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  Subjects -> STATISTICS (Total: 130 journals)
Showing 1 - 151 of 151 Journals sorted alphabetically
Advances in Complex Systems     Hybrid Journal   (Followers: 10)
Advances in Data Analysis and Classification     Hybrid Journal   (Followers: 52)
Applied Categorical Structures     Hybrid Journal   (Followers: 5)
Argumentation et analyse du discours     Open Access   (Followers: 8)
Asian Journal of Mathematics & Statistics     Open Access   (Followers: 8)
AStA Advances in Statistical Analysis     Hybrid Journal   (Followers: 2)
Australian & New Zealand Journal of Statistics     Hybrid Journal   (Followers: 13)
Biometrical Journal     Hybrid Journal   (Followers: 9)
Biometrics     Hybrid Journal   (Followers: 52)
British Journal of Mathematical and Statistical Psychology     Full-text available via subscription   (Followers: 18)
Building Simulation     Hybrid Journal   (Followers: 2)
CHANCE     Hybrid Journal   (Followers: 5)
Communications in Statistics - Simulation and Computation     Hybrid Journal   (Followers: 9)
Communications in Statistics - Theory and Methods     Hybrid Journal   (Followers: 11)
Computational Statistics     Hybrid Journal   (Followers: 15)
Computational Statistics & Data Analysis     Hybrid Journal   (Followers: 36)
Current Research in Biostatistics     Open Access   (Followers: 8)
Decisions in Economics and Finance     Hybrid Journal   (Followers: 15)
Demographic Research     Open Access   (Followers: 14)
Engineering With Computers     Hybrid Journal   (Followers: 5)
Environmental and Ecological Statistics     Hybrid Journal   (Followers: 7)
ESAIM: Probability and Statistics     Open Access   (Followers: 4)
Extremes     Hybrid Journal   (Followers: 2)
Fuzzy Optimization and Decision Making     Hybrid Journal   (Followers: 8)
Geneva Papers on Risk and Insurance - Issues and Practice     Hybrid Journal   (Followers: 13)
Handbook of Numerical Analysis     Full-text available via subscription   (Followers: 4)
Handbook of Statistics     Full-text available via subscription   (Followers: 7)
IEA World Energy Statistics and Balances -     Full-text available via subscription   (Followers: 2)
International Journal of Computational Economics and Econometrics     Hybrid Journal   (Followers: 6)
International Journal of Quality, Statistics, and Reliability     Open Access   (Followers: 17)
International Journal of Stochastic Analysis     Open Access   (Followers: 2)
International Statistical Review     Hybrid Journal   (Followers: 12)
Journal of Algebraic Combinatorics     Hybrid Journal   (Followers: 3)
Journal of Applied Statistics     Hybrid Journal   (Followers: 20)
Journal of Biopharmaceutical Statistics     Hybrid Journal   (Followers: 24)
Journal of Business & Economic Statistics     Full-text available via subscription   (Followers: 40, SJR: 3.664, CiteScore: 2)
Journal of Combinatorial Optimization     Hybrid Journal   (Followers: 7)
Journal of Computational & Graphical Statistics     Full-text available via subscription   (Followers: 21)
Journal of Econometrics     Hybrid Journal   (Followers: 84)
Journal of Educational and Behavioral Statistics     Hybrid Journal   (Followers: 7)
Journal of Forecasting     Hybrid Journal   (Followers: 20)
Journal of Global Optimization     Hybrid Journal   (Followers: 6)
Journal of Mathematics and Statistics     Open Access   (Followers: 6)
Journal of Nonparametric Statistics     Hybrid Journal   (Followers: 6)
Journal of Probability and Statistics     Open Access   (Followers: 10)
Journal of Risk and Uncertainty     Hybrid Journal   (Followers: 33)
Journal of Statistical and Econometric Methods     Open Access   (Followers: 3)
Journal of Statistical Physics     Hybrid Journal   (Followers: 12)
Journal of Statistical Planning and Inference     Hybrid Journal   (Followers: 7)
Journal of Statistical Software     Open Access   (Followers: 16, SJR: 13.802, CiteScore: 16)
