A family of models for uniform and serial dependence in repeated measurements studies

Data arising from a randomized double-masked clinical trial for multiple sclerosis have provided particularly variable longitudinal repeated measurements responses. Specific models for such data, other than those based on the multivariate normal distribution, would be a valuable addition to the applied statistician's toolbox. A useful family of multivariate distributions can be generated by substituting the integrated intensity of one distribution into a second (outer) distribution. The parameters in the second distribution are then used to create a dependence structure among observations on a unit. These may either be a form of serial dependence for longitudinal data or of uniform dependence within clusters. These are respectively analogous to the Kalman filter of state space models and to copulas, but they have the major advantage that they do not require any explicit integration. One useful outer distribution for constructing such multivariate distributions is the Pareto distribution. Certain special models based on it have previously been used in event history analysis, but those considered here have much wider application.     

A latent variable model for analyzing mixed longitudinal (k,l)-inflated count and ordinal responses

A random effects model for analyzing mixed longitudinal count and ordinal data is presented where the count response is inflated in two points (k and l) and an (k,l)-Inflated Power series distribution is used as its distribution. A full likelihood-based approach is used to obtain maximum likelihood estimates of parameters of the model. For data with non-ignorable missing values models with probit model for missing mechanism are used.The dependence between longitudinal sequences of responses and inflation parameters are investigated using a random effects approach. Also, to investigate the correlation between mixed ordinal and count responses of each individuals at each time, a shared random effect is used. In order to assess the performance of the model, a simulation study is performed for a case that the count response has (k,l)-Inflated Binomial distribution. Performance comparisons of count-ordinal random effect model, Zero-Inflated ordinal random effects model and (k,l)-Inflated ordinal random effects model are also given. The model is applied to a real social data set from the first two waves of the national longitudinal study of adolescent to adult health (Add Health study). In this data set, the joint responses are the number of days in a month that each individual smoked as the count response and the general health condition of each individual as the ordinal response. For the count response there is incidence of excess values of 0 and 30.     

General mixed‐data model: Extension of general location and grouped continuous models

The authors propose a general model for the joint distribution of nominal, ordinal and continuous variables. Their work is motivated by the treatment of various types of data. They show how to construct parameter estimates for their model, based on the maximization of the full likelihood. They provide algorithms to implement it, and present an alternative estimation method based on the pairwise likelihood approach. They also touch upon the issue of statistical inference. They illustrate their methodology using data from a foreign language achievement study.     

Bivariate transition model for analysing ordinal and nominal categorical responses: an application to the Labour Force Survey data

In many panel studies, bivariate ordinal–nominal responses are measured and the aim is to investigate the effects of explanatory variables on these responses. A regression analysis for these types of data must allow for the correlation among responses of the same individual. To analyse such ordinal–nominal responses using a proper weighting approach, an ordinal–nominal bivariate transition model is proposed and maximum likelihood is used to find the parameter estimates. We propose a method in which the likelihood function can be partitioned to make possible the use of existing software. The approach is applied to the Labour Force Survey data in Iran, where the ordinal response, at the first period, is the duration of unemployment for unemployed people and the nominal response, in the second period, is economic activity status of these individuals. The interest is to find the reasons for staying unemployed or moving to another status of economic activity.     

Modeling a mixture of ordinal and continuous repeated measures

We study the correlation structure for a mixture of ordinal and continuous repeated measures using a Bayesian approach. We assume a multivariate probit model for the ordinal variables and a normal linear regression for the continuous variables, where latent normal variables underlying the ordinal data are correlated with continuous variables in the model. Due to the probit model assumption, we are required to sample a covariance matrix with some of the diagonal elements equal to one. The key computational idea is to use parameter-extended data augmentation, which involves applying the Metropolis-Hastings algorithm to get a sample from the posterior distribution of the covariance matrix incorporating the relevant restrictions. The methodology is illustrated through a simulated example and through an application to data from the UCLA Brain Injury Research Center.     

