Estimation of the joint survival function for three successive duration times under double truncation and right censoring

In incident cohort studies, it is common to include subjects who have experienced a certain event within a calendar time window. For all the included individuals, the time of the previous events is retrospectively confirmed and the occurrence of subsequent events is observed during the follow-up periods. During the follow-up periods, subjects may undergo three successive events. Since the second/third duration process becomes observable only if the first/second event has occurred, the data is subject to double truncation and right censoring. We consider two cases: the case when the first event time is subject to double truncation and the case when the second event time is subject to double truncation. Using the inverse-probability-weighted approach, we propose nonparametric and semiparametric estimators for the estimation of the joint survival function of three successive duration times. We establish the asymptotic properties of the proposed estimators and conduct a simulation study to investigate the finite sample properties of the proposed estimators.     

Interval estimation of the joint survival function for successive duration times under left truncation and right censoring

In incident cohort studies, survival data often include subjects who have had an initiate event at recruitment and may potentially experience two successive events (first and second) during the follow-up period. Since the second duration process becomes observable only if the first event has occurred, left truncation and dependent censoring arise if the two duration times are correlated. To confront the two potential sampling biases, we propose two inverse-probability-weighted (IPW) estimators for the estimation of the joint survival function of two successive duration times. One of them is similar to the estimator proposed by Chang and Tzeng [Nonparametric estimation of sojourn time distributions for truncated serial event data – a weight adjusted approach, Lifetime Data Anal. 12 (2006), pp. 53–67]. The other is the extension of the nonparametric estimator proposed by Wang and Wells [Nonparametric estimation of successive duration times under dependent censoring, Biometrika 85 (1998), pp. 561–572]. The weak convergence of both estimators are established. Furthermore, the delete-one jackknife and simple bootstrap methods are used to estimate standard deviations and construct interval estimators. A simulation study is conducted to compare the two IPW approaches.     

Nonparametric Estimation of Sojourn Time Distributions for Truncated Serial Event Data—a Weight-adjusted Approach

In follow-up studies, survival data often include subjects who have had a certain event at recruitment and may potentially experience a series of subsequent events during the follow-up period. This kind of survival data collected under a cross-sectional sampling criterion is called truncated serial event data. The outcome variables of interest in this paper are serial sojourn times between successive events. To analyze the sojourn times in truncated serial event data, we need to confront two potential sampling biases arising simultaneously from a sampling criterion and induced informative censoring. In this study, nonparametric estimation of the joint probability function of serial sojourn times is developed by using inverse probabilities of the truncation and censoring times as weight functions to accommodate these two sampling biases under various situations of truncation and censoring. Relevant statistical properties of the proposed estimators are also discussed. Simulation studies and two real data are presented to illustrate the proposed methods.     

Estimation of the joint survival function for successive duration times under double-truncation and right-censoring

ABSTRACT

In incident cohort studies, survival data often include subjects who have had an initiate event at recruitment and may potentially experience two successive events (first and second) during the follow-up period. When disease registries or surveillance systems collect data based on incidence occurring within a specific calendar time interval, the initial event is usually subject to double truncation. Furthermore, since the second duration process is observable only if the first event has occurred, double truncation and dependent censoring arise. In this article, under the two sampling biases with an unspecified distribution of truncation variables, we propose a nonparametric estimator of the joint survival function of two successive duration times using the inverse-probability-weighted (IPW) approach. The consistency of the proposed estimator is established. Based on the estimated marginal survival functions, we also propose a two-stage estimation procedure for estimating the parameters of copula model. The bootstrap method is used to construct confidence interval. Numerical studies demonstrate that the proposed estimation approaches perform well with moderate sample sizes.     

Nonparametric analysis of recurrent gap time data

Abstract

Recurrent event data are frequently encountered in longitudinal studies. In many applications, the times between successive recurrent events (gap times) are often of interest and lead to problems that have received much attention recently. In this article, using the approach of inverse probability-of-censoring weights (IPCW), we propose nonparametric estimators for the estimation of the bivariate distribution and survival functions for gap times of recurrent event data. We also consider the estimation of Kendall’s tau for two gap times by expressing it as an integral functional of the bivariate survival function. The asymptotic properties of the proposed estimators are established. Simulation studies are conducted to investigate their finite sample performance.     

