A flexible marginal modelling strategy for non-monotone missing data

Summary.  Much research has been devoted to modelling strategies for longitudinal data with missingness, recently especially within the missingness not at random context. In this paper, the relatively unexplored but practically highly relevant domain of non-monotone missingness with multivariate ordinal responses is broached. For this, a dedicated version of the multivariate Dale model is formulated. Furthermore, we also assess the sensitivity of these models to their assumptions, by using the technique of global influence.     

Univaiuate anu multivariate categorical data analysis for block designs

Analysis for univariate and multivariate categorical data in block designs is given and illustrated through examples. The univariate analysis compares the treatments on the basis of their pooled frequency distributions (pooled over blocks). The test statistic used is called Q after Cochran (1950). The large sample null distribution of Q is a chi-square. Analysis of p-variate categorical data (kth variable having ck classes, K=1,...,p) can be done by treating it as a univariate categorical problem with [d] classes. Very often [d] is large in relation to the size of the experiment. This makes the expected frequencies for some of the cells very small, making the univariate method inapplicable. In these circumstances it may be reasonable to compare the treatments on the basis of marginal distributions up to the mth dimension, 1[d] , which is given in this paper. This method is also illustrated for missing observations     

Skew-mixed effects model for multivariate longitudinal data with categorical outcomes and missingness

A longitudinal study commonly follows a set of variables, measured for each individual repeatedly over time, and usually suffers from incomplete data problem. A common approach for dealing with longitudinal categorical responses is to use the Generalized Linear Mixed Model (GLMM). This model induces the potential relation between response variables over time via a vector of random effects, assumed to be shared parameters in the non-ignorable missing mechanism. Most GLMMs assume that the random-effects parameters follow a normal or symmetric distribution and this leads to serious problems in real applications. In this paper, we propose GLMMs for the analysis of incomplete multivariate longitudinal categorical responses with a non-ignorable missing mechanism based on a shared parameter framework with the less restrictive assumption of skew-normality for the random effects. These models may contain incomplete data with monotone and non-monotone missing patterns. The performance of the model is evaluated using simulation studies and a well-known longitudinal data set extracted from a fluvoxamine trial is analyzed to determine the profile of fluvoxamine in ambulatory clinical psychiatric practice.     

Joint models for mixed categorical outcomes: a study of HIV risk perception and disease status in Mozambique

Two types of bivariate models for categorical response variables are introduced to deal with special categories such as ‘unsure’ or ‘unknown’ in combination with other ordinal categories, while taking additional hierarchical data structures into account. The latter is achieved by the use of different covariance structures for a trivariate random effect. The models are applied to data from the INSIDA survey, where interest goes to the effect of covariates on the association between HIV risk perception (quadrinomial with an ‘unknown risk’ category) and HIV infection status (binary). The final model combines continuation-ratio with cumulative link logits for the risk perception, together with partly correlated and partly shared trivariate random effects for the household level. The results indicate that only age has a significant effect on the association between HIV risk perception and infection status. The proposed models may be useful in various fields of application such as social and biomedical sciences, epidemiology and public health.     

A skew-normal random effects model for longitudinal ordinal categorical responses with missing data

Missing values are common in longitudinal data studies. The missing data mechanism is termed non-ignorable (NI) if the probability of missingness depends on the non-response (missing) observations. This paper presents a model for the ordinal categorical longitudinal data with NI non-monotone missing values. We assumed two separate models for the response and missing procedure. The response is modeled as ordinal logistic, whereas the logistic binary model is considered for the missing process. We employ these models in the context of so-called shared-parameter models, where the outcome and missing data models are connected by a common set of random effects. It is commonly assumed that the random effect follows the normal distribution in longitudinal data with or without missing data. This can be extremely restrictive in practice, and it may result in misleading statistical inferences. In this paper, we instead adopt a more flexible alternative distribution which is called the skew-normal distribution. The methodology is illustrated through an application to Schizophrenia Collaborative Study data [19 D. Hedeker, Generalized linear mixed models, in Encyclopedia of Statistics in Behavioral Science, B. Everitt and D. Howell, eds., John Wiley, London, 2005, pp. 729–738. [Google Scholar]] and a simulation.     

