Analysis of transformation models with right-truncated data

In many applications, statistical data are frequently observed subject to a retrospective sampling criterion resulting in right-truncated data. In this article, a general class of semiparametric transformation models that include proportional hazards model and proportional odds model as special cases is studied for the analysis of right-truncated data. We proposed two estimators for regression coefficients. The first estimator is based on martingale estimating equations. The second estimator is based on the conditional likelihood function given the truncation times. The asymptotic properties of both estimators are derived. The finite sample performance is examined through a simulation study.     

Pseudo MLE for semiparametric transformation model with doubly truncated data

In this article, we consider the efficient estimation of the semiparametric transformation model with doubly truncated data. We propose a two-step approach for obtaining the pseudo maximum likelihood estimators (PMLE) of regression parameters. In the first step, the truncation time distribution is estimated by the nonparametric maximum likelihood estimator (Shen, 2010a) when the distribution function $K$ of the truncation time is unspecified or by the conditional maximum likelihood estimator (Bilker and Wang, 1996) when $K$ is parameterized. In the second step, using the pseudo complete-data likelihood function with the estimated distribution of truncation time, we propose expectation–maximization algorithms for obtaining the PMLE. We establish the consistency of the PMLE. The simulation study indicates that the PMLE performs well in finite samples. The proposed method is illustrated using an AIDS data set.     

Imputation for semiparametric transformation models with biased-sampling data

Widely recognized in many fields including economics, engineering, epidemiology, health sciences, technology and wildlife management, length-biased sampling generates biased and right-censored data but often provide the best information available for statistical inference. Different from traditional right-censored data, length-biased data have unique aspects resulting from their sampling procedures. We exploit these unique aspects and propose a general imputation-based estimation method for analyzing length-biased data under a class of flexible semiparametric transformation models. We present new computational algorithms that can jointly estimate the regression coefficients and the baseline function semiparametrically. The imputation-based method under the transformation model provides an unbiased estimator regardless whether the censoring is independent or not on the covariates. We establish large-sample properties using the empirical processes method. Simulation studies show that under small to moderate sample sizes, the proposed procedure has smaller mean square errors than two existing estimation procedures. Finally, we demonstrate the estimation procedure by a real data example.     

Analysing competing risks data with transformation models

We present a flexible class of marginal models for the cumulative incidence function. The semiparametric transformation model is utilized in a decomposition for the marginal failure probabilities which extends previous work on Farewell's cure model. Novel estimation, inference and prediction procedures are developed, with large sample properties derived from the theory of martingales and U-statistics. A small simulation study demonstrates that the methods are appropriate for practical use. The methods are illustrated with a thorough analysis of a prostate cancer clinical trial. Simple graphical displays are used to check for the goodness of fit.     

Nonparametric estimation of the ROC curve for length-biased and right-censored data

Abstract

ROC curve is a fundamental evaluation tool in medical researches and survival analysis. The estimation of ROC curve has been studied extensively with complete data and right-censored survival data. However, these methods are not suitable to analyze the length-biased and right-censored data. Since this kind of data includes the auxiliary information that truncation time and residual time share the same distribution, the two new estimators for the ROC curve are proposed by taking into account this auxiliary information to improve estimation efficiency. Numerical simulation studies with different assumed cases and real data analysis are conducted.     

Estimation in the Cox proportional hazards model with doubly censored and truncated data

In longitudinal studies, the proportional hazard model is often used to analyse covariate effects on the duration time, defined as the elapsed time between the first and second event. In this article, we consider the situation when the first event suffers partly interval-censoring and the second event suffers left-truncation and right-censoring. We proposed a two-step estimation procedure for estimating the regression coefficients of the proportional model. A simulation study is conducted to investigate the performance of the proposed estimator.     

Inference on semiparametric transformation model with general interval-censored failure time data

Failure time data occur in many areas and in various censoring forms and many models have been proposed for their regression analysis such as the proportional hazards model and the proportional odds model. Another choice that has been discussed in the literature is a general class of semiparmetric transformation models, which include the two models above and many others as special cases. In this paper, we consider this class of models when one faces a general type of censored data, case K informatively interval-censored data, for which there does not seem to exist an established inference procedure. For the problem, we present a two-step estimation procedure that is quite flexible and can be easily implemented, and the consistency and asymptotic normality of the proposed estimators of regression parameters are established. In addition, an extensive simulation study is conducted and suggests that the proposed procedure works well for practical situations. An application is also provided.     