Non parametric analysis of gap times for bivariate recurrent event data with cure fraction

Recurrent event data often arise in longitudinal studies. In many applications, subjects may experience two different types of events alternatively over time or a pair of subjects may experience recurrent events of the same type. Medical advances have made it possible for some patients to be cured such that the disease of interest does not recur. In this article, we consider non parametric analysis of bivariate recurrent event data with cure fraction. Using the inverse-probability weighted (IPW) approach, we propose non parametric estimators for the proportion of cured patients and for the joint distribution functions of bivariate recurrence times of the uncured ones. The asymptotic properties of the proposed estimators are established. Simulation study indicates that the proposed estimators perform well in finite samples.     

SEMIPARAMETRIC ADDITIVE RISKS REGRESSION FOR TWO-STAGE DESIGN SURVIVAL STUDIES

In this article we study a semiparametric additive risks model (McKeague and Sasieni (1994)) for two-stage design survival data where accurate information is available only on second stage subjects, a subset of the first stage study. We derive two-stage estimators by combining data from both stages. Large sample inferences are developed. As a by-product, we also obtain asymptotic properties of the single stage estimators of McKeague and Sasieni (1994) when the semiparametric additive risks model is misspecified. The proposed two-stage estimators are shown to be asymptotically more efficient than the second stage estimators. They also demonstrate smaller bias and variance for finite samples. The developed methods are illustrated using small intestine cancer data from the SEER (Surveillance, Epidemiology, and End Results) Program.     

An additive marginal regression model for clustered recurrent event in the presence of a terminal event

In this article, an additive rate model is proposed for clustered recurrent event with a terminal event. The subjects are clustered by some property. For the clustered subjects, the recurrent event is precluded by the death. An estimating equation is developed for the model parameter and the baseline rate function. The asymptotic properties of the resulting estimators are established. In addition, a goodness-of-fit test is presented to assess the adequacy of the model. The finite-sample behavior of the proposed estimators is evaluated through simulation studies, and an application to a bladder cancer data is illustrated.     

Estimating treatment effects on the marginal recurrent event mean in the presence of a terminating event

In biomedical studies where the event of interest is recurrent (e.g., hospitalization), it is often the case that the recurrent event sequence is subject to being stopped by a terminating event (e.g., death). In comparing treatment options, the marginal recurrent event mean is frequently of interest. One major complication in the recurrent/terminal event setting is that censoring times are not known for subjects observed to die, which renders standard risk set based methods of estimation inapplicable. We propose two semiparametric methods for estimating the difference or ratio of treatment-specific marginal mean numbers of events. The first method involves imputing unobserved censoring times, while the second methods uses inverse probability of censoring weighting. In each case, imbalances in the treatment-specific covariate distributions are adjusted out through inverse probability of treatment weighting. After the imputation and/or weighting, the treatment-specific means (then their difference or ratio) are estimated nonparametrically. Large-sample properties are derived for each of the proposed estimators, with finite sample properties assessed through simulation. The proposed methods are applied to kidney transplant data.     

Simultaneous Modelling of Survival and Longitudinal Data with an Application to Repeated Quality of Life Measures

In biomedical studies, interest often focuses on the relationship between patients characteristics or some risk factors and both quality of life and survival time of subjects under study. In this paper, we propose a simultaneous modelling of both quality of life and survival time using the observed covariates. Moreover, random effects are introduced into the simultaneous models to account for dependence between quality of life and survival time due to unobserved factors. EM algorithms are used to derive the point estimates for the parameters in the proposed model and profile likelihood function is used to estimate their variances. The asymptotic properties are established for our proposed estimators. Finally, simulation studies are conducted to examine the finite-sample properties of the proposed estimators and a liver transplantation data set is analyzed to illustrate our approaches.     

