Semiparametric Efficient Estimation for a Class of Generalized Proportional Odds Cure Models

We present a mixture cure model with the survival time of the "uncured" group coming from a class of linear transformation models, which is an extension of the proportional odds model. This class of model, first proposed by Dabrowska and Doksum (1988), which we term "generalized proportional odds model," is well suited for the mixture cure model setting due to a clear separation between long-term and short-term effects. A standard expectation-maximization algorithm can be employed to locate the nonparametric maximum likelihood estimators, which are shown to be consistent and semiparametric efficient. However, there are difficulties in the M-step due to the nonparametric component. We overcome these difficulties by proposing two different algorithms. The first is to employ an majorize-minimize (MM) algorithm in the M-step instead of the usual Newton-Raphson method, and the other is based on an alternative form to express the model as a proportional hazards frailty model. The two new algorithms are compared in a simulation study with an existing estimating equation approach by Lu and Ying (2004). The MM algorithm provides both computational stability and efficiency. A case study of leukemia data is conducted to illustrate the proposed procedures.     

Non-mixture cure correlated frailty models in Bayesian approach

In this article, we develop a Bayesian approach for the estimation of two cure correlated frailty models that have been extended to the cure frailty models introduced by Yin [34]. We used the two different type of frailty with bivariate log-normal distribution instead of gamma distribution. A likelihood function was constructed based on a piecewise exponential distribution function. The model parameters were estimated by the Markov chain Monte Carlo method. The comparison of models is based on the Cox correlated frailty model with log-normal distribution. A real data set of bilateral corneal graft rejection was used to compare these models. The results of this data, based on deviance information criteria, showed the advantage of the proposed models.     

Smooth Semi‐nonparametric Analysis for Mixture Cure Models and Its Application to Breast Cancer

Mixture cure models are widely used when a proportion of patients are cured. The proportional hazards mixture cure model and the accelerated failure time mixture cure model are the most popular models in practice. Usually the expectation–maximisation (EM) algorithm is applied to both models for parameter estimation. Bootstrap methods are used for variance estimation. In this paper we propose a smooth semi‐nonparametric (SNP) approach in which maximum likelihood is applied directly to mixture cure models for parameter estimation. The variance can be estimated by the inverse of the second derivative of the SNP likelihood. A comprehensive simulation study indicates good performance of the proposed method. We investigate stage effects in breast cancer by applying the proposed method to breast cancer data from the South Carolina Cancer Registry.     

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.     

Nonlinear semiparametric autoregressive model with finite mixtures of scale mixtures of skew normal innovations

We propose data generating structures which can be represented as the nonlinear autoregressive models with single and finite mixtures of scale mixtures of skew normal innovations. This class of models covers symmetric/asymmetric and light/heavy-tailed distributions, so provide a useful generalization of the symmetrical nonlinear autoregressive models. As semiparametric and nonparametric curve estimation are the approaches for exploring the structure of a nonlinear time series data set, in this article the semiparametric estimator for estimating the nonlinear function of the model is investigated based on the conditional least square method and nonparametric kernel approach. Also, an Expectation–Maximization-type algorithm to perform the maximum likelihood (ML) inference of unknown parameters of the model is proposed. Furthermore, some strong and weak consistency of the semiparametric estimator in this class of models are presented. Finally, to illustrate the usefulness of the proposed model, some simulation studies and an application to real data set are considered.     

Influence Diagnostics of Semiparametric Nonlinear Reproductive Dispersion Models

This article proposes a semiparametric nonlinear reproductive dispersion model (SNRDM) which is an extension of nonlinear reproductive dispersion model and semiparametric regression model. Maximum penalized likelihood estimators (MPLEs) of unknown parameters and nonparametric functions in SNRDMs are presented. Some novel diagnostic statistics such as Cook distance and difference deviance for parametric and nonparametric parts are developed to identify influence observations in SNRDMs on the basis of case-deletion method, and some formulae readily computed with the MPLEs algorithm for diagnostic measures are given. The equivalency of case-deletion models and mean-shift outlier models in SNRDM is investigated. A simulation study and a real example are used to illustrate the proposed diagnostic measures.     

Kernel-based global MLE of partial linear random effects models for longitudinal data

Random effects models have been playing a critical role for modelling longitudinal data. However, there are little studies on the kernel-based maximum likelihood method for semiparametric random effects models. In this paper, based on kernel and likelihood methods, we propose a pooled global maximum likelihood method for the partial linear random effects models. The pooled global maximum likelihood method employs the local approximations of the nonparametric function at a group of grid points simultaneously, instead of one point. Gaussian quadrature is used to approximate the integration of likelihood with respect to random effects. The asymptotic properties of the proposed estimators are rigorously studied. Simulation studies are conducted to demonstrate the performance of the proposed approach. We also apply the proposed method to analyse correlated medical costs in the Medical Expenditure Panel Survey data set.     

