Estimated estimating equations: semiparametric inference for clustered and longitudinal data

Summary.  We introduce a flexible marginal modelling approach for statistical inference for clustered and longitudinal data under minimal assumptions. This estimated estimating equations approach is semiparametric and the proposed models are fitted by quasi-likelihood regression, where the unknown marginal means are a function of the fixed effects linear predictor with unknown smooth link, and variance–covariance is an unknown smooth function of the marginal means. We propose to estimate the nonparametric link and variance–covariance functions via smoothing methods, whereas the regression parameters are obtained via the estimated estimating equations. These are score equations that contain nonparametric function estimates. The proposed estimated estimating equations approach is motivated by its flexibility and easy implementation. Moreover, if data follow a generalized linear mixed model, with either a specified or an unspecified distribution of random effects and link function, the model proposed emerges as the corresponding marginal (population-average) version and can be used to obtain inference for the fixed effects in the underlying generalized linear mixed model, without the need to specify any other components of this generalized linear mixed model. Among marginal models, the estimated estimating equations approach provides a flexible alternative to modelling with generalized estimating equations. Applications of estimated estimating equations include diagnostics and link selection. The asymptotic distribution of the proposed estimators for the model parameters is derived, enabling statistical inference. Practical illustrations include Poisson modelling of repeated epileptic seizure counts and simulations for clustered binomial responses.     

Adaptive varying-coefficient linear models

Summary. Varying-coefficient linear models arise from multivariate nonparametric regression, non-linear time series modelling and forecasting, functional data analysis, longitudinal data analysis and others. It has been a common practice to assume that the varying coefficients are functions of a given variable, which is often called an index . To enlarge the modelling capacity substantially, this paper explores a class of varying-coefficient linear models in which the index is unknown and is estimated as a linear combination of regressors and/or other variables. We search for the index such that the derived varying-coefficient model provides the least squares approximation to the underlying unknown multidimensional regression function. The search is implemented through a newly proposed hybrid backfitting algorithm. The core of the algorithm is the alternating iteration between estimating the index through a one-step scheme and estimating coefficient functions through one-dimensional local linear smoothing. The locally significant variables are selected in terms of a combined use of the t -statistic and the Akaike information criterion. We further extend the algorithm for models with two indices. Simulation shows that the methodology proposed has appreciable flexibility to model complex multivariate non-linear structure and is practically feasible with average modern computers. The methods are further illustrated through the Canadian mink–muskrat data in 1925–1994 and the pound–dollar exchange rates in 1974–1983.     

Comparison of Nonparametric Components in Two Partially Linear Models

In this article, we are concerned with whether the nonparametric functions are parallel from two partial linear models, and propose a test statistic to check the difference of the two functions. The unknown constant α is estimated by using moment method under null models. Nonparametric functions under both null and full models are estimated by using local linear method. The asymptotic properties of parametric and nonparametric components are derived. The test statistic under the null hypothesis is calculated and shown to be asymptotically normal.     

Efficient estimation in partially linear single‐index models for longitudinal data

In this paper, we consider the estimation of both the parameters and the nonparametric link function in partially linear single‐index models for longitudinal data that may be unbalanced. In particular, a new three‐stage approach is proposed to estimate the nonparametric link function using marginal kernel regression and the parametric components with generalized estimating equations. The resulting estimators properly account for the within‐subject correlation. We show that the parameter estimators are asymptotically semiparametrically efficient. We also show that the asymptotic variance of the link function estimator is minimized when the working error covariance matrices are correctly specified. The new estimators are more efficient than estimators in the existing literature. These asymptotic results are obtained without assuming normality. The finite‐sample performance of the proposed method is demonstrated by simulation studies. In addition, two real‐data examples are analyzed to illustrate the methodology.     