Bayesian factor models for multivariate categorical data obtained from questionnaires

Factor analysis is a flexible technique for assessment of multivariate dependence and codependence. Besides being an exploratory tool used to reduce the dimensionality of multivariate data, it allows estimation of common factors that often have an interesting theoretical interpretation in real problems. However, standard factor analysis is only applicable when the variables are scaled, which is often inappropriate, for example, in data obtained from questionnaires in the field of psychology, where the variables are often categorical. In this framework, we propose a factor model for the analysis of multivariate ordered and non-ordered polychotomous data. The inference procedure is done under the Bayesian approach via Markov chain Monte Carlo methods. Two Monte Carlo simulation studies are presented to investigate the performance of this approach in terms of estimation bias, precision and assessment of the number of factors. We also illustrate the proposed method to analyze participants'' responses to the Motivational State Questionnaire dataset, developed to study emotions in laboratory and field settings.     

A multivariate logit model with marginal canonical association

In this work, a generalization of the Goodman Association Model to the case of q, q > 2, categorical variables which is based on the idea of marginal modelling discussed by Gloneck–McCullagh is introduced; the difference between the proposed generalization and two models, previously introduced by Becker and Colombi, is discussed. The Becker generalization is not a marginal model because it does not imply Logit Models for the marginal probabilities, and because it is based on the conditional approach of modelling the association. The Colombi model is only partially a marginal model because it uses simple logit models for the univariate marginal probabilities but is based on the conditional approach of modelling the association. It is also shown that the maximum likelihood estimation of the parameters of the new model is feasible and, to compute the maximum likelihood estimates, an algorithm is proposed, which is a numerically convenient compromise between the constrained optimization approach of Lang and the straightforward use of the Fisher Scoring Algorithm suggested by Glonek–McCullagh.Finally, the proposed model is used to analyze a data set concerning work accidents which occurred to workers at some Italian firms during the years 1994–1996.     

A model for comparisons with ordered categorical data

A model developed by Andrich for ordered categorical data is extended to develop tests for treatment effects with paired or matched samples. In particular, this includes analysis for pre-post studies and crossover designs. Some advantages of this model are that it allows for misclassification of subjects, yields reasonable conditional requirements for exact analysis, a normal approximation is good for all but the smallest of sample sizes, and it is relatively simple mathematically. Furthermore, the form of the tests derived are logical extensions of tests for unordered categories.     

Time-varying coefficient cumulative gap time models for intensive longitudinal ecological momentary assessment data with missingness

Ecological momentary assessment (EMA) studies investigate intensive repeated observations of the current behavior and experiences of subjects in real time. In particular, such studies aim to minimize recall bias and maximize ecological validity, thereby strengthening the investigation and inference of microprocesses that influence behavior in real-world contexts by gathering intensive information on the temporal patterning of behavior of study subjects. Throughout this paper, we focus on the data analysis of an EMA study that examined behavior of intermittent smokers (ITS). Specifically, we sought to explore the pattern of clustered smoking behavior of ITS, or smoking ‘bouts’, as well as the covariates that predict such smoking behavior. To do this, in this paper we introduce a framework for characterizing the temporal behavior of ITS via the functions of event gap time to distinguish the smoking bouts. We used the time-varying coefficient models for the cumulative log gap time and to characterize the temporal patterns of smoking behavior, while simultaneously adjusting for behavioral covariates, and incorporated the inverse probability weighting into the models to accommodate missing data. Simulation studies showed that irrespective of whether missing by design or missing at random, the model was able to reliably determine prespecified time-varying functional forms of a given covariate coefficient, provided the the within-subject level was small.     