Bayesian quantile regression for skew-normal linear mixed models

Linear mixed models have been widely used to analyze repeated measures data which arise in many studies. In most applications, it is assumed that both the random effects and the within-subjects errors are normally distributed. This can be extremely restrictive, obscuring important features of within-and among-subject variations. Here, quantile regression in the Bayesian framework for the linear mixed models is described to carry out the robust inferences. We also relax the normality assumption for the random effects by using a multivariate skew-normal distribution, which includes the normal ones as a special case and provides robust estimation in the linear mixed models. For posterior inference, we propose a Gibbs sampling algorithm based on a mixture representation of the asymmetric Laplace distribution and multivariate skew-normal distribution. The procedures are demonstrated by both simulated and real data examples.     

Nonlinear mixed effects modelling for estimation of steady state attainment

Assessment of the time needed to attain steady state is a key pharmacokinetic objective during drug development. Traditional approaches for assessing steady state include ANOVA‐based methods for comparing mean plasma concentration values from each sampling day, with either a difference or equivalence test. However, hypothesis‐testing approaches are ill suited for assessment of steady state. This paper presents a nonlinear mixed effects modelling approach for estimation of steady state attainment, based on fitting a simple nonlinear mixed model to observed trough plasma concentrations. The simple nonlinear mixed model is developed and proposed for use under certain pharmacokinetic assumptions. The nonlinear mixed modelling estimation approach is described and illustrated by application to trough data from a multiple dose trial in healthy subjects. The performance of the nonlinear mixed modelling approach is compared to ANOVA‐based approaches by means of simulation techniques. Copyright © 2005 John Wiley & Sons, Ltd.     

Distributional properties of a model for the spread or drug abuse

We examine the properties of the distribution of the number of drug abusers in a previously free community assuming that initiators enter the community during a ‘latent’ period in which they randomly infect other members of the community, and that during the subsequent ‘control’ period the spread of abuse follows a linear birth and death process. The form of distribution is shown to be unaltered by a series of steady changes in the birth and death parameters. The distribution can be regarded as the convolution of three distributions:pseudo-binomial or binomial, negative binomial, and Polya-Aeppli. Special cases include the Laguerre series distribution.     

Joint modeling of mixed skewed continuous and ordinal longitudinal responses: a Bayesian approach

In this paper, a joint model for analyzing multivariate mixed ordinal and continuous responses, where continuous outcomes may be skew, is presented. For modeling the discrete ordinal responses, a continuous latent variable approach is considered and for describing continuous responses, a skew-normal mixed effects model is used. A Bayesian approach using Markov Chain Monte Carlo (MCMC) is adopted for parameter estimation. Some simulation studies are performed for illustration of the proposed approach. The results of the simulation studies show that the use of the separate models or the normal distributional assumption for shared random effects and within-subject errors of continuous and ordinal variables, instead of the joint modeling under a skew-normal distribution, leads to biased parameter estimates. The approach is used for analyzing a part of the British Household Panel Survey (BHPS) data set. Annual income and life satisfaction are considered as the continuous and the ordinal longitudinal responses, respectively. The annual income variable is severely skewed, therefore, the use of the normality assumption for the continuous response does not yield acceptable results. The results of data analysis show that gender, marital status, educational levels and the amount of money spent on leisure have a significant effect on annual income, while marital status has the highest impact on life satisfaction.     

General location multivariate latent variable models for mixed correlated bounded continuous,ordinal, and nominal responses with non-ignorable missing data

Using a multivariate latent variable approach, this article proposes some new general models to analyze the correlated bounded continuous and categorical (nominal or/and ordinal) responses with and without non-ignorable missing values. First, we discuss regression methods for jointly analyzing continuous, nominal, and ordinal responses that we motivated by analyzing data from studies of toxicity development. Second, using the beta and Dirichlet distributions, we extend the models so that some bounded continuous responses are replaced for continuous responses. The joint distribution of the bounded continuous, nominal and ordinal variables is decomposed into a marginal multinomial distribution for the nominal variable and a conditional multivariate joint distribution for the bounded continuous and ordinal variables given the nominal variable. We estimate the regression parameters under the new general location models using the maximum-likelihood method. Sensitivity analysis is also performed to study the influence of small perturbations of the parameters of the missing mechanisms of the model on the maximal normal curvature. The proposed models are applied to two data sets: BMI, Steatosis and Osteoporosis data and Tehran household expenditure budgets.     