Quantile regression estimation of partially linear additive models

In this paper, we consider the estimation of partially linear additive quantile regression models where the conditional quantile function comprises a linear parametric component and a nonparametric additive component. We propose a two-step estimation approach: in the first step, we approximate the conditional quantile function using a series estimation method. In the second step, the nonparametric additive component is recovered using either a local polynomial estimator or a weighted Nadaraya–Watson estimator. Both consistency and asymptotic normality of the proposed estimators are established. Particularly, we show that the first-stage estimator for the finite-dimensional parameters attains the semiparametric efficiency bound under homoskedasticity, and that the second-stage estimators for the nonparametric additive component have an oracle efficiency property. Monte Carlo experiments are conducted to assess the finite sample performance of the proposed estimators. An application to a real data set is also illustrated.     

Longitudinal data analysis based on generalized linear partially varying-coefficient models

In this article, we consider a generalized linear partially varying-coefficient model for longitudinal data analysis. A local quasi-likelihood method is proposed to estimate the constant-coefficient and varying-coefficient functions simultaneously based on the local polynomial kernel regression. The corresponding standard error estimates are derived. Large sample properties are investigated. The proposed methodologies are demonstrated by extensive simulation studies and a real example.     

Model selection and model averaging for semiparametric partially linear models with missing data

We study model selection and model averaging in semiparametric partially linear models with missing responses. An imputation method is used to estimate the linear regression coefficients and the nonparametric function. We show that the corresponding estimators of the linear regression coefficients are asymptotically normal. Then a focused information criterion and frequentist model average estimators are proposed and their theoretical properties are established. Simulation studies are performed to demonstrate the superiority of the proposed methods over the existing strategies in terms of mean squared error and coverage probability. Finally, the approach is applied to a real data case.     

Robust estimation and wavelet thresholding in partially linear models

This paper is concerned with a semiparametric partially linear regression model with unknown regression coefficients, an unknown nonparametric function for the non-linear component, and unobservable Gaussian distributed random errors. We present a wavelet thresholding based estimation procedure to estimate the components of the partial linear model by establishing a connection between an l ₁-penalty based wavelet estimator of the nonparametric component and Huber’s M-estimation of a standard linear model with outliers. Some general results on the large sample properties of the estimates of both the parametric and the nonparametric part of the model are established. Simulations are used to illustrate the general results and to compare the proposed methodology with other methods available in the recent literature.     

Estimation of regression parameters in a semiparametric transformation model

A semiparametric approach to model skewed/heteroscedastic regression data is discussed. We work with a semiparametric transform-both-sides regression model, which contains a parametric regression function and a nonparametric transformation. This model is adequate when the relationship between the median response and the explanatory variable has been specified by a theoretical result or a previous empirical study. The transform-both-sides model with a parametric transformation has been studied extensively and applied successfully to a number data sets. Allowing a nonparametric transformation function increases the flexibility of the model. In this article, we estimate the nonparametric transformation function by the conditional kernel density approach developed by Wang and Ruppert (1995), and then use a pseudo-maximum likelihood estimator to estimate the regression parameters. This estimate of the regression parameters has not been studied previously. In this article, the asymptotic distribution of this pseudo-MLE is derived. We also show that when σ, the standard deviation of the error, goes to zero (small σ asymptotics), this estimator is adaptive. Adaptive means that the regression parameters are estimated as precisely as when the transformation is known exactly. A similar result holds in the parametric approaches of Carroll and Ruppert (1984) and Ruppert and Aldershof (1989). Simulated and real examples are provided to illustrate the performance of the proposed estimator for finite sample size.     

Local Influence Analysis for The Ridge Regression Under Stochastic Linear Restrictions

In this article, we assess the local influence for the ridge regression of linear models with stochastic linear restrictions in the spirit of Cook by using the log-likelihood of the stochastic restricted ridge regression estimator. The diagnostics under the perturbations of constant variance, responses and individual explanatory variables are derived. We also assess the local influence of the stochastic restricted ridge regression estimator under the approach suggested by Billor and Loynes. At the end, a numerical example on the Longley data is given to illustrate the theoretic results.     