A nonparametric test for proving noninferiority in clinical trials with ordered categorical data

We consider the problem of proving noninferiority when the comparison is based on ordered categorical data. We apply a rank test based on the Wilcoxon–Mann–Whitney effect where the asymptotic variance is estimated consistently under the alternative and a small‐sample approximation is given. We give the associated 100(1?α)% confidence interval and propose a formula for sample size determination. Finally, we illustrate the procedure and possible choices of the noninferiority margin using data from a clinical trial. Copyright © 2003 John Wiley & Sons, Ltd.     

Imputation procedures for categorical data: their effects on the goodness-of-fit chi-square statistic

An imputation procedure is a procedure by which each missing value in a data set is replaced (imputed) by an observed value using a predetermined resampling procedure. The distribution of a statistic computed from a data set consisting of observed and imputed values, called a completed data set, is affecwd by the imputation procedure used. In a Monte Carlo experiment, three imputation procedures are compared with respect to the empirical behavior of the goodness-of- fit chi-square statistic computed from a completed data set. The results show that each imputation procedure affects the distribution of the goodness-of-fit chi-square statistic in 3. different manner. However, when the empirical behavior of the goodness-of-fit chi-square statistic is compared u, its appropriate asymptotic distribution, there are no substantial differences between these imputation procedures.     

Longitudinal conditional models with intermittent missingness: SAS code and applications

This work provides a set of macros performed with SAS (Statistical Analysis System) for Windows, which can be used to fit conditional models under intermittent missingness in longitudinal data. A formalized transition model, including random effects for individuals and measurement error, is presented. Model fitting is based on the missing completely at random or missing at random assumptions, and the separability condition. The problem translates to maximization of the marginal observed data density only, which for Gaussian data is again Gaussian, meaning that the likelihood can be expressed in terms of the mean and covariance matrix of the observed data vector. A simulation study is presented and misspecification issues are considered. A practical application is also given, where conditional models are fitted to the data from a clinical trial that assessed the effect of a Cuban medicine on a disease of the respiratory system.     

Joint regression modeling for missing categorical covariates in generalized linear models

Missing covariates data is a common issue in generalized linear models (GLMs). A model-based procedure arising from properly specifying joint models for both the partially observed covariates and the corresponding missing indicator variables represents a sound and flexible methodology, which lends itself to maximum likelihood estimation as the likelihood function is available in computable form. In this paper, a novel model-based methodology is proposed for the regression analysis of GLMs when the partially observed covariates are categorical. Pair-copula constructions are used as graphical tools in order to facilitate the specification of the high-dimensional probability distributions of the underlying missingness components. The model parameters are estimated by maximizing the weighted log-likelihood function by using an EM algorithm. In order to compare the performance of the proposed methodology with other well-established approaches, which include complete-cases and multiple imputation, several simulation experiments of Binomial, Poisson and Normal regressions are carried out under both missing at random and non-missing at random mechanisms scenarios. The methods are illustrated by modeling data from a stage III melanoma clinical trial. The results show that the methodology is rather robust and flexible, representing a competitive alternative to traditional techniques.     

Assessing goodness-of-fit of categorical regression models based on case-control data

Demonstrated equivalence between a categorical regression model based on case‐control data and an I‐sample semiparametric selection bias model leads to a new goodness‐of‐fit test. The proposed test statistic is an extension of an existing Kolmogorov–Smirnov‐type statistic and is the weighted average of the absolute differences between two estimated distribution functions in each response category. The paper establishes an optimal property for the maximum semiparametric likelihood estimator of the parameters in the I‐sample semiparametric selection bias model. It also presents a bootstrap procedure, some simulation results and an analysis of two real datasets.     

Two simple measures of variability for categorical data

This paper proposes two new variability measures for categorical data. The first variability measure is obtained as one minus the square root of the sum of the squares of the relative frequencies of the different categories. The second measure is obtained by standardizing the first measure. The measures proposed are functions of the variability measure proposed by Gini [Variabilitá e Mutuabilitá Contributo allo Studio delle Distribuzioni e delle Relazioni Statistiche, C. Cuppini, Bologna, 1912] and approximate the coefficient of nominal variation introduced by Kvålseth [Coefficients of variation for nominal and ordinal categorical data, Percept. Motor Skills 80 (1995), pp. 843–847] when the number of categories increases. Different mathematical properties of the proposed variability measures are studied and analyzed. Several examples illustrate how the variability measures can be interpreted and used in practice.     

