Lifetime Data AnalysisPub Date : 2024-01-01Epub Date: 2023-03-22DOI: 10.1007/s10985-023-09595-7
Yun-Lin Ho, Ju-Sheng Hong, Yen-Tsung Huang
{"title":"Model-based hypothesis tests for the causal mediation of semi-competing risks.","authors":"Yun-Lin Ho, Ju-Sheng Hong, Yen-Tsung Huang","doi":"10.1007/s10985-023-09595-7","DOIUrl":"10.1007/s10985-023-09595-7","url":null,"abstract":"<p><p>Analyzing the causal mediation of semi-competing risks has become important in medical research. Semi-competing risks refers to a scenario wherein an intermediate event may be censored by a primary event but not vice versa. Causal mediation analyses decompose the effect of an exposure on the primary outcome into an indirect (mediation) effect: an effect mediated through a mediator, and a direct effect: an effect not through the mediator. Here we proposed a model-based testing procedure to examine the indirect effect of the exposure on the primary event through the intermediate event. Under the counterfactual outcome framework, we defined a causal mediation effect using counting process. To assess statistical evidence for the mediation effect, we proposed two tests: an intersection-union test (IUT) and a weighted log-rank test (WLR). The test statistic was developed from a semi-parametric estimator of the mediation effect using a Cox proportional hazards model for the primary event and a series of logistic regression models for the intermediate event. We built a connection between the IUT and WLR. Asymptotic properties of the two tests were derived, and the IUT was determined to be a size [Formula: see text] test and statistically more powerful than the WLR. In numerical simulations, both the model-based IUT and WLR can properly adjust for confounding covariates, and the Type I error rates of the proposed methods are well protected, with the IUT being more powerful than the WLR. Our methods demonstrate the strongly significant effects of hepatitis B or C on the risk of liver cancer mediated through liver cirrhosis incidence in a prospective cohort study. The proposed method is also applicable to surrogate endpoint analyses in clinical trials.</p>","PeriodicalId":49908,"journal":{"name":"Lifetime Data Analysis","volume":" ","pages":"119-142"},"PeriodicalIF":1.3,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9529993","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 : 2024-01-01Epub Date: 2023-08-24DOI: 10.1007/s10985-023-09606-7
Iván Díaz, Katherine L Hoffman, Nima S Hejazi
{"title":"Causal survival analysis under competing risks using longitudinal modified treatment policies.","authors":"Iván Díaz, Katherine L Hoffman, Nima S Hejazi","doi":"10.1007/s10985-023-09606-7","DOIUrl":"10.1007/s10985-023-09606-7","url":null,"abstract":"<p><p>Longitudinal modified treatment policies (LMTP) have been recently developed as a novel method to define and estimate causal parameters that depend on the natural value of treatment. LMTPs represent an important advancement in causal inference for longitudinal studies as they allow the non-parametric definition and estimation of the joint effect of multiple categorical, ordinal, or continuous treatments measured at several time points. We extend the LMTP methodology to problems in which the outcome is a time-to-event variable subject to a competing event that precludes observation of the event of interest. We present identification results and non-parametric locally efficient estimators that use flexible data-adaptive regression techniques to alleviate model misspecification bias, while retaining important asymptotic properties such as [Formula: see text]-consistency. We present an application to the estimation of the effect of the time-to-intubation on acute kidney injury amongst COVID-19 hospitalized patients, where death by other causes is taken to be the competing event.</p>","PeriodicalId":49908,"journal":{"name":"Lifetime Data Analysis","volume":" ","pages":"213-236"},"PeriodicalIF":1.3,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10423108","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 : 2024-01-01Epub Date: 2023-08-12DOI: 10.1007/s10985-023-09607-6
Jeffrey Zhang, Dylan S Small
{"title":"Sensitivity Analysis for Observational Studies with Recurrent Events.","authors":"Jeffrey Zhang, Dylan S Small","doi":"10.1007/s10985-023-09607-6","DOIUrl":"10.1007/s10985-023-09607-6","url":null,"abstract":"<p><p>We conduct an observational study of the effect of sickle cell trait Haemoglobin AS (HbAS) on the hazard rate of malaria fevers in children. Assuming no unmeasured confounding, there is strong evidence that HbAS reduces the rate of malarial fevers. Since this is an observational study, however, the no unmeasured confounding assumption is strong. A sensitivity analysis considers how robust a conclusion is to a potential unmeasured confounder. We propose a new sensitivity analysis method for recurrent event data and apply it to the malaria study. We find that for the causal conclusion that HbAS is protective against malarial fevers to be overturned, the hypothesized unmeasured confounder must be as influential as all but one of the measured confounders.</p>","PeriodicalId":49908,"journal":{"name":"Lifetime Data Analysis","volume":" ","pages":"237-261"},"PeriodicalIF":1.3,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10353741","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 : 2023-10-01Epub Date: 2023-05-09DOI: 10.1007/s10985-023-09600-z