Case-cohort analysis with semiparametric transformation models

Semiparametric transformation models provide flexible regression models for survival analysis, including the Cox proportional hazards and the proportional odds models as special cases. We consider the application of semiparametric transformation models in case-cohort studies, where the covariate data are observed only on cases and on a subcohort randomly sampled from the full cohort. We first propose an approximate profile likelihood approach with full-cohort data, which amounts to the pseudo-partial likelihood approach of Zucker [2005. A pseudo-partial likelihood method for semiparametric survival regression with covariate errors. J. Amer. Statist. Assoc. 100, 1264–1277]. Simulation results show that our proposal is almost as efficient as the nonparametric maximum likelihood estimator. We then extend this approach to the case-cohort design, applying the Horvitz–Thompson weighting method to the estimating equations from the approximated profile likelihood. Two levels of weights can be utilized to achieve unbiasedness and to gain efficiency. The resulting estimator has a closed-form asymptotic covariance matrix, and is found in simulations to be substantially more efficient than the estimator based on martingale estimating equations. The extension to left-truncated data will be discussed. We illustrate the proposed method on data from a cardiovascular risk factor study conducted in Taiwan.     

Profile likelihood approaches for semiparametric copula and frailty models for clustered survival data

ABSTRACT

In clustered survival data, the dependence among individual survival times within a cluster has usually been described using copula models and frailty models. In this paper we propose a profile likelihood approach for semiparametric copula models with different cluster sizes. We also propose a likelihood ratio method based on profile likelihood for testing the absence of association parameter (i.e. test of independence) under the copula models, leading to the boundary problem of the parameter space. For this purpose, we show via simulation study that the proposed likelihood ratio method using an asymptotic chi-square mixture distribution performs well as sample size increases. We compare the behaviors of the two models using the profile likelihood approach under a semiparametric setting. The proposed method is demonstrated using two well-known data sets.     

A Bayesian semiparametric method for analyzing length-biased data

Survival data obtained from prevalent cohort study designs are often subject to length-biased sampling. Frequentist methods including estimating equation approaches, as well as full likelihood methods, are available for assessing covariate effects on survival from such data. Bayesian methods allow a perspective of probability interpretation for the parameters of interest, and may easily provide the predictive distribution for future observations while incorporating weak prior knowledge on the baseline hazard function. There is lack of Bayesian methods for analyzing length-biased data. In this paper, we propose Bayesian methods for analyzing length-biased data under a proportional hazards model. The prior distribution for the cumulative hazard function is specified semiparametrically using I-Splines. Bayesian conditional and full likelihood approaches are developed for analyzing simulated and real data.     

A class of semiparametric transformation models for survival data with a cured proportion

We propose a new class of semiparametric regression models based on a multiplicative frailty assumption with a discrete frailty, which may account for cured subgroup in population. The cure model framework is then recast as a problem with a transformation model. The proposed models can explain a broad range of nonproportional hazards structures along with a cured proportion. An efficient and simple algorithm based on the martingale process is developed to locate the nonparametric maximum likelihood estimator. Unlike existing expectation-maximization based methods, our approach directly maximizes a nonparametric likelihood function, and the calculation of consistent variance estimates is immediate. The proposed method is useful for resolving identifiability features embedded in semiparametric cure models. Simulation studies are presented to demonstrate the finite sample properties of the proposed method. A case study of stage III soft-tissue sarcoma is given as an illustration.     

Empirical likelihood inference in mixture of semiparametric varying-coefficient models for longitudinal data with non-ignorable dropout

In this paper, empirical likelihood inference in mixture of semiparametric varying-coefficient models for longitudinal data with non-ignorable dropout is investigated. We estimate the non-parametric function based on the estimating equations and the local linear profile-kernel method. An empirical log-likelihood ratio statistic for parametric components is proposed to construct confidence regions and is shown to be an asymptotically chi-squared distribution. The non-parametric version of Wilk's theorem is also derived. A simulation study is undertaken to illustrate the finite sample performance of the proposed method.     

Empirical likelihood ratio with doubly truncated data

Doubly truncated data appear in a number of applications, including astronomy and survival analysis. For doubly-truncated data, the lifetime T is observable only when U≤T≤V, where U and V are the left-truncated and right-truncated time, respectively. Based on the empirical likelihood approach of Zhou [21 Zhou, M. 2005. Empirical likelihood ratio with arbitrarily censored/truncated data by EM algorithm. J. Comput. Graph. Statist., 14: 643–656. [Taylor & Francis Online], [Web of Science ®] [Google Scholar]], we propose a modified EM algorithm of Turnbull [19 Turnbull, B. W. 1976. The empirical distribution function with arbitrarily grouped censored and truncated data. J. R. Stat. Soc. Ser. B, 38: 290–295.  [Google Scholar]] to construct the interval estimator of the distribution function of T. Simulation results indicate that the empirical likelihood method can be more efficient than the bootstrap method.     