Statistical Analysis of Heat Waves in the State of Victoria in Australia

The recent blistering heat waves of 2009 in the state of Victoria in Australia were so unprecedented in terms of duration and intensity that society was largely unprepared. These heat waves caused serious health, social and economic problems. In this paper, the daily maximum temperatures at ten selected stations are studied. Auto‐regressive integrated moving‐average models are used to prewhiten the time series. Uncorrelated, non‐normal and heavy‐tailed residuals are analyzed by means of a new skew t‐mixture distribution. The number of mixture components is effectively determined by an innovative penalisation procedure. It is shown that the resulting skew t‐mixture models provide an acceptable fit in all cases. Possible future temperature patterns are obtained through simulation. It is forecast that the average duration of high temperature episodes will increase by two to three days per year and a new eight‐year high temperature level is very likely in the coming few years. The relationship between heavy tail behaviour of the fitted distribution and heat waves is noteworthy.     

The dynamics of perception: modelling subjective wellbeing in a short panel

Summary.  We consider the issue of the dynamics of perceptions, as expressed in responses to survey questions on subjective wellbeing. We develop a simulated maximum likelihood method for estimation of dynamic linear models, where the dependent variable is partially observed through ordinal scales. This latent auto-regression model is often more appropriate than the usual state dependence model for attitudinal and interval variables. The paper contains an application to a model of households' perceptions of their financial wellbeing, demonstrating the superior fit of the latent auto-regression model to both the usual static model and the state dependence model.     

A goodness of fit test for the multilevel proportional odds model

It is essential to test the goodness of fit of the model before making inferences based on it. Multilevel modeling of ordinal categorical responses is not as developed as for continuous responses. Assessing model adequacy in terms of the goodness of fit with ordinal categorical responses is still being developed and no satisfactory tests are available so far. As a consequence of that, this study concentrates on developing such a goodness of fit test for Multilevel Proportional Odds models and to study the properties of the test.     

A Bayesian conditional model for bivariate mixed ordinal and skew continuous longitudinal responses using quantile regression

In this paper, we develop a conditional model for analyzing mixed bivariate continuous and ordinal longitudinal responses. We propose a quantile regression model with random effects for analyzing continuous responses. For this purpose, an Asymmetric Laplace Distribution (ALD) is allocated for continuous response given random effects. For modeling ordinal responses, a cumulative logit model is used, via specifying a latent variable model, with considering other random effects. Therefore, the intra-association between continuous and ordinal responses is taken into account using their own exclusive random effects. But, the inter-association between two mixed responses is taken into account by adding a continuous response term in the ordinal model. We use a Bayesian approach via Markov chain Monte Carlo method for analyzing the proposed conditional model and to estimate unknown parameters, a Gibbs sampler algorithm is used. Moreover, we illustrate an application of the proposed model using a part of the British Household Panel Survey data set. The results of data analysis show that gender, age, marital status, educational level and the amount of money spent on leisure have significant effects on annual income. Also, the associated parameter is significant in using the best fitting proposed conditional model, thus it should be employed rather than analyzing separate models.     

Statistical models for the analysis of controlled trials on acute migraine

Specific efficacy criteria were defined by the International Headache Society for controlled clinical trials on acute migraine. They are derived from the pain profile and the timing of rescue medication intake. We present a methodology to improve the analysis of such trials. Instead of analysing each endpoint separately, we model the joint distribution and derive success rates in any criteria as predictions. We use cumulative regression models for each response at a time and a multivariate normal copula to model the dependence between responses. Parameters are estimated using maximum likelihood. Benefits of the method include a reduction in the number of tests performed and an increase in their power. The method is well suited to dose–response trials from which predictions can be used to select doses and optimize the design of subsequent trials. More generally, our method permits a very flexible modelling of longitudinal series of ordinal data. Copyright © 2003 John Wiley & Sons, Ltd.     