Yang Qu, Yu Cheng
{"title":"Volume under the ROC surface for high-dimensional independent screening with ordinal competing risk outcomes.","authors":"Yang Qu, Yu Cheng","doi":"10.1007/s10985-023-09600-z","DOIUrl":"10.1007/s10985-023-09600-z","url":null,"abstract":"<p><p>We propose a screening method for high-dimensional data with ordinal competing risk outcomes, which is time-dependent and model-free. Existing methods are designed for cause-specific variable screening and fail to evaluate how a biomarker is associated with multiple competing events simultaneously. The proposed method utilizes the Volume under the ROC surface (VUS), which measures the concordance between values of a biomarker and event status at certain time points and provides an overall evaluation of the discrimination capacity of a biomarker. We show that the VUS possesses the sure screening property, i.e., true important covariates can be retained with probability tending to one, and the size of the selected set can be bounded with high probability. The VUS appears to be a viable model-free screening metric as compared to some existing methods in simulation studies, and it is especially robust to data contamination. Through an analysis of breast-cancer gene-expression data, we illustrate the unique insights into the overall discriminatory capability provided by the VUS.</p>","PeriodicalId":49908,"journal":{"name":"Lifetime Data Analysis","volume":" ","pages":"735-751"},"PeriodicalIF":1.3,"publicationDate":"2023-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9432326","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 : 2023-10-01Epub Date: 2023-05-07DOI: 10.1007/s10985-023-09598-4
Hongkai Liang, Xiaoguang Wang, Yingwei Peng, Yi Niu
{"title":"Improving marginal hazard ratio estimation using quadratic inference functions.","authors":"Hongkai Liang, Xiaoguang Wang, Yingwei Peng, Yi Niu","doi":"10.1007/s10985-023-09598-4","DOIUrl":"10.1007/s10985-023-09598-4","url":null,"abstract":"<p><p>Clustered and multivariate failure time data are commonly encountered in biomedical studies and a marginal regression approach is often employed to identify the potential risk factors of a failure. We consider a semiparametric marginal Cox proportional hazards model for right-censored survival data with potential correlation. We propose to use a quadratic inference function method based on the generalized method of moments to obtain the optimal hazard ratio estimators. The inverse of the working correlation matrix is represented by the linear combination of basis matrices in the context of the estimating equation. We investigate the asymptotic properties of the regression estimators from the proposed method. The optimality of the hazard ratio estimators is discussed. Our simulation study shows that the estimator from the quadratic inference approach is more efficient than those from existing estimating equation methods whether the working correlation structure is correctly specified or not. Finally, we apply the model and the proposed estimation method to analyze a study of tooth loss and have uncovered new insights that were previously inaccessible using existing methods.</p>","PeriodicalId":49908,"journal":{"name":"Lifetime Data Analysis","volume":" ","pages":"823-853"},"PeriodicalIF":1.3,"publicationDate":"2023-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9470989","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 : 2023-10-01Epub Date: 2023-09-15DOI: 10.1007/s10985-023-09609-4
David Oakes
{"title":"Cox (1972): recollections and reflections.","authors":"David Oakes","doi":"10.1007/s10985-023-09609-4","DOIUrl":"10.1007/s10985-023-09609-4","url":null,"abstract":"<p><p>I present some personal memories and thoughts on Cox's 1972 paper \"Regression Models and Life-Tables\".</p>","PeriodicalId":49908,"journal":{"name":"Lifetime Data Analysis","volume":" ","pages":"699-708"},"PeriodicalIF":1.3,"publicationDate":"2023-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10235914","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 : 2023-10-01Epub Date: 2023-01-20DOI: 10.1007/s10985-022-09588-y
Bernard Rosner, Camden Bay, Robert J Glynn, Gui-Shuang Ying, Maureen G Maguire, Mei-Ling Ting Lee
{"title":"Estimation and testing for clustered interval-censored bivariate survival data with application using the semi-parametric version of the Clayton-Oakes model.","authors":"Bernard Rosner, Camden Bay, Robert J Glynn, Gui-Shuang Ying, Maureen G Maguire, Mei-Ling Ting Lee","doi":"10.1007/s10985-022-09588-y","DOIUrl":"10.1007/s10985-022-09588-y","url":null,"abstract":"<p><p>The Kaplan-Meier estimator is ubiquitously used to estimate survival probabilities for time-to-event data. It is nonparametric, and thus does not require specification of a survival distribution, but it does assume that the risk set at any time t consists of independent observations. This assumption does not hold for data from paired organ systems such as occur in ophthalmology (eyes) or otolaryngology (ears), or for