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  Subjects -> STATISTICS (Total: 130 journals)
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Lifetime Data Analysis
Journal Prestige (SJR): 0.985
Citation Impact (citeScore): 1
Number of Followers: 7  
 
  Hybrid Journal Hybrid journal (It can contain Open Access articles)
ISSN (Print) 1572-9249 - ISSN (Online) 1380-7870
Published by Springer-Verlag Homepage  [2469 journals]
  • Median regression models for clustered, interval-censored survival data -
           An application to prostate surgery study

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      Abstract: Abstract 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.
      PubDate: 2022-08-07
       
  • Double bias correction for high-dimensional sparse additive hazards
           regression with covariate measurement errors

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      Abstract: Abstract We propose an inferential procedure for additive hazards regression with high-dimensional survival data, where the covariates are prone to measurement errors. We develop a double bias correction method by first correcting the bias arising from measurement errors in covariates through an estimating function for the regression parameter. By adopting the convex relaxation technique, a regularized estimator for the regression parameter is obtained by elaborately designing a feasible loss based on the estimating function, which is solved via linear programming. Using the Neyman orthogonality, we propose an asymptotically unbiased estimator which further corrects the bias caused by the convex relaxation and regularization. We derive the convergence rate of the proposed estimator and establish the asymptotic normality for the low-dimensional parameter estimator and the linear combination thereof, accompanied with a consistent estimator for the variance. Numerical experiments are carried out on both simulated and real datasets to demonstrate the promising performance of the proposed double bias correction method.
      PubDate: 2022-07-22
       
  • Semiparametric regression analysis of doubly-censored data with
           applications to incubation period estimation

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      Abstract: Abstract The incubation period is a key characteristic of an infectious disease. In the outbreak of a novel infectious disease, accurate evaluation of the incubation period distribution is critical for designing effective prevention and control measures . Estimation of the incubation period distribution based on limited information from retrospective inspection of infected cases is highly challenging due to censoring and truncation. In this paper, we consider a semiparametric regression model for the incubation period and propose a sieve maximum likelihood approach for estimation based on the symptom onset time, travel history, and basic demographics of reported cases. The approach properly accounts for the pandemic growth and selection bias in data collection. We also develop an efficient computation method and establish the asymptotic properties of the proposed estimators. We demonstrate the feasibility and advantages of the proposed methods through extensive simulation studies and provide an application to a dataset on the outbreak of COVID-19.
      PubDate: 2022-07-13
       
  • On logistic regression with right censored data, with or without competing
           risks, and its use for estimating treatment effects

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      Abstract: Abstract Simple logistic regression can be adapted to deal with right-censoring by inverse probability of censoring weighting (IPCW). We here compare two such IPCW approaches, one based on weighting the outcome, the other based on weighting the estimating equations. We study the large sample properties of the two approaches and show that which of the two weighting methods is the most efficient depends on the censoring distribution. We show by theoretical computations that the methods can be surprisingly different in realistic settings. We further show how to use the two weighting approaches for logistic regression to estimate causal treatment effects, for both observational studies and randomized clinical trials (RCT). Several estimators for observational studies are compared and we present an application to registry data. We also revisit interesting robustness properties of logistic regression in the context of RCTs, with a particular focus on the IPCW weighting. We find that these robustness properties still hold when the censoring weights are correctly specified, but not necessarily otherwise.
      PubDate: 2022-07-07
       
  • A new approach to estimation of the proportional hazards model based on
           interval-censored data with missing covariates

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      Abstract: Abstract This paper discusses the fitting of the proportional hazards model to interval-censored failure time data with missing covariates. Many authors have discussed the problem when complete covariate information is available or the missing is completely at random. In contrast to this, we will focus on the situation where the missing is at random. For the problem, a sieve maximum likelihood estimation approach is proposed with the use of I-spline functions to approximate the unknown cumulative baseline hazard function in the model. For the implementation of the proposed method, we develop an EM algorithm based on a two-stage data augmentation. Furthermore, we show that the proposed estimators of regression parameters are consistent and asymptotically normal. The proposed approach is then applied to a set of the data concerning Alzheimer Disease that motivated this study.
      PubDate: 2022-07-01
       