Bayesian multiple change-point estimation for exponential distribution with truncated and censored data

This paper considers the multiple change-point estimation for exponential distribution with truncated and censored data by Gibbs sampling. After all the missing data of interest is filled in by some sampling methods such as rejection sampling method, the complete-data likelihood function is obtained. The full conditional distributions of all parameters are discussed. The means of Gibbs samples are taken as Bayesian estimations of the parameters. The implementation steps of Gibbs sampling are introduced in detail. Finally random simulation test is developed, and the results show that Bayesian estimations are fairly accurate.     

Conditions for consistent estimation in mixed-effects models for binary matched-pairs data

Parametric mixed-effects logistic models can provide effective analysis of binary matched-pairs data. Responses are assumed to follow a logistic model within pairs, with an intercept which varies across pairs according to a specified family of probability distributions G. In this paper we give necessary and sufficient conditions for consistent covariate effect estimation and present a geometric view of estimation which shows that when the assumed family of mixture distributions is rich enough, estimates of the effect of the binary covariate are typically consistent. The geometric view also shows that under the conditions for consistent estimation, the mixed-model estimator is identical to the familar conditional-likelihood estimator for matched pairs. We illustrate the findings with some examples.     

General partially linear varying-coefficient transformation models for ranking data

In this paper,we propose a class of general partially linear varying-coefficient transformation models for ranking data. In the models, the functional coefficients are viewed as nuisance parameters and approximated by B-spline smoothing approximation technique. The B-spline coefficients and regression parameters are estimated by rank-based maximum marginal likelihood method. The three-stage Monte Carlo Markov Chain stochastic approximation algorithm based on ranking data is used to compute estimates and the corresponding variances for all the B-spline coefficients and regression parameters. Through three simulation studies and a Hong Kong horse racing data application, the proposed procedure is illustrated to be accurate, stable and practical.     

A gradient-based algorithm for semiparametric models with missing covariates

In the parametric regression model, the covariate missing problem under missing at random is considered. It is often desirable to use flexible parametric or semiparametric models for the covariate distribution, which can reduce a potential misspecification problem. Recently, a completely nonparametric approach was developed by [H.Y. Chen, Nonparametric and semiparametric models for missing covariates in parameter regression, J. Amer. Statist. Assoc. 99 (2004), pp. 1176–1189; Z. Zhang and H.E. Rockette, On maximum likelihood estimation in parametric regression with missing covariates, J. Statist. Plann. Inference 47 (2005), pp. 206–223]. Although it does not require a model for the covariate distribution or the missing data mechanism, the proposed method assumes that the covariate distribution is supported only by observed values. Consequently, their estimator is a restricted maximum likelihood estimator (MLE) rather than the global MLE. In this article, we show the restricted semiparametric MLE could be very misleading in some cases. We discuss why this problem occurs and suggest an algorithm to obtain the global MLE. Then, we assess the performance of the proposed method via some simulation experiments.     

Estimation in semiparametric models using an auxiliary model

Two classes of semiparametric and nonparametric mixture models are defined to represent general kinds of prior information. For these models the nonparametric maximum likelihood estimator (NPMLE) of an unknown probability distribution is derived and is shown to be consistent and relative efficient. Linear functionals are used for the estimation of parameters. Their consistency is proved, the gain of efficiency is derived and asymptotical distributions are given.     

Estimating survival curves with left truncated and interval censored data via the ems algorithm

It is well-known that the nonparametric maximum likelihood estimator (NPMLE) of a survival function may severely underestimate the survival probabilities at very early times for left truncated data. This problem might be overcome by instead computing a smoothed nonparametric estimator (SNE) via the EMS algorithm. The close connection between the SNE and the maximum penalized likelihood estimator is also established. Extensive Monte Carlo simulations demonstrate the superior performance of the SNE over that of the NPMLE, in terms of either bias or variance, even for moderately large Samples. The methodology is illustrated with an application to the Massachusetts Health Care Panel Study dataset to estimate the probability of being functionally independent for non-poor male and female groups rcspectively.