An R package for model fitting,model selection and the simulation for longitudinal data with dropout missingness

Abstract

Missing data arise frequently in clinical and epidemiological fields, in particular in longitudinal studies. This paper describes the core features of an R package wgeesel, which implements marginal model fitting (i.e., weighted generalized estimating equations, WGEE; doubly robust GEE) for longitudinal data with dropouts under the assumption of missing at random. More importantly, this package comprehensively provide existing information criteria for WGEE model selection on marginal mean or correlation structures. Also, it can serve as a valuable tool for simulating longitudinal data with missing outcomes. Lastly, a real data example and simulations are presented to illustrate and validate our package.     

A Bayesian shared parameter model for joint modeling of longitudinal continuous and binary outcomes

Joint modeling of associated mixed biomarkers in longitudinal studies leads to a better clinical decision by improving the efficiency of parameter estimates. In many clinical studies, the observed time for two biomarkers may not be equivalent and one of the longitudinal responses may have recorded in a longer time than the other one. In addition, the response variables may have different missing patterns. In this paper, we propose a new joint model of associated continuous and binary responses by accounting different missing patterns for two longitudinal outcomes. A conditional model for joint modeling of the two responses is used and two shared random effects models are considered for intermittent missingness of two responses. A Bayesian approach using Markov Chain Monte Carlo (MCMC) is adopted for parameter estimation and model implementation. The validation and performance of the proposed model are investigated using some simulation studies. The proposed model is also applied for analyzing a real data set of bariatric surgery.     

Recurrent time-to-event models with ordinal outcomes

A model to accommodate time-to-event ordinal outcomes was proposed by Berridge and Whitehead. Very few studies have adopted this approach, despite its appeal in incorporating several ordered categories of event outcome. More recently, there has been increased interest in utilizing recurrent events to analyze practical endpoints in the study of disease history and to help quantify the changing pattern of disease over time. For example, in studies of heart failure, the analysis of a single fatal event no longer provides sufficient clinical information to manage the disease. Similarly, the grade/frequency/severity of adverse events may be more important than simply prolonged survival in studies of toxic therapies in oncology. We propose an extension of the ordinal time-to-event model to allow for multiple/recurrent events in the case of marginal models (where all subjects are at risk for each recurrence, irrespective of whether they have experienced previous recurrences) and conditional models (subjects are at risk of a recurrence only if they have experienced a previous recurrence). These models rely on marginal and conditional estimates of the instantaneous baseline hazard and provide estimates of the probabilities of an event of each severity for each recurrence over time. We outline how confidence intervals for these probabilities can be constructed and illustrate how to fit these models and provide examples of the methods, together with an interpretation of the results.     

Likelihood analysis of joint marginal and conditional models for longitudinal categorical data

The authors develop a Markov model for the analysis of longitudinal categorical data which facilitates modelling both marginal and conditional structures. A likelihood formulation is employed for inference, so the resulting estimators enjoy the optimal properties such as efficiency and consistency, and remain consistent when data are missing at random. Simulation studies demonstrate that the proposed method performs well under a variety of situations. Application to data from a smoking prevention study illustrates the utility of the model and interpretation of covariate effects. The Canadian Journal of Statistics © 2009 Statistical Society of Canada     

Jointly modeling skew longitudinal survival data with missingness and mismeasured covariates

Jointly modeling longitudinal and survival data has been an active research area. Most researches focus on improving the estimating efficiency but ignore many data features frequently encountered in practice. In the current study, we develop the joint models that concurrently accounting for longitudinal and survival data with multiple features. Specifically, the proposed model handles skewness, missingness and measurement errors in covariates which are typically observed in the collection of longitudinal survival data from many studies. We employ a Bayesian inferential method to make inference on the proposed model. We applied the proposed model to an real data study. A few alternative models under different conditions are compared. We conduct extensive simulations in order to evaluate how the method works.