  • Accounting for delayed entry into observational studies and clinical
           trials: length-biased sampling and restricted mean survival time

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      Abstract: Abstract Individuals in many observational studies and clinical trials for chronic diseases are enrolled well after onset or diagnosis of their disease. Times to events of interest after enrollment are therefore residual or left-truncated event times. Individuals entering the studies have disease that has advanced to varying extents. Moreover, enrollment usually entails probability sampling of the study population. Finally, event times over a short to moderate time horizon are often of interest in these investigations, rather than more speculative and remote happenings that lie beyond the study period. This research report looks at the issue of delayed entry into these kinds of studies and trials. Time to event for an individual is modelled as a first hitting time of an event threshold by a latent disease process, which is taken to be a Wiener process. It is emphasized that recruitment into these studies often involves length-biased sampling. The requisite mathematics for this kind of sampling and delayed entry are presented, including explicit formulas needed for estimation and inference. Restricted mean survival time (RMST) is taken as the clinically relevant outcome measure. Exact parametric formulas for this measure are derived and presented. The results are extended to settings that involve study covariates using threshold regression methods. Methods adapted for clinical trials are presented. An extensive case illustration for a clinical trial setting is then presented to demonstrate the methods, the interpretation of results, and the harvesting of useful insights. The closing discussion covers a number of important issues and concepts.
      PubDate: 2022-07-01
       
  • Inference for transition probabilities in non-Markov multi-state models

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      Abstract: Abstract 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.
      PubDate: 2022-06-28
       
  • Model selection among Dimension-Reduced generalized Cox models

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      Abstract: Abstract Conventional semiparametric hazards regression models rely on the specification of particular model formulations, such as proportional-hazards feature and single-index structures. Instead of checking these modeling assumptions one-by-one, we proposed a class of dimension-reduced generalized Cox models, and then a consistent model selection procedure among this class to select covariates with proportional-hazards feature and a proper model formulation for non-proportional-hazards covariates. In this class, the non-proportional-hazards covariates are treated in a nonparametric manner, and a partial sufficient dimension reduction is introduced to reduce the curse of dimensionality. A semiparametric efficient estimation is proposed to estimate these models. Based on the proposed estimation, we further constructed a cross-validation type criterion to consistently select the correct model among this class. Most importantly, this class of hazards regression models contains the fully nonparametric hazards regression model as the most saturated submodel, and hence no further model diagnosis is required. Overall speaking, this model selection approach is more effective than performing a sequence of conventional model checking. The proposed method is illustrated by simulation studies and a data example.
      PubDate: 2022-06-28
       
  • Semi-supervised approach to event time annotation using longitudinal
           electronic health records

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      Abstract: Abstract Large clinical datasets derived from insurance claims and electronic health record (EHR) systems are valuable sources for precision medicine research. These datasets can be used to develop models for personalized prediction of risk or treatment response. Efficiently deriving prediction models using real world data, however, faces practical and methodological challenges. Precise information on important clinical outcomes such as time to cancer progression are not readily available in these databases. The true clinical event times typically cannot be approximated well based on simple extracts of billing or procedure codes. Whereas, annotating event times manually is time and resource prohibitive. In this paper, we propose a two-step semi-supervised multi-modal automated time annotation (MATA) method leveraging multi-dimensional longitudinal EHR encounter records. In step I, we employ a functional principal component analysis approach to estimate the underlying intensity functions based on observed point processes from the unlabeled patients. In step II, we fit a penalized proportional odds model to the event time outcomes with features derived in step I in the labeled data where the non-parametric baseline function is approximated using B-splines. Under regularity conditions, the resulting estimator of the feature effect vector is shown as root-n consistent. We demonstrate the superiority of our approach relative to existing approaches through simulations and a real data example on annotating lung cancer recurrence in an EHR cohort of lung cancer patients from Veteran Health Administration.
      PubDate: 2022-06-26
       
  • Screening for chronic diseases: optimizing lead time through balancing
           prescribed frequency and individual adherence