other types of clustered data. In this article, we estimate marginal survival probabilities in the setting of clustered data, and provide confidence limits for these estimates with intra-cluster correlation accounted for by an interval-censored version of the Clayton-Oakes model. We develop a goodness-of-fit test for general bivariate interval-censored data and apply it to the proposed interval-censored version of the Clayton-Oakes model. We also propose a likelihood ratio test for the comparison of survival distributions between two groups in the setting of clustered data under the assumption of a constant between-group hazard ratio. This methodology can be used both for balanced and unbalanced cluster sizes, and also when the cluster size is informative. We compare our test to the ordinary log rank test and the Lin-Wei (LW) test based on the marginal Cox proportional Hazards model with robust standard errors obtained from the sandwich estimator. Simulation results indicate that the ordinary log rank test over-inflates type I error, while the proposed unconditional likelihood ratio test has appropriate type I error and higher power than the LW test. The method is demonstrated in real examples from the Sorbinil Retinopathy Trial, and the Age-Related Macular Degeneration Study. Raw data from these two trials are provided.</p>","PeriodicalId":49908,"journal":{"name":"Lifetime Data Analysis","volume":" ","pages":"854-887"},"PeriodicalIF":1.2,"publicationDate":"2023-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10614833/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9879574","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 : 2023-10-01Epub Date: 2023-05-20DOI: 10.1007/s10985-023-09602-x
Daewoo Pak, Jing Ning, Richard J Kryscio, Yu Shen
{"title":"Evaluation of the natural history of disease by combining incident and prevalent cohorts: application to the Nun Study.","authors":"Daewoo Pak, Jing Ning, Richard J Kryscio, Yu Shen","doi":"10.1007/s10985-023-09602-x","DOIUrl":"10.1007/s10985-023-09602-x","url":null,"abstract":"<p><p>The Nun study is a well-known longitudinal epidemiology study of aging and dementia that recruited elderly nuns who were not yet diagnosed with dementia (i.e., incident cohort) and who had dementia prior to entry (i.e., prevalent cohort). In such a natural history of disease study, multistate modeling of the combined data from both incident and prevalent cohorts is desirable to improve the efficiency of inference. While important, the multistate modeling approaches for the combined data have been scarcely used in practice because prevalent samples do not provide the exact date of disease onset and do not represent the target population due to left-truncation. In this paper, we demonstrate how to adequately combine both incident and prevalent cohorts to examine risk factors for every possible transition in studying the natural history of dementia. We adapt a four-state nonhomogeneous Markov model to characterize all transitions between different clinical stages, including plausible reversible transitions. The estimating procedure using the combined data leads to efficiency gains for every transition compared to those from the incident cohort data only.</p>","PeriodicalId":49908,"journal":{"name":"Lifetime Data Analysis","volume":" ","pages":"752-768"},"PeriodicalIF":1.2,"publicationDate":"2023-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10199741/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9509992","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 : 2023-10-01Epub Date: 2023-08-15DOI: 10.1007/s10985-023-09608-5
An-Min Tang, Nian-Sheng Tang, Dalei Yu
{"title":"Bayesian semiparametric joint model of multivariate longitudinal and survival data with dependent censoring.","authors":"An-Min Tang, Nian-Sheng Tang, Dalei Yu","doi":"10.1007/s10985-023-09608-5","DOIUrl":"10.1007/s10985-023-09608-5","url":null,"abstract":"<p><p>We consider a novel class of semiparametric joint models for multivariate longitudinal and survival data with dependent censoring. In these models, unknown-fashion cumulative baseline hazard functions are fitted by a novel class of penalized-splines (P-splines) with linear constraints. The dependence between the failure time of interest and censoring time is accommodated by a normal transformation model, where both nonparametric marginal survival function and censoring function are transformed to standard normal random variables with bivariate normal joint distribution. Based on a hybrid algorithm together with the Metropolis-Hastings algorithm within the Gibbs sampler, we propose a feasible Bayesian method to simultaneously estimate unknown parameters of interest, and to fit baseline survival and censoring functions. Intensive simulation studies are conducted to assess the performance of the proposed method. The use of the proposed method is also illustrated in the analysis of a data set from the International Breast Cancer Study Group.</p>","PeriodicalId":49908,"journal":{"name":"Lifetime Data Analysis","volume":" ","pages":"888-918"},"PeriodicalIF":1.3,"publicationDate":"2023-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10373335","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}