A composite class of estimators using scrambled response mechanism for sensitive population mean in successive sampling

This paper considers the problem of estimation of population mean of a sensitive characteristics using non-sensitive auxiliary variable at current move in two move successive sampling. The proposed estimator is studied under five different scrambled response models. Various estimators have been elaborated to be the member of the proposed class of estimators. The properties of the proposed estimators have been analysed. Many estimators belonging to the proposed class have been explored under five scrambled response models. In order to identify the scrambled model effect, the proposed composite class of estimators is compared to the direct methods. Respondents privacy protection have also been elaborated under different models. Theoretical results are supplemented with numerical demonstrations using real data. Simulation has been carried out to show the applicability of proposed estimators and hence suitable recommendations are forwarded.     

Recurrent Events Analysis in the Presence of Terminal Event and Zero-recurrence Subjects

Recurrent event data are often encountered in longitudinal follow-up studies related to biomedical science, econometrics, reliability, and demography. In many situations, a terminal event such as death can happen during the follow-up period that precludes further recurrences. In this article, we will review some existing models for recurrent event with information censoring, and then extend them to allow zero-recurrence subjects as well as a terminal event. Estimating equations and partial likelihood are employed to estimate the coefficients of covariates, accumulative rate functions and the proportions of zero-recurrence subjects. The large-sample properties ofthe estimators are established as well. Simulations are performed to evaluate the estimationprocedure and an example of application on a set of migration data is provided to illustrateour proposed models and methods.     

A Marginal Additive Rates Model for Recurrent Event Data with a Terminal Event

Recurrent events data with a terminal event often arise in many longitudinal studies. Most of existing models assume multiplicative covariate effects and model the conditional recurrent event rate given survival. In this article, we propose a marginal additive rates model for recurrent events with a terminal event, and develop two procedures for estimating the model parameters. The asymptotic properties of the resulting estimators are established. In addition, some numerical procedures are presented for model checking. The finite-sample behavior of the proposed methods is examined through simulation studies, and an application to a bladder cancer study is also illustrated.     

Proportional hazards regression in the presence of missing study eligibility information

We consider the study of censored survival times in the situation where the available data consist of both eligible and ineligible subjects, and information distinguishing the two groups is sometimes missing. A complete-case analysis in this context would use only subjects known to be eligible, resulting in inefficient and potentially biased estimators. We propose a two-step procedure which resembles the EM algorithm but is computationally much faster. In the first step, one estimates the conditional expectation of the missing eligibility indicators given the observed data using a logistic regression based on the complete cases (i.e., subjects with non-missing eligibility indicator). In the second step, maximum likelihood estimators are obtained from a weighted Cox proportional hazards model, with the weights being either observed eligibility indicators or estimated conditional expectations thereof. Under ignorable missingness, the estimators from the second step are proven to be consistent and asymptotically normal, with explicit variance estimators. We demonstrate through simulation that the proposed methods perform well for moderate sized samples and are robust in the presence of eligibility indicators that are missing not at random. The proposed procedure is more efficient and more robust than the complete case analysis and, unlike the EM algorithm, does not require time-consuming iteration. Although the proposed methods are applicable generally, they would be most useful for large data sets (e.g., administrative data), for which the computational savings outweigh the price one has to pay for making various approximations in avoiding iteration. We apply the proposed methods to national kidney transplant registry data.     

The analysis of multivariate recurrent events with partially missing event types

In many clinical studies, subjects are at risk of experiencing more than one type of potentially recurrent event. In some situations, however, the occurrence of an event is observed, but the specific type is not determined. We consider the analysis of this type of incomplete data when the objectives are to summarize features of conditional intensity functions and associated treatment effects, and to study the association between different types of event. Here we describe a likelihood approach based on joint models for the multi-type recurrent events where parameter estimation is obtained from a Monte-Carlo EM algorithm. Simulation studies show that the proposed method gives unbiased estimators for regression coefficients and variance–covariance parameters, and the coverage probabilities of confidence intervals for regression coefficients are close to the nominal level. When the distribution of the frailty variable is misspecified, the method still provides estimators of the regression coefficients with good properties. The proposed method is applied to a motivating data set from an asthma study in which exacerbations were to be sub-typed by cellular analysis of sputum samples as eosinophilic or non-eosinophilic.     