Robust testing with generalized partial linear models for longitudinal data

By approximating the nonparametric component using a regression spline in generalized partial linear models (GPLM), robust generalized estimating equations (GEE), involving bounded score function and leverage-based weighting function, can be used to estimate the regression parameters in GPLM robustly for longitudinal data or clustered data. In this paper, score test statistics are proposed for testing the regression parameters with robustness, and their asymptotic distributions under the null hypothesis and a class of local alternative hypotheses are studied. The proposed score tests reply on the estimation of a smaller model without the testing parameters involved, and perform well in the simulation studies and real data analysis conducted in this paper.     

General purpose multiply robust data integration procedures for handling nonprobability samples

In recent years, there has been an increased interest in combining probability and nonprobability samples. Nonprobability sample are cheaper and quicker to conduct but the resulting estimators are vulnerable to bias as the participation probabilities are unknown. To adjust for the potential bias, estimation procedures based on parametric or nonparametric models have been discussed in the literature. However, the validity of the resulting estimators relies heavily on the validity of the underlying models. Also, nonparametric approaches may suffer from the curse of dimensionality and poor efficiency. We propose a data integration approach by combining multiple outcome regression models and propensity score models. The proposed approach can be used for estimating general parameters including totals, means, distribution functions, and percentiles. The resulting estimators are multiply robust in the sense that they remain consistent if all but one model are misspecified. The asymptotic properties of point and variance estimators are established. The results from a simulation study show the benefits of the proposed method in terms of bias and efficiency. Finally, we apply the proposed method using data from the Korea National Health and Nutrition Examination Survey and data from the National Health Insurance Sharing Services.     

Efficient Computation of Reduced Regression Models

We consider settings where it is of interest to fit and assess regression submodels that arise as various explanatory variables are excluded from a larger regression model. The larger model is referred to as the full model; the submodels are the reduced models. We show that a computationally efficient approximation to the regression estimates under any reduced model can be obtained from a simple weighted least squares (WLS) approach based on the estimated regression parameters and covariance matrix from the full model. This WLS approach can be considered an extension to unbiased estimating equations of a first-order Taylor series approach proposed by Lawless and Singhal. Using data from the 2010 Nationwide Inpatient Sample (NIS), a 20% weighted, stratified, cluster sample of approximately 8 million hospital stays from approximately 1000 hospitals, we illustrate the WLS approach when fitting interval censored regression models to estimate the effect of type of surgery (robotic versus nonrobotic surgery) on hospital length-of-stay while adjusting for three sets of covariates: patient-level characteristics, hospital characteristics, and zip-code level characteristics. Ordinarily, standard fitting of the reduced models to the NIS data takes approximately 10 hours; using the proposed WLS approach, the reduced models take seconds to fit.     

Diagnostics for non-linear regression

Sensitivity analysis in regression is concerned with assessing the sensitivity of the results of a regression model (e.g., the objective function, the regression parameters, and the fitted values) to changes in the data. Sensitivity analysis in least squares linear regression has seen a great surge of research activities over the last three decades. By contrast, sensitivity analysis in non-linear regression has received very little attention. This paper deals with the problem of local sensitivity analysis in non-linear regression. Closed-form general formulas are provided for the sensitivities of three standard methods for the estimation of the parameters of a non-linear regression model based on a set of data. These methods are the least squares, the minimax, and the least absolute value methods. The effectiveness of the proposed measures is illustrated by application to several non-linear models including the ultrasonic data and the onion yield data. The proposed sensitivity measures are shown to deal effectively with the detection of influential observations in non-linear regression models.     

Partially linear censored quantile regression

Censored regression quantile (CRQ) methods provide a powerful and flexible approach to the analysis of censored survival data when standard linear models are felt to be appropriate. In many cases however, greater flexibility is desired to go beyond the usual multiple regression paradigm. One area of common interest is that of partially linear models: one (or more) of the explanatory covariates are assumed to act on the response through a non-linear function. Here the CRQ approach of Portnoy (J Am Stat Assoc 98:1001–1012, 2003) is extended to this partially linear setting. Basic consistency results are presented. A simulation experiment and unemployment example justify the value of the partially linear approach over methods based on the Cox proportional hazards model and on methods not permitting nonlinearity.     