Journal of the American Statistical Association     Full-text available via subscription   (Followers: 73, SJR: 3.746, CiteScore: 2)
Journal of the Korean Statistical Society     Hybrid Journal  
Journal of the Royal Statistical Society Series C (Applied Statistics)     Hybrid Journal   (Followers: 37)
Journal of the Royal Statistical Society, Series A (Statistics in Society)     Hybrid Journal   (Followers: 28)
Journal of the Royal Statistical Society, Series B (Statistical Methodology)     Hybrid Journal   (Followers: 40)
Journal of Theoretical Probability     Hybrid Journal   (Followers: 3)
Journal of Time Series Analysis     Hybrid Journal   (Followers: 16)
Journal of Urbanism: International Research on Placemaking and Urban Sustainability     Hybrid Journal   (Followers: 26)
Law, Probability and Risk     Hybrid Journal   (Followers: 6)
Lifetime Data Analysis     Hybrid Journal   (Followers: 7)
Mathematical Methods of Statistics     Hybrid Journal   (Followers: 4)
Measurement Interdisciplinary Research and Perspectives     Hybrid Journal   (Followers: 1)
Metrika     Hybrid Journal   (Followers: 4)
Monthly Statistics of International Trade - Statistiques mensuelles du commerce international     Full-text available via subscription   (Followers: 3)
Multivariate Behavioral Research     Hybrid Journal   (Followers: 8)
Optimization Letters     Hybrid Journal   (Followers: 2)
Optimization Methods and Software     Hybrid Journal   (Followers: 5)
Oxford Bulletin of Economics and Statistics     Hybrid Journal   (Followers: 34)
Pharmaceutical Statistics     Hybrid Journal   (Followers: 15)
Queueing Systems     Hybrid Journal   (Followers: 7)
Research Synthesis Methods     Hybrid Journal   (Followers: 7)
Review of Economics and Statistics     Hybrid Journal   (Followers: 160)
Review of Socionetwork Strategies     Hybrid Journal  
Risk Management     Hybrid Journal   (Followers: 16)
Sankhya A     Hybrid Journal   (Followers: 3)
Scandinavian Journal of Statistics     Hybrid Journal   (Followers: 9)
Sequential Analysis: Design Methods and Applications     Hybrid Journal  
Significance     Hybrid Journal   (Followers: 7)
Sociological Methods & Research     Hybrid Journal   (Followers: 45)
SourceOECD Measuring Globalisation Statistics - SourceOCDE Mesurer la mondialisation - Base de donnees statistiques     Full-text available via subscription  
Stata Journal     Full-text available via subscription   (Followers: 8)
Statistica Neerlandica     Hybrid Journal   (Followers: 1)
Statistical Inference for Stochastic Processes     Hybrid Journal   (Followers: 3)
Statistical Methods and Applications     Hybrid Journal   (Followers: 6)
Statistical Methods in Medical Research     Hybrid Journal   (Followers: 30)
Statistical Modelling     Hybrid Journal   (Followers: 18)
Statistical Papers     Hybrid Journal   (Followers: 4)
Statistics & Probability Letters     Hybrid Journal   (Followers: 13)
Statistics and Computing     Hybrid Journal   (Followers: 14)
Statistics and Economics     Open Access  
Statistics in Medicine     Hybrid Journal   (Followers: 152)
Statistics: A Journal of Theoretical and Applied Statistics     Hybrid Journal   (Followers: 12)
Stochastic Models     Hybrid Journal   (Followers: 2)
Stochastics An International Journal of Probability and Stochastic Processes: formerly Stochastics and Stochastics Reports     Hybrid Journal   (Followers: 2)
Structural and Multidisciplinary Optimization     Hybrid Journal   (Followers: 12)
Teaching Statistics     Hybrid Journal   (Followers: 8)
Technology Innovations in Statistics Education (TISE)     Open Access   (Followers: 2)
TEST     Hybrid Journal   (Followers: 2)
The American Statistician     Full-text available via subscription   (Followers: 26)
The Canadian Journal of Statistics / La Revue Canadienne de Statistique     Hybrid Journal   (Followers: 10)
Wiley Interdisciplinary Reviews - Computational Statistics     Hybrid Journal   (Followers: 1)