Semiparametric proportional means model for marker data contingent on recurrent event

In many biomedical studies with recurrent events, some markers can only be measured when events happen. For example, medical cost attributed to hospitalization can only incur when patients are hospitalized. Such marker data are contingent on recurrent events. In this paper, we present a proportional means model for modelling the markers using the observed covariates contingent on the recurrent event. We also model the recurrent event via a marginal rate model. Estimating equations are constructed to derive the point estimators for the parameters in the proposed models. The estimators are shown to be asymptotically normal. Simulation studies are conducted to examine the finite-sample properties of the proposed estimators and the proposed method is applied to a data set from the Vitamin A Community Trial.     

Use of multiauxiliary variables to cope with non response in estimation of current population mean in two-occasion successive sampling

ABSTRACT

The present work utilizes the information on multiauxiliary variables to neutralize the negative effect of non response in estimation of current population mean in two-occasion successive sampling. Generalized exponential type estimators have been proposed for the situation when non response occurs at both occasion or at first occasion or at current (second) occasion in two-occasion successive sampling. Properties of the proposed estimators have studied and empirical studies are carried out to justify the preposition of estimators. Suitable recommendations have been made.     

An effective estimation strategy for population mean under random non-response situations in two-phase successive sampling

This paper presents a modified exponential type estimation strategy for the current population mean in the presence of random non-response situations in two-occasion successive sampling under two-phase set-up. The properties of the proposed estimators have been examined with the assumption that numbers of sampling units follow a distribution due to random non-response. The performances of the proposed estimators are compared with the estimators designated for the complete response situations. Empirical studies are carried out to show the dominance nature of the proposed estimators over the estimator defined for complete response situations. Appropriate recommendations have been made to the survey practitioners/researchers for their real-life practical applications.     

Nonparametric estimators for a survivor function of paired recurrent events

Recurrent event data arise in longitudinal studies where each study subject may experience multiple events during the follow-up. In many situations in survival studies, pairs of individuals can potentially experience recurrent events. The analysis of such data is not straightforward as it involves two kinds of dependences, namely, dependence between the individuals in the same pair and dependence among a sequence of pairs. In the present paper, we introduce a new stochastic model for the analysis of such recurrent event data. Nonparametric estimators for a bivariate survivor function are developed. Asymptotic properties of the estimators are discussed. Simulation studies are carried out to assess the finite sample properties of the estimator. We illustrate the procedure with real life data on eye disease.     

Evaluating bias correction in weighted proportional hazards regression

Often in observational studies of time to an event, the study population is a biased (i.e., unrepresentative) sample of the target population. In the presence of biased samples, it is common to weight subjects by the inverse of their respective selection probabilities. Pan and Schaubel (Can J Stat 36:111–127, 2008) recently proposed inference procedures for an inverse selection probability weighted (ISPW) Cox model, applicable when selection probabilities are not treated as fixed but estimated empirically. The proposed weighting procedure requires auxiliary data to estimate the weights and is computationally more intense than unweighted estimation. The ignorability of sample selection process in terms of parameter estimators and predictions is often of interest, from several perspectives: e.g., to determine if weighting makes a significant difference to the analysis at hand, which would in turn address whether the collection of auxiliary data is required in future studies; to evaluate previous studies which did not correct for selection bias. In this article, we propose methods to quantify the degree of bias corrected by the weighting procedure in the partial likelihood and Breslow-Aalen estimators. Asymptotic properties of the proposed test statistics are derived. The finite-sample significance level and power are evaluated through simulation. The proposed methods are then applied to data from a national organ failure registry to evaluate the bias in a post-kidney transplant survival model.