded for each patient. Our analysis finds three latent clusters for which the effects of the covariates and the median non-relapse probabilities substantially differ. Through a simulation study we illustrate the effectiveness of the proposed simultaneous clustering and joint modeling.</p>","PeriodicalId":49908,"journal":{"name":"Lifetime Data Analysis","volume":" ","pages":""},"PeriodicalIF":1.2,"publicationDate":"2025-07-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144621008","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}
引用次数: 0
Analysis of interval censored survival data in sequential multiple assignment randomized trials. 序贯多任务随机试验中间隔截尾生存数据分析。
IF 1.2 3区 数学
Lifetime Data Analysis Pub Date : 2025-07-11 DOI: 10.1007/s10985-025-09665-y
Zhiguo Li
{"title":"Analysis of interval censored survival data in sequential multiple assignment randomized trials.","authors":"Zhiguo Li","doi":"10.1007/s10985-025-09665-y","DOIUrl":"https://doi.org/10.1007/s10985-025-09665-y","url":null,"abstract":"<p><p>Data analysis methods have been well developed for analyzing data to make inferences about adaptive treatment strategies in sequential multiple assignment randomized trials (SMART), when data are continuous or right-censored. However, in some clinical studies, time-to-event outcomes are interval censored, meaning that, for example, the time of interest is only observed between two random visit times to the clinic, which is common in some areas such as psychology studies. In this case, the appropriate analysis methods in SMART studies have not been considered in the literature. This article tries to fill this gap by developing methods for this purpose. Based on a proportional hazards model, we propose to use a weighted spline-based sieve maximum likelihood method to make inference about the group differences using a Wald test. Asymptotic properties of the estimator for the hazard ratio are derived, and variance estimation is considered. We conduct a simulation to assess its finite sample performance, and then analyze data from the Sequenced Treatment Alternatives to Relieve Depression (STAR*D) trial.</p>","PeriodicalId":49908,"journal":{"name":"Lifetime Data Analysis","volume":" ","pages":""},"PeriodicalIF":1.2,"publicationDate":"2025-07-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144621007","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}
引用次数: 0
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