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Similar Journals
Journal Cover
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  [2467 journals]
  • Bayesian Design of Clinical Trials Using Joint Cure Rate Models for
           Longitudinal and Time-to-Event Data

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      Abstract: Abstract For clinical trial design and analysis, there has been extensive work related to using joint models for longitudinal and time-to-event data without a cure fraction (i.e., when all patients are at risk for the event of interest), but comparatively little treatment has been given to design and analysis of clinical trials using joint models that incorporate a cure fraction. In this paper, we develop a Bayesian clinical trial design methodology focused on evaluating the treatment’s effect on a time-to-event endpoint using a promotion time cure rate model, where the longitudinal process is incorporated into the hazard model for the promotion times. A piecewise linear hazard model for the period after assessment of the longitudinal measure ends is proposed as an alternative to extrapolating the longitudinal trajectory. This may be advantageous in scenarios where the period of time from the end of longitudinal measurements until the end of observation is substantial. Inference for the time-to-event endpoint is based on a novel estimand which combines the treatment’s effect on the probability of cure and its effect on the promotion time distribution, mediated by the longitudinal outcome. We propose an approach for sample size determination such that the design has a high power and a well-controlled type I error rate with both operating characteristics defined from a Bayesian perspective. We demonstrate the methodology by designing a breast cancer clinical trial with a primary time-to-event endpoint where longitudinal outcomes are measured periodically during follow up.
      PubDate: 2022-11-10
       
  • Targeted maximum likelihood estimation for causal inference in survival
           and competing risks analysis

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      Abstract: Abstract Targeted maximum likelihood estimation (TMLE) provides a general methodology for estimation of causal parameters in presence of high-dimensional nuisance parameters. Generally, TMLE consists of a two-step procedure that combines data-adaptive nuisance parameter estimation with semiparametric efficiency and rigorous statistical inference obtained via a targeted update step. In this paper, we demonstrate the practical applicability of TMLE based causal inference in survival and competing risks settings where event times are not confined to take place on a discrete and finite grid. We focus on estimation of causal effects of time-fixed treatment decisions on survival and absolute risk probabilities, considering different univariate and multidimensional parameters. Besides providing a general guidance to using TMLE for survival and competing risks analysis, we further describe how the previous work can be extended with the use of loss-based cross-validated estimation, also known as super learning, of the conditional hazards. We illustrate the usage of the considered methods using publicly available data from a trial on adjuvant chemotherapy for colon cancer. R software code to implement all considered algorithms and to reproduce all analyses is available in an accompanying online appendix on Github.
      PubDate: 2022-11-07
       
  • Cox regression can be collapsible and Aalen regression can be
           non-collapsible

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      Abstract: Abstract It is well-known that the additive hazards model is collapsible, in the sense that when omitting one covariate from a model with two independent covariates, the marginal model is still an additive hazards model with the same regression coefficient or function for the remaining covariate. In contrast, for the proportional hazards model under the same covariate assumption, the marginal model is no longer a proportional hazards model and is not collapsible. These results, however, relate to the model specification and not to the regression parameter estimators. We point out that if covariates in risk sets at all event times are independent then both Cox and Aalen regression estimators are collapsible, in the sense that the parameter estimators in the full and marginal models are consistent for the same value. Vice-versa, if this assumption fails, then the estimates will change systematically both for Cox and Aalen regression. In particular, if the data are generated by an Aalen model with censoring independent of covariates both Cox and Aalen regression is collapsible, but if generated by a proportional hazards model neither estimators are. We will also discuss settings where survival times are generated by proportional hazards models with censoring patterns providing uncorrelated covariates and hence collapsible Cox and Aalen regression estimates. Furthermore, possible consequences for instrumental variable analyses are discussed.
      PubDate: 2022-10-21
       
  • A flexible parametric approach for analyzing arbitrarily censored data
           that are potentially subject to left truncation under the proportional
           hazards model

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      Abstract: Abstract The proportional hazards (PH) model is, arguably, the most popular model for the analysis of lifetime data arising from epidemiological studies, among many others. In such applications, analysts may be faced with censored outcomes and/or studies which institute enrollment criterion leading to left truncation. Censored outcomes arise when the event of interest is not observed but rather is known relevant to an observation time(s). Left truncated data occur in studies that exclude participants who have experienced the event prior to being enrolled in the study. If not accounted for, both of these features can lead to inaccurate inferences about the population under study. Thus, to overcome this challenge, herein we propose a novel unified PH model that can be used to accommodate both of these features. In particular, our approach can seamlessly analyze exactly observed failure times along with interval-censored observations, while aptly accounting for left truncation. To facilitate model fitting, an expectation–maximization algorithm is developed through the introduction of carefully structured latent random variables. To provide modeling flexibility, a monotone spline representation is used to approximate the cumulative baseline hazard function. The performance of our methodology is evaluated through a simulation study and is further illustrated through the analysis of two motivating data sets; one that involves child mortality in Nigeria and the other prostate cancer.
      PubDate: 2022-10-08
       