Nonparametric regression for dependent data in the errors-in-variables problem

We consider the nonparametric estimation of the regression functions for dependent data. Suppose that the covariates are observed with additive errors in the data and we employ nonparametric deconvolution kernel techniques to estimate the regression functions in this paper. We investigate how the strength of time dependence affects the asymptotic properties of the local constant and linear estimators. We treat both short-range dependent and long-range dependent linear processes in a unified way and demonstrate that the long-range dependence (LRD) of the covariates affects the asymptotic properties of the nonparametric estimators as well as the LRD of regression errors does.     

Variance estimation in complex survey sampling for generalized linear models

Summary.  Complex survey sampling is often used to sample a fraction of a large finite population. In general, the survey is conducted so that each unit (e.g. subject) in the sample has a different probability of being selected into the sample. For generalizability of the sample to the population, both the design and the probability of being selected into the sample must be incorporated in the analysis. In this paper we focus on non-standard regression models for complex survey data. In our motivating example, which is based on data from the Medical Expenditure Panel Survey, the outcome variable is the subject's 'total health care expenditures in the year 2002'. Previous analyses of medical cost data suggest that the variance is approximately equal to the mean raised to the power of 1.5, which is a non-standard variance function. Currently, the regression parameters for this model cannot be easily estimated in standard statistical software packages. We propose a simple two-step method to obtain consistent regression parameter and variance estimates; the method proposed can be implemented within any standard sample survey package. The approach is applicable to complex sample surveys with any number of stages.     

Partially Linear Additive Models with Unknown Link Functions

In this paper, we consider partially linear additive models with an unknown link function, which include single‐index models and additive models as special cases. We use polynomial spline method for estimating the unknown link function as well as the component functions in the additive part. We establish that convergence rates for all nonparametric functions are the same as in one‐dimensional nonparametric regression. For a faster rate of the parametric part, we need to define appropriate ‘projection’ that is more complicated than that defined previously for partially linear additive models. Compared to previous approaches, a distinct advantage of our estimation approach in implementation is that estimation directly reduces estimation in the single‐index model and can thus deal with much larger dimensional problems than previous approaches for additive models with unknown link functions. Simulations and a real dataset are used to illustrate the proposed model.     

On Semiparametric Mode Regression Estimation

It has been found that, for a variety of probability distributions, there is a surprising linear relation between mode, mean, and median. In this article, the relation between mode, mean, and median regression functions is assumed to follow a simple parametric model. We propose a semiparametric conditional mode (mode regression) estimation for an unknown (unimodal) conditional distribution function in the context of regression model, so that any m-step-ahead mean and median forecasts can then be substituted into the resultant model to deliver m-step-ahead mode prediction. In the semiparametric model, Least Squared Estimator (LSEs) for the model parameters and the simultaneous estimation of the unknown mean and median regression functions by the local linear kernel method are combined to infer about the parametric and nonparametric components of the proposed model. The asymptotic normality of these estimators is derived, and the asymptotic distribution of the parameter estimates is also given and is shown to follow usual parametric rates in spite of the presence of the nonparametric component in the model. These results are applied to obtain a data-based test for the dependence of mode regression over mean and median regression under a regression model.     

Local Linear Regression in Proportional Hazards Model with Censored Data

In this article we study the method of nonparametric regression based on a transformation model, under which an unknown transformation of the survival time is nonlinearly, even more, nonparametrically, related to the covariates with various error distributions, which are parametrically specified with unknown parameters. Local linear approximations and locally weighted least squares are applied to obtain estimators for the effects of covariates with censored observations. We show that the estimators are consistent and asymptotically normal. This transformation model, coupled with local linear approximation techniques, provides many alternatives to the more general proportional hazards models with nonparametric covariates.