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.     

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.     

Locally Efficient Semiparametric Estimators for Proportional Hazards Models with Measurement Error

We propose a new class of semiparametric estimators for proportional hazards models in the presence of measurement error in the covariates, where the baseline hazard function, the hazard function for the censoring time, and the distribution of the true covariates are considered as unknown infinite dimensional parameters. We estimate the model components by solving estimating equations based on the semiparametric efficient scores under a sequence of restricted models where the logarithm of the hazard functions are approximated by reduced rank regression splines. The proposed estimators are locally efficient in the sense that the estimators are semiparametrically efficient if the distribution of the error‐prone covariates is specified correctly and are still consistent and asymptotically normal if the distribution is misspecified. Our simulation studies show that the proposed estimators have smaller biases and variances than competing methods. We further illustrate the new method with a real application in an HIV clinical trial.     

On minimum Hellinger distance estimation

Efficiency and robustness are two fundamental concepts in parametric estimation problems. It was long thought that there was an inherent contradiction between the aims of achieving robustness and efficiency; that is, a robust estimator could not be efficient and vice versa. It is now known that the minimum Hellinger distance approached introduced by Beran [R. Beran, Annals of Statistics 1977;5:445–463] is one way of reconciling the conflicting concepts of efficiency and robustness. For parametric models, it has been shown that minimum Hellinger estimators achieve efficiency at the model density and simultaneously have excellent robustness properties. In this article, we examine the application of this approach in two semiparametric models. In particular, we consider a two‐component mixture model and a two‐sample semiparametric model. In each case, we investigate minimum Hellinger distance estimators of finite‐dimensional Euclidean parameters of particular interest and study their basic asymptotic properties. Small sample properties of the proposed estimators are examined using a Monte Carlo study. The results can be extended to semiparametric models of general form as well. The Canadian Journal of Statistics 37: 514–533; 2009 © 2009 Statistical Society of Canada     

Buckley–James Type Estimator for Censored Data with Covariates Missing by Design

Abstract. The Buckley–James estimator (BJE) is a well‐known estimator for linear regression models with censored data. Ritov has generalized the BJE to a semiparametric setting and demonstrated that his class of Buckley–James type estimators is asymptotically equivalent to the class of rank‐based estimators proposed by Tsiatis. In this article, we revisit such relationship in censored data with covariates missing by design. By exploring a similar relationship between our proposed class of Buckley–James type estimating functions to the class of rank‐based estimating functions recently generalized by Nan, Kalbfleisch and Yu, we establish asymptotic properties of our proposed estimators. We also conduct numerical studies to compare asymptotic efficiencies from various estimators.     

Semiparametric estimation of the cumulative incidence function under competing risks and left-truncated sampling

In this article, we propose semiparametric methods to estimate the cumulative incidence function of two dependent competing risks for left-truncated and right-censored data. The proposed method is based on work by Huang and Wang (1995). We extend previous model by allowing for a general parametric truncation distribution and a third competing risk before recruitment. Based on work by Vardi (1989), several iterative algorithms are proposed to obtain the semiparametric estimates of cumulative incidence functions. The asymptotic properties of the semiparametric estimators are derived. Simulation results show that a semiparametric approach assuming the parametric truncation distribution is correctly specified produces estimates with smaller mean squared error than those obtained in a fully nonparametric model.     

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.     

Variance Estimation under Two‐Phase Sampling

We consider the variance estimation of the weighted likelihood estimator (WLE) under two‐phase stratified sampling without replacement. Asymptotic variance of the WLE in many semiparametric models contains unknown functions or does not have a closed form. The standard method of the inverse probability weighted (IPW) sample variances of an estimated influence function is then not available in these models. To address this issue, we develop the variance estimation procedure for the WLE in a general semiparametric model. The phase I variance is estimated by taking a numerical derivative of the IPW log likelihood. The phase II variance is estimated based on the bootstrap for a stratified sample in a finite population. Despite a theoretical difficulty of dependent observations due to sampling without replacement, we establish the (bootstrap) consistency of our estimators. Finite sample properties of our method are illustrated in a simulation study.     

Estimation of tobit-type models with individual specific effects

The aim of this paper is two-fold. First, we review recent estimators for censored regression and sample selection panel data models with unobservable individual specific effects, and show how the idea behind these estimators can be used to construct estimators for a variety of other Tobit-type models. The estimators presented in this paper are semiparametric, in the sense that they do not require the parametrization of the distribution of the unobservables. The second aim of the paper is to introduce a new class of estimators for the censored regression model. The advantage of the new estimators is that they can be applied under a stationarity assumption on the transitory error terms, which is weaker than the exchangeability assumption that is usually made in this literature. A similar generalization does not seem feasible for the estimators of the other models that are considered.     