A semiparametric regression cure model for interval-censored and left-truncated data

Medical advancements have made it possible for patients to be cured of certain types of diseases. In follow-up studies, the disease event time can be subject to left truncation and interval censoring. In this article, we propose a semiparametric nonmixture cure model for the regression analysis of left-truncated and interval-censored (LTIC) data. We develop semiparametric maximum likelihood estimation for the nonmixture cure model with LTIC data. A simulation study is conducted to investigate the performance of the proposed estimators.     

Semiparametric Analysis of Truncated Data

Randomly truncated data are frequently encountered in many studies where truncation arises as a result of the sampling design. In the literature, nonparametric and semiparametric methods have been proposed to estimate parameters in one-sample models. This paper considers a semiparametric model and develops an efficient method for the estimation of unknown parameters. The model assumes that K populations have a common probability distribution but the populations are observed subject to different truncation mechanisms. Semiparametric likelihood estimation is studied and the corresponding inferences are derived for both parametric and nonparametric components in the model. The method can also be applied to two-sample problems to test the difference of lifetime distributions. Simulation results and a real data analysis are presented to illustrate the methods.     

Empirical Likelihood Inferences for Semiparametric Varying-Coefficient Partially Linear Models with Longitudinal Data

In this article, empirical likelihood inferences for semiparametric varying-coefficient partially linear models with longitudinal data are investigated. We propose a groupwise empirical likelihood procedure to handle the inter-series dependence of the longitudinal data. By using residual-adjustment, an empirical likelihood ratio function for the nonparametric component is constructed, and a nonparametric version Wilks' phenomenons is proved. Compared with methods based on normal approximations, the empirical likelihood does not require consistent estimators for the asymptotic variance and bias. A simulation study is undertaken to assess the finite sample performance of the proposed confidence regions.     

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.     

Using Adjacent-category Logits Procedure for Estimating Receiver Operating Characteristic Surface

This article presents a semiparametric method for estimating receiver operating characteristic surface under density ratio model. The construction of the proposed method is based on the adjacent-category logit model and the empirical likelihood approach. A bootstrap approach for the VUS estimator inference is presented. In a simulation study, the proposed estimator is compared with the existing parametric and nonparametric estimators in terms of bias, standard error, and mean square error. Finally, a real data example and some discussions on the proposed method are provided.     

Maximum likelihood computation for fitting semiparametric mixture models

Three general algorithms that use different strategies are proposed for computing the maximum likelihood estimate of a semiparametric mixture model. They seek to maximize the likelihood function by, respectively, alternating the parameters, profiling the likelihood and modifying the support set. All three algorithms make a direct use of the recently proposed fast and stable constrained Newton method for computing the nonparametric maximum likelihood of a mixing distribution and employ additionally an optimization algorithm for unconstrained problems. The performance of the algorithms is numerically investigated and compared for solving the Neyman-Scott problem, overcoming overdispersion in logistic regression models and fitting two-level mixed effects logistic regression models. Satisfactory results have been obtained.     

Regression analysis of informative current status data with the semiparametric linear transformation model

Many methods have been developed in the literature for regression analysis of current status data with noninformative censoring and also some approaches have been proposed for semiparametric regression analysis of current status data with informative censoring. However, the existing approaches for the latter situation are mainly on specific models such as the proportional hazards model and the additive hazard model. Corresponding to this, in this paper, we consider a general class of semiparametric linear transformation models and develop a sieve maximum likelihood estimation approach for the inference. In the method, the copula model is employed to describe the informative censoring or relationship between the failure time of interest and the censoring time, and Bernstein polynomials are used to approximate the nonparametric functions involved. The asymptotic consistency and normality of the proposed estimators are established, and an extensive simulation study is conducted and indicates that the proposed approach works well for practical situations. In addition, an illustrative example is provided.     