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      Abstract: Abstract Screening for chronic diseases, such as cancer, is an important public health priority, but traditionally only the frequency or rate of screening has received attention. In this work, we study the importance of adhering to recommended screening policies and develop new methodology to better optimize screening policies when adherence is imperfect. We consider a progressive disease model with four states (healthy, undetectable preclinical, detectable preclinical, clinical), and overlay this with a stochastic screening–behavior model using the theory of renewal processes that allows us to capture imperfect adherence to screening programs in a transparent way. We show that decreased adherence leads to reduced efficacy of screening programs, quantified here using elements of the lead time distribution (i.e., the time between screening diagnosis and when diagnosis would have occurred clinically in the absence of screening). Under the assumption of an inverse relationship between prescribed screening frequency and individual adherence, we show that the optimal screening frequency generally decreases with increasing levels of non-adherence. We apply this model to an example in breast cancer screening, demonstrating how accounting for imperfect adherence affects the recommended screening frequency.
      PubDate: 2022-06-24
       
  • Bias correction via outcome reassignment for cross-sectional data with
           binary disease outcome

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      Abstract: Abstract 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.
      PubDate: 2022-06-24
       
  • On the targets of inference with multivariate failure time data

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      Abstract: Abstract 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.
      PubDate: 2022-06-21
       
  • Marker-dependent observation and carry-forward of internal covariates in
           Cox regression

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      Abstract: Abstract 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.
      PubDate: 2022-06-20
       
  • Optimum test planning for heterogeneous inverse Gaussian processes

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      Abstract: Abstract The heterogeneous inverse Gaussian (IG) process is one of the most popular and most considered degradation models for highly reliable products. One difficulty with heterogeneous IG processes is the lack of analytic expressions for the Fisher information matrix (FIM). Thus, it is a challenge to find an optimum test plan using any information-based criteria with decision variables such as the termination time, the number of measurements and sample size. In this article, the FIM of an IG process with random slopes can be derived explicitly in an algebraic expression to reduce uncertainty caused by the numerical approximation. The D- and V-optimum test plans with/without a cost constraint can be obtained by using a profile optimum plan. Sensitivity analysis is studied to elucidate how optimum planning is influenced by the experimental costs and planning values of the model parameters. The theoretical results are illustrated by numerical simulation and case studies. Simulations, technical derivations and auxiliary formulae are available online as supplementary materials.
      PubDate: 2022-06-13
       
  • Longitudinal mediation analysis of time-to-event endpoints in the presence
           of competing risks

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      Abstract: Abstract This proposal is motivated by an analysis of the English Longitudinal Study of Ageing (ELSA), which aims to investigate the role of loneliness in explaining the negative impact of hearing loss on dementia. The methodological challenges that complicate this mediation analysis include the use of a time-to-event endpoint subject to competing risks, as well as the presence of feedback relationships between the mediator and confounders that are both repeatedly measured over time. To account for these challenges, we introduce path-specific effect proportional (cause-specific) hazard models. These extend marginal structural proportional (cause-specific) hazard models to enable effect decomposition on either the cause-specific hazard ratio scale or the cumulative incidence function scale. We show that under certain ignorability assumptions, the path-specific direct and indirect effects indexing this model are identifiable from the observed data. We next propose an inverse probability weighting approach to estimate these effects. On the ELSA data, this approach reveals little evidence that the total effect of hearing loss on dementia is mediated through the feeling of loneliness, with a non-statistically significant indirect effect equal to 1.01 (hazard ratio (HR) scale; 95% confidence interval (CI) 0.99 to 1.05).
      PubDate: 2022-06-02
       
  • Privacy-preserving estimation of an optimal individualized treatment rule:
           a case study in maximizing time to severe depression-related outcomes