Analysis of generalized semiparametric mixed varying‐coefficients models for longitudinal data

The generalized semiparametric mixed varying‐coefficient effects model for longitudinal data can accommodate a variety of link functions and flexibly model different types of covariate effects, including time‐constant, time‐varying and covariate‐varying effects. The time‐varying effects are unspecified functions of time and the covariate‐varying effects are nonparametric functions of a possibly time‐dependent exposure variable. A semiparametric estimation procedure is developed that uses local linear smoothing and profile weighted least squares, which requires smoothing in the two different and yet connected domains of time and the time‐dependent exposure variable. The asymptotic properties of the estimators of both nonparametric and parametric effects are investigated. In addition, hypothesis testing procedures are developed to examine the covariate effects. The finite‐sample properties of the proposed estimators and testing procedures are examined through simulations, indicating satisfactory performances. The proposed methods are applied to analyze the AIDS Clinical Trial Group 244 clinical trial to investigate the effects of antiretroviral treatment switching in HIV‐infected patients before and after developing the T215Y antiretroviral drug resistance mutation. The Canadian Journal of Statistics 47: 352–373; 2019 © 2019 Statistical Society of Canada     

On the asymptotic non‐equivalence of efficient‐GMM and MEL estimators in models with missing data

The generalized method of moments (GMM) and empirical likelihood (EL) are popular methods for combining sample and auxiliary information. These methods are used in very diverse fields of research, where competing theories often suggest variables satisfying different moment conditions. Results in the literature have shown that the efficient‐GMM (GMM_E) and maximum empirical likelihood (MEL) estimators have the same asymptotic distribution to order n^?1/2 and that both estimators are asymptotically semiparametric efficient. In this paper, we demonstrate that when data are missing at random from the sample, the utilization of some well‐known missing‐data handling approaches proposed in the literature can yield GMM_E and MEL estimators with nonidentical properties; in particular, it is shown that the GMM_E estimator is semiparametric efficient under all the missing‐data handling approaches considered but that the MEL estimator is not always efficient. A thorough examination of the reason for the nonequivalence of the two estimators is presented. A particularly strong feature of our analysis is that we do not assume smoothness in the underlying moment conditions. Our results are thus relevant to situations involving nonsmooth estimating functions, including quantile and rank regressions, robust estimation, the estimation of receiver operating characteristic (ROC) curves, and so on.     

Estimating heritability for cause specific mortality based on twin studies

There has been considerable interest in studying the magnitude and type of inheritance of specific diseases. This is typically derived from family or twin studies, where the basic idea is to compare the correlation for different pairs that share different amount of genes. We here consider data from the Danish twin registry and discuss how to define heritability for cancer occurrence. The key point is that this should be done taking censoring as well as competing risks due to e.g.  death into account. We describe the dependence between twins on the probability scale and show that various models can be used to achieve sensible estimates of the dependence within monozygotic and dizygotic twin pairs that may vary over time. These dependence measures can subsequently be decomposed into a genetic and environmental component using random effects models. We here present several novel models that in essence describe the association in terms of the concordance probability, i.e., the probability that both twins experience the event, in the competing risks setting. We also discuss how to deal with the left truncation present in the Nordic twin registries, due to sampling only of twin pairs where both twins are alive at the initiation of the registries.     

Estimation of tobit-type models with individual specific effects

The aim of this paper is two-fold. First, we review recent estimators for censored regression and sample selection panel data models with unobservable individual specific effects, and show how the idea behind these estimators can be used to construct estimators for a variety of other Tobit-type models. The estimators presented in this paper are semiparametric, in the sense that they do not require the parametrization of the distribution of the unobservables. The second aim of the paper is to introduce a new class of estimators for the censored regression model. The advantage of the new estimators is that they can be applied under a stationarity assumption on the transitory error terms, which is weaker than the exchangeability assumption that is usually made in this literature. A similar generalization does not seem feasible for the estimators of the other models that are considered.     

Conditional maximum likelihood estimation in semiparametric transformation model with LTRC data

Left-truncated data often arise in epidemiology and individual follow-up studies due to a biased sampling plan since subjects with shorter survival times tend to be excluded from the sample. Moreover, the survival time of recruited subjects are often subject to right censoring. 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 left-truncated and right-censored data. We propose a conditional likelihood approach and develop the conditional maximum likelihood estimators (cMLE) for the regression parameters and cumulative hazard function of these models. The derived score equations for regression parameter and infinite-dimensional function suggest an iterative algorithm for cMLE. The cMLE is shown to be consistent and asymptotically normal. The limiting variances for the estimators can be consistently estimated using the inverse of negative Hessian matrix. Intensive simulation studies are conducted to investigate the performance of the cMLE. An application to the Channing House data is given to illustrate the methodology.     