Defective models induced by gamma frailty term for survival data with cured fraction

In this paper, we propose a defective model induced by a frailty term for modeling the proportion of cured. Unlike most of the cure rate models, defective models have advantage of modeling the cure rate without adding any extra parameter in model. The introduction of an unobserved heterogeneity among individuals has bring advantages for the estimated model. The influence of unobserved covariates is incorporated using a proportional hazard model. The frailty term assumed to follow a gamma distribution is introduced on the hazard rate to control the unobservable heterogeneity of the patients. We assume that the baseline distribution follows a Gompertz and inverse Gaussian defective distributions. Thus we propose and discuss two defective distributions: the defective gamma-Gompertz and gamma-inverse Gaussian regression models. Simulation studies are performed to verify the asymptotic properties of the maximum likelihood estimator. Lastly, in order to illustrate the proposed model, we present three applications in real data sets, in which one of them we are using for the first time, related to a study about breast cancer in the A.C.Camargo Cancer Center, São Paulo, Brazil.     

Bayesian spectral analysis models for quantile regression with Dirichlet process mixtures

This paper presents a Bayesian analysis of partially linear additive models for quantile regression. We develop a semiparametric Bayesian approach to quantile regression models using a spectral representation of the nonparametric regression functions and the Dirichlet process (DP) mixture for error distribution. We also consider Bayesian variable selection procedures for both parametric and nonparametric components in a partially linear additive model structure based on the Bayesian shrinkage priors via a stochastic search algorithm. Based on the proposed Bayesian semiparametric additive quantile regression model referred to as BSAQ, the Bayesian inference is considered for estimation and model selection. For the posterior computation, we design a simple and efficient Gibbs sampler based on a location-scale mixture of exponential and normal distributions for an asymmetric Laplace distribution, which facilitates the commonly used collapsed Gibbs sampling algorithms for the DP mixture models. Additionally, we discuss the asymptotic property of the sempiparametric quantile regression model in terms of consistency of posterior distribution. Simulation studies and real data application examples illustrate the proposed method and compare it with Bayesian quantile regression methods in the literature.     

Accelerated hazards mixture cure model

We propose a new cure model for survival data with a surviving or cure fraction. The new model is a mixture cure model where the covariate effects on the proportion of cure and the distribution of the failure time of uncured patients are separately modeled. Unlike the existing mixture cure models, the new model allows covariate effects on the failure time distribution of uncured patients to be negligible at time zero and to increase as time goes by. Such a model is particularly useful in some cancer treatments when the treat effect increases gradually from zero, and the existing models usually cannot handle this situation properly. We develop a rank based semiparametric estimation method to obtain the maximum likelihood estimates of the parameters in the model. We compare it with existing models and methods via a simulation study, and apply the model to a breast cancer data set. The numerical studies show that the new model provides a useful addition to the cure model literature.     

Cox regression of clustered event times with covariates missing not at random

Motivated by a recent tuberculosis (TB) study, this paper is concerned with covariates missing not at random (MNAR) and models the potential intracluster correlation by a frailty. We consider the regression analysis of right‐censored event times from clustered subjects under a Cox proportional hazards frailty model and present the semiparametric maximum likelihood estimator (SPMLE) of the model parameters. An easy‐to‐implement pseudo‐SPMLE is then proposed to accommodate more realistic situations using readily available supplementary information on the missing covariates. Algorithms are provided to compute the estimators and their consistent variance estimators. We demonstrate that both the SPMLE and the pseudo‐SPMLE are consistent and asymptotically normal by the arguments based on the theory of modern empirical processes. The proposed approach is examined numerically via simulation and illustrated with an analysis of the motivating TB study data.     

样本选择模型的一个简单半参数估计量

本文发展了一个针对样本选择模型的两阶段半参数估计量,其首先在第一阶段基于对数欧几里得分布差异测度估计离散选择概率,进而在第二阶段利用非参数sieve方法估计一个包含参数和非参数部分的部分线性模型以得到模型参数的估计。相对于文献中已有的半参数估计量,该估计量的计算更加简便,且计算负担相对较小。我们说明了该半参数估计量的一致性和渐近正态性,同时给出了其渐近方差的计算公式。蒙特卡洛模拟结果符合我们的理论结论。     

Maximum Penalized Likelihood Estimation in a Gamma-Frailty Model

The shared frailty models allow for unobserved heterogeneity or for statistical dependence between observed survival data. The most commonly used estimation procedure in frailty models is the EM algorithm, but this approach yields a discrete estimator of the distribution and consequently does not allow direct estimation of the hazard function. We show how maximum penalized likelihood estimation can be applied to nonparametric estimation of a continuous hazard function in a shared gamma-frailty model with right-censored and left-truncated data. We examine the problem of obtaining variance estimators for regression coefficients, the frailty parameter and baseline hazard functions. Some simulations for the proposed estimation procedure are presented. A prospective cohort (Paquid) with grouped survival data serves to illustrate the method which was used to analyze the relationship between environmental factors and the risk of dementia.