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      Abstract: Abstract Estimating individualized treatment rules—particularly in the context of right-censored outcomes—is challenging because the treatment effect heterogeneity of interest is often small, thus difficult to detect. While this motivates the use of very large datasets such as those from multiple health systems or centres, data privacy may be of concern with participating data centres reluctant to share individual-level data. In this case study on the treatment of depression, we demonstrate an application of distributed regression for privacy protection used in combination with dynamic weighted survival modelling (DWSurv) to estimate an optimal individualized treatment rule whilst obscuring individual-level data. In simulations, we demonstrate the flexibility of this approach to address local treatment practices that may affect confounding, and show that DWSurv retains its double robustness even when performed through a (weighted) distributed regression approach. The work is motivated by, and illustrated with, an analysis of treatment for unipolar depression using the United Kingdom’s Clinical Practice Research Datalink.
      PubDate: 2022-05-02
      DOI: 10.1007/s10985-022-09554-8
       
  • Mixture survival trees for cancer risk classification

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      Abstract: Abstract In oncology studies, it is important to understand and characterize disease heterogeneity among patients so that patients can be classified into different risk groups and one can identify high-risk patients at the right time. This information can then be used to identify a more homogeneous patient population for developing precision medicine. In this paper, we propose a mixture survival tree approach for direct risk classification. We assume that the patients can be classified into a pre-specified number of risk groups, where each group has distinct survival profile. Our proposed tree-based methods are devised to estimate latent group membership using an EM algorithm. The observed data log-likelihood function is used as the splitting criterion in recursive partitioning. The finite sample performance is evaluated by extensive simulation studies and the proposed method is illustrated by a case study in breast cancer.
      PubDate: 2022-04-29
      DOI: 10.1007/s10985-022-09552-w
       
  • A boosting first-hitting-time model for survival analysis in
           high-dimensional settings

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      Abstract: Abstract In this paper we propose a boosting algorithm to extend the applicability of a first hitting time model to high-dimensional frameworks. Based on an underlying stochastic process, first hitting time models do not require the proportional hazards assumption, hardly verifiable in the high-dimensional context, and represent a valid parametric alternative to the Cox model for modelling time-to-event responses. First hitting time models also offer a natural way to integrate low-dimensional clinical and high-dimensional molecular information in a prediction model, that avoids complicated weighting schemes typical of current methods. The performance of our novel boosting algorithm is illustrated in three real data examples.
      PubDate: 2022-04-27
      DOI: 10.1007/s10985-022-09553-9
       
  • Bayesian nonparametric dynamic hazard rates in evolutionary life tables

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      Abstract: Abstract In the study of life tables the random variable of interest is usually assumed discrete since mortality rates are studied for integer ages. In dynamic life tables a time domain is included to account for the evolution effect of the hazard rates in time. In this article we follow a survival analysis approach and use a nonparametric description of the hazard rates. We construct a discrete time stochastic processes that reflects dependence across age as well as in time. This process is used as a bayesian nonparametric prior distribution for the hazard rates for the study of evolutionary life tables. Prior properties of the process are studied and posterior distributions are derived. We present a simulation study, with the inclusion of right censored observations, as well as a real data analysis to show the performance of our model.
      PubDate: 2022-03-17
      DOI: 10.1007/s10985-022-09551-x
       
  • Bayesian penalized Buckley-James method for high dimensional bivariate
           censored regression models

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      Abstract: Abstract For high dimensional gene expression data, one important goal is to identify a small number of genes that are associated with progression of the disease or survival of the patients. In this paper, we consider the problem of variable selection for multivariate survival data. We propose an estimation procedure for high dimensional accelerated failure time (AFT) models with bivariate censored data. The method extends the Buckley-James method by minimizing a penalized \(L_2\) loss function with a penalty function induced from a bivariate spike-and-slab prior specification. In the proposed algorithm, censored observations are imputed using the Kaplan-Meier estimator, which avoids a parametric assumption on the error terms. Our empirical studies demonstrate that the proposed method provides better performance compared to the alternative procedures designed for univariate survival data regardless of whether the true events are correlated or not, and conceptualizes a formal way of handling bivariate survival data for AFT models. Findings from the analysis of a myeloma clinical trial using the proposed method are also presented.
      PubDate: 2022-03-03
      DOI: 10.1007/s10985-022-09549-5
       
 
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