Semiparametric estimation in copula models

The author recalls the limiting behaviour of the empirical copula process and applies it to prove some asymptotic properties of a minimum distance estimator for a Euclidean parameter in a copula model. The estimator in question is semiparametric in that no knowledge of the marginal distributions is necessary. The author also proposes another semiparametric estimator which he calls “rank approximate Z‐estimator” and whose asymptotic normality he derives. He further presents Monte Carlo simulation results for the comparison of various estimators in four well‐known bivariate copula models.     

Locally efficient semiparametric estimators for a class of Poisson models with measurement error

The presence of measurement error may cause bias in parameter estimation and can lead to incorrect conclusions in data analyses. Despite a large body of literature on general measurement error problems, relatively few works exist to handle Poisson models. In this article we thoroughly study Poisson models with errors in covariates and propose consistent and locally efficient semiparametric estimators. We assess the finite sample performance of the estimators through extensive simulation studies and illustrate the proposed methodologies by analyzing data from the Stroke Recovery in Underserved Populations Study. The Canadian Journal of Statistics 47: 157–181; 2019 © 2019 Statistical Society of Canada     

Relative risk regression for current status data in case‐cohort studies

We propose using the weighted likelihood method to fit a general relative risk regression model for the current status data with missing data as arise, for example, in case‐cohort studies. The missingness probability is either known or can be reasonably estimated. Asymptotic properties of the weighted likelihood estimators are established. For the case of using estimated weights, we construct a general theorem that guarantees the asymptotic normality of the M‐estimator of a finite dimensional parameter in a class of semiparametric models, where the infinite dimensional parameter is allowed to converge at a slower than parametric rate, and some other parameters in the objective function are estimated a priori. The weighted bootstrap method is employed to estimate the variances. Simulations show that the proposed method works well for finite sample sizes. A motivating example of the case‐cohort study from an HIV vaccine trial is used to demonstrate the proposed method. The Canadian Journal of Statistics 39: 557–577; 2011. © 2011 Statistical Society of Canada     

A Semiparametric Regression Model for Longitudinal Data with Non‐stationary Errors

Motivated by the need to analyze the National Longitudinal Surveys data, we propose a new semiparametric longitudinal mean‐covariance model in which the effects on dependent variable of some explanatory variables are linear and others are non‐linear, while the within‐subject correlations are modelled by a non‐stationary autoregressive error structure. We develop an estimation machinery based on least squares technique by approximating non‐parametric functions via B‐spline expansions and establish the asymptotic normality of parametric estimators as well as the rate of convergence for the non‐parametric estimators. We further advocate a new model selection strategy in the varying‐coefficient model framework, for distinguishing whether a component is significant and subsequently whether it is linear or non‐linear. Besides, the proposed method can also be employed for identifying the true order of lagged terms consistently. Monte Carlo studies are conducted to examine the finite sample performance of our approach, and an application of real data is also illustrated.     

A class of nonparametric bivariate survival function estimators for randomly censored and truncated data

This paper proposes a class of nonparametric estimators for the bivariate survival function estimation under both random truncation and random censoring. In practice, the pair of random variables under consideration may have certain parametric relationship. The proposed class of nonparametric estimators uses such parametric information via a data transformation approach and thus provides more accurate estimates than existing methods without using such information. The large sample properties of the new class of estimators and a general guidance of how to find a good data transformation are given. The proposed method is also justified via a simulation study and an application on an economic data set.     

A two-stage estimation in the Clayton–Oakes model with marginal linear transformation models for multivariate failure time data

This paper considers the analysis of multivariate survival data where the marginal distributions are specified by semiparametric transformation models, a general class including the Cox model and the proportional odds model as special cases. First, consideration is given to the situation where the joint distribution of all failure times within the same cluster is specified by the Clayton–Oakes model (Clayton, Biometrika 65:141–151, l978; Oakes, J R Stat Soc B 44:412–422, 1982). A two-stage estimation procedure is adopted by first estimating the marginal parameters under the independence working assumption, and then the association parameter is estimated from the maximization of the full likelihood function with the estimators of the marginal parameters plugged in. The asymptotic properties of all estimators in the semiparametric model are derived. For the second situation, the third and higher order dependency structures are left unspecified, and interest focuses on the pairwise correlation between any two failure times. Thus, the pairwise association estimate can be obtained in the second stage by maximizing the pairwise likelihood function. Large sample properties for the pairwise association are also derived. Simulation studies show that the proposed approach is appropriate for practical use. To illustrate, a subset of the data from the Diabetic Retinopathy Study is used.