On Modelling Agreement and Category Distinguishability on an Ordinal Scale

It is quite common that raters may need to classify a sample of subjects on a categorical scale. Perfect agreement can rarely be observed partly because of different perceptions about the meanings of the category labels between raters and partly because of factors such as intrarater variability. Usually, category indistinguishability occurs between adjacent categories. In this article, we propose a simple log-linear model combining ordinal scale information and category distinguishability between ordinal categories for modelling agreement between two raters. For the proposed model, no score assignment is required to the ordinal categories. An algorithm and statistical properties will be provided.     

Model-based clustering of Gaussian copulas for mixed data

Clustering of mixed data is important yet challenging due to a shortage of conventional distributions for such data. In this article, we propose a mixture model of Gaussian copulas for clustering mixed data. Indeed copulas, and Gaussian copulas in particular, are powerful tools for easily modeling the distribution of multivariate variables. This model clusters data sets with continuous, integer, and ordinal variables (all having a cumulative distribution function) by considering the intra-component dependencies in a similar way to the Gaussian mixture. Indeed, each component of the Gaussian copula mixture produces a correlation coefficient for each pair of variables and its univariate margins follow standard distributions (Gaussian, Poisson, and ordered multinomial) depending on the nature of the variable (continuous, integer, or ordinal). As an interesting by-product, this model generalizes many well-known approaches and provides tools for visualization based on its parameters. The Bayesian inference is achieved with a Metropolis-within-Gibbs sampler. The numerical experiments, on simulated and real data, illustrate the benefits of the proposed model: flexible and meaningful parameterization combined with visualization features.     

Hierarchical marginal models with latent uncertainty

In responding to a rating question, an individual may give answers either according to his/her knowledge/awareness or to his/her level of indecision/uncertainty, typically driven by a response style. As ignoring this dual behavior may lead to misleading results, we define a multivariate model for ordinal rating responses by introducing, for every item and every respondent, a binary latent variable that discriminates aware from uncertain responses. Some independence assumptions among latent and observable variables characterize the uncertain behavior and make the model easier to interpret. Uncertain responses are modeled by specifying probability distributions that can depict different response styles. A marginal parameterization allows a simple and direct interpretation of the parameters in terms of association among aware responses and their dependence on explanatory factors. The effectiveness of the proposed model is attested through an application to real data and supported by a Monte Carlo study.     

Testing the proportional odds assumption in multiply imputed ordinal longitudinal data

A popular choice when analyzing ordinal data is to consider the cumulative proportional odds model to relate the marginal probabilities of the ordinal outcome to a set of covariates. However, application of this model relies on the condition of identical cumulative odds ratios across the cut-offs of the ordinal outcome; the well-known proportional odds assumption. This paper focuses on the assessment of this assumption while accounting for repeated and missing data. In this respect, we develop a statistical method built on multiple imputation (MI) based on generalized estimating equations that allows to test the proportionality assumption under the missing at random setting. The performance of the proposed method is evaluated for two MI algorithms for incomplete longitudinal ordinal data. The impact of both MI methods is compared with respect to the type I error rate and the power for situations covering various numbers of categories of the ordinal outcome, sample sizes, rates of missingness, well-balanced and skewed data. The comparison of both MI methods with the complete-case analysis is also provided. We illustrate the use of the proposed methods on a quality of life data from a cancer clinical trial.     

Modelling Uncertainty and Overdispersion in Ordinal Data

In this article we introduce a probability distribution generated by a mixture of discrete random variables to capture uncertainty, feeling, and overdispersion, possibly present in ordinal data surveys. The choice of the components of the new model is motivated by a study on the data generating process. Inferential issues concerning the maximum likelihood estimates and the validation steps are presented; then, some empirical analyses are given to support the usefulness of the approach. Discussion on further extensions of the model ends the article.