  • A uniformisation-driven algorithm for inference-related estimation of a
           phase-type ageing model

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      Abstract: Abstract We develop an efficient algorithm to compute the likelihood of the phase-type ageing model. The proposed algorithm uses the uniformisation method to stabilise the numerical calculation. It also utilises a vectorised formula to only calculate the necessary elements of the probability distribution. Our algorithm, with an error’s upper bound, could be adjusted easily to tackle the likelihood calculation of the Coxian models. Furthermore, we compare the speed and the accuracy of the proposed algorithm with those of the traditional method using the matrix exponential. Our algorithm is faster and more accurate than the traditional method in calculating the likelihood. Based on our experiments, we recommend using 20 sets of randomly-generated initial values for the optimisation to get a reliable estimate for which the evaluated likelihood is close to the maximum likelihood.
      PubDate: 2022-10-02
       
  • 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: 2022-10-01
      DOI: 10.1007/s10985-022-09559-3
       
  • 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: 2022-10-01
      DOI: 10.1007/s10985-022-09561-9
       
  • 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: 2022-10-01
      DOI: 10.1007/s10985-022-09558-4
       
  • Flexible two-piece distributions for right censored survival data

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      Abstract: Abstract 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).
      PubDate: 2022-09-20
      DOI: 10.1007/s10985-022-09574-4
       
  • A general class of promotion time cure rate models with a new biological
           interpretation

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      Abstract: Abstract Over the last decades, the challenges in survival models have been changing considerably and full probabilistic modeling is crucial in many medical applications. Motivated from a new biological interpretation of cancer metastasis, we introduce a general method for obtaining more flexible cure rate models. The proposal model extended the promotion time cure rate model. Furthermore, it includes several well-known models as special cases and defines many new special models. We derive several properties of the hazard function for the proposed model and establish mathematical relationships with the promotion time cure rate model. We consider a frequentist approach to perform inferences, and the maximum likelihood method is employed to estimate the model parameters. Simulation studies are conducted to evaluate its performance with a discussion of the obtained results. A real dataset from population-based study of incident cases of melanoma diagnosed in the state of São Paulo, Brazil, is discussed in detail.
      PubDate: 2022-09-16
      DOI: 10.1007/s10985-022-09575-3
       
  • Special issue dedicated to David Oakes

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      PubDate: 2022-09-06
      DOI: 10.1007/s10985-022-09572-6
       
  • Joint modeling of generalized scale-change models for recurrent event and
           failure time data

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      Abstract: Abstract 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.
      PubDate: 2022-09-06
      DOI: 10.1007/s10985-022-09573-5
       
  • Choice of time scale for analysis of recurrent events data

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      Abstract: Abstract 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.
      PubDate: 2022-08-15
      DOI: 10.1007/s10985-022-09569-1
       
  • Assessing dynamic covariate effects with survival data

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      Abstract: Abstract Dynamic (or varying) covariate effects often manifest meaningful physiological mechanisms underlying chronic diseases. However, a static view of covariate effects is typically adopted by standard approaches to evaluating disease prognostic factors, which can result in depreciation of some important disease markers. To address this issue, in this work, we take the perspective of globally concerned quantile regression, and propose a flexible testing framework suited to assess either constant or dynamic covariate effects. We study the powerful Kolmogorov–Smirnov (K–S) and Cramér–Von Mises (C–V) type test statistics and develop a simple resampling procedure to tackle their complicated limit distributions. We provide rigorous theoretical results, including the limit null distributions and consistency under a general class of alternative hypotheses of the proposed tests, as well as the justifications for the presented resampling procedure. Extensive simulation studies and a real data example demonstrate the utility of the new testing procedures and their advantages over existing approaches in assessing dynamic covariate effects.
      PubDate: 2022-08-13
      DOI: 10.1007/s10985-022-09571-7
       
  • Semiparametric single-index models for optimal treatment regimens with
           censored outcomes

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      Abstract: Abstract 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.
      PubDate: 2022-08-08
      DOI: 10.1007/s10985-022-09566-4
       
  • 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
      DOI: 10.1007/s10985-022-09570-8
       
  • 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
      DOI: 10.1007/s10985-022-09568-2
       
  • 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
      DOI: 10.1007/s10985-022-09562-8
       
  • 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
      DOI: 10.1007/s10985-022-09560-w
       
  • 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
      DOI: 10.1007/s10985-022-09563-7
       
 
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