Joint modelling of longitudinal response and time-to-event data using conditional distributions: a Bayesian perspective

Over the last 20 or more years a lot of clinical applications and methodological development in the area of joint models of longitudinal and time-to-event outcomes have come up. In these studies, patients are followed until an event, such as death, occurs. In most of the work, using subject-specific random-effects as frailty, the dependency of these two processes has been established. In this article, we propose a new joint model that consists of a linear mixed-effects model for longitudinal data and an accelerated failure time model for the time-to-event data. These two sub-models are linked via a latent random process. This model will capture the dependency of the time-to-event on the longitudinal measurements more directly. Using standard priors, a Bayesian method has been developed for estimation. All computations are implemented using OpenBUGS. Our proposed method is evaluated by a simulation study, which compares the conditional model with a joint model with local independence by way of calibration. Data on Duchenne muscular dystrophy (DMD) syndrome and a set of data in AIDS patients have been analysed.     

The social contract revisited: a re-examination of the influence individualistic and collectivistic value systems have on the psychological wellbeing of young people

ABSTRACT

The prevalence of psychological health problems experienced by young people living in Western societies is increasing. Evidence suggests the cultural dynamism of individualism may play a role in this, but this evidence is conflicting. Here, we focus on both the concepts of individualism and collectivism, distinguishing between their horizontal and vertical dimensions. We examine the influence of these dimensions on the psychological wellbeing of a sample of 507 Australian emerging adults (aged 18–25). We found that orientations towards vertical (but not horizontal) individualism predicted lower levels of psychological wellbeing, while orientations towards horizontal (but not vertical) collectivism predicted higher psychological wellbeing. These findings add clarity to the way in which key Western social values play an understated role in the increasing prevalence of psychological health problems experienced by young people today. They also provide an understanding of how various traits embedded within these concepts relate to psychological wellbeing.     

Validation of surrogate end points in multiple randomized clinical trials with failure time end points

Before a surrogate end point can replace a final (true) end point in the evaluation of an experimental treatment, it must be formally 'validated'. The validation will typically require large numbers of observations. It is therefore useful to consider situations in which data are available from several randomized experiments. For two normally distributed end points Buyse and co-workers suggested a new definition of validity in terms of the quality of both trial level and individual level associations between the surrogate and true end points. This paper extends this approach to the important case of two failure time end points, using bivariate survival modelling. The method is illustrated by using two actual sets of data from cancer clinical trials.     

Simple Fitting Algorithms for Incomplete Categorical Data

A popular approach to estimation based on incomplete data is the EM algorithm. For categorical data, this paper presents a simple expression of the observed data log-likelihood and its derivatives in terms of the complete data for a broad class of models and missing data patterns. We show that using the observed data likelihood directly is easy and has some advantages. One can gain considerable computational speed over the EM algorithm and a straightforward variance estimator is obtained for the parameter estimates. The general formulation treats a wide range of missing data problems in a uniform way. Two examples are worked out in full.     

High dimensional multivariate mixed models for binary questionnaire data

Summary.  Questionnaires that are used to measure the effect of an intervention often consist of different sets of items, each set possibly measuring another concept. Mixed models with set-specific random effects are a flexible tool to model the different sets of items jointly. However, computational problems typically arise as the number of sets increases. This is especially true when the random-effects distribution cannot be integrated out analytically, as with mixed models for binary data. A pairwise modelling strategy, in which all possible bivariate mixed models are fitted and where inference follows from pseudolikelihood theory, has been proposed as a solution. This approach has been applied to assess the effect of physical activity on psychocognitive functioning, the latter measured by a battery of questionnaires.     

Measuring Young Children's Perceptions of Support, Control, and Maintenance in Their Own Social Networks

Thirty‐eight children (aged 3;7–7;6) and one of their parents took part in a study concerning children's perceptions of their social networks. The study made use of a newly developed instrument—the Support, Control and Maintenance Pictures Interview (SCAMPI). The SCAMPI offers an individualized testing environment, employing computer presentation of questions based on photographs of significant persons familiar to the child. SCAMPI is designed to allow data analysis to be carried out with the aid of built‐in statistical procedures based on permutation and bootstrap techniques that are optimally adapted to the requirements of individualized testing. The study examines the differentiations young children make between persons in their social networks and the functions they fulfil, the level of agreement between children and their parents and the stability of the children's responses.     

Marginal Correlation from Logit- and Probit-Beta-Normal Models for Hierarchical Binary Data

In hierarchical data settings, be it of a longitudinal, spatial, multi-level, clustered, or otherwise repeated nature, often the association between repeated measurements attracts at least part of the scientific interest. Quantifying the association frequently takes the form of a correlation function, including but not limited to intraclass correlation. Vangeneugden et al. (2010 Vangeneugden, T., Molenberghs, G., Laenen, A., Geys, H., Beunckens, C., Sotto, C. (2010). Marginal correlation in longitudinal binary data based on generalized linear mixed models. Communi. Stati. Theory &; Methods. 39:3542–3557. [Google Scholar]) derived approximate correlation functions for longitudinal sequences of general data type, Gaussian and non-Gaussian, based on generalized linear mixed-effects models. Here, we consider the extended model family proposed by Molenberghs et al. (2010 Molenberghs, G., Verbeke, G., Demétrio, C., Vieira, A. (2010). A family of generalized linear models for repeated measures with normal and conjugate random effects. Stat. Sci. 25:325–347.[Crossref], [Web of Science ®] , [Google Scholar]). This family flexibly accommodates data hierarchies, intra-sequence correlation, and overdispersion. The family allows for closed-form means, variance functions, and correlation function, for a variety of outcome types and link functions. Unfortunately, for binary data with logit link, closed forms cannot be obtained. This is in contrast with the probit link, for which such closed forms can be derived. It is therefore that we concentrate on the probit case. It is of interest, not only in its own right, but also as an instrument to approximate the logit case, thanks to the well-known probit-logit ‘conversion.’ Next to the general situation, some important special cases such as exchangeable clustered outcomes receive attention because they produce insightful expressions. The closed-form expressions are contrasted with the generic approximate expressions of Vangeneugden et al. (2010 Vangeneugden, T., Molenberghs, G., Laenen, A., Geys, H., Beunckens, C., Sotto, C. (2010). Marginal correlation in longitudinal binary data based on generalized linear mixed models. Communi. Stati. Theory &; Methods. 39:3542–3557. [Google Scholar]) and with approximations derived for the so-called logistic-beta-normal combined model. A simulation study explores performance of the method proposed. Data from a schizophrenia trial are analyzed and correlation functions derived.     

Empirical Bayes estimates for correlated hierarchical data with overdispersion

An extension of the generalized linear mixed model was constructed to simultaneously accommodate overdispersion and hierarchies present in longitudinal or clustered data. This so‐called combined model includes conjugate random effects at observation level for overdispersion and normal random effects at subject level to handle correlation, respectively. A variety of data types can be handled in this way, using different members of the exponential family. Both maximum likelihood and Bayesian estimation for covariate effects and variance components were proposed. The focus of this paper is the development of an estimation procedure for the two sets of random effects. These are necessary when making predictions for future responses or their associated probabilities. Such (empirical) Bayes estimates will also be helpful in model diagnosis, both when checking the fit of the model as well as when investigating outlying observations. The proposed procedure is applied to three datasets of different outcome types. Copyright © 2014 John Wiley & Sons, Ltd.