Linear Increments with Non‐monotone Missing Data and Measurement Error

Linear increments (LI) are used to analyse repeated outcome data with missing values. Previously, two LI methods have been proposed, one allowing non‐monotone missingness but not independent measurement error and one allowing independent measurement error but only monotone missingness. In both, it was suggested that the expected increment could depend on current outcome. We show that LI can allow non‐monotone missingness and either independent measurement error of unknown variance or dependence of expected increment on current outcome but not both. A popular alternative to LI is a multivariate normal model ignoring the missingness pattern. This gives consistent estimation when data are normally distributed and missing at random (MAR). We clarify the relation between MAR and the assumptions of LI and show that for continuous outcomes multivariate normal estimators are also consistent under (non‐MAR and non‐normal) assumptions not much stronger than those of LI. Moreover, when missingness is non‐monotone, they are typically more efficient.     

A martingale residual diagnostic for longitudinal and recurrent event data

One method of assessing the fit of an event history model is to plot the empirical standard deviation of standardised martingale residuals. We develop an alternative procedure which is valid also in the presence of measurement error and applicable to both longitudinal and recurrent event data. Since the covariance between martingale residuals at times t ₀ and t > t ₀ is independent of t, a plot of these covariances should, for fixed t ₀, have no time trend. A test statistic is developed from the increments in the estimated covariances, and we investigate its properties under various types of model misspecification. Applications of the approach are presented using two Brazilian studies measuring daily prevalence and incidence of infant diarrhoea and a longitudinal study into treatment of schizophrenia.     

Plaid designs for the evaluation of training for medical practitioners

The training of medical practitioners to improve the practitioner/patient relationship may be difficult, as limitations often exist on the choice of patients included in the study. A specific study of this type of training is given. It is proposed that a simple modification and generalization of Yates' plaid-square designs be used. It is shown that a replicated plaid-design incorporates as a special case the criss-cross or strip-plot design. The usefulness of these designs in studies of the training of medical practitioners is illustrated. The basic characteristics of their analysis are outlined.     

Conditional inference for subject-specific and marginal agreement: Two families of agreement measures

A two-stage procedure is described for assessing subject-specific and marginal agreement for data from a test-retest reliability study of a binary classification procedure. Subject-specific agreement is parametrized through the log odds ratio, while marginal agreement is reflected by the log ratio of the off-diagonal Poisson means. A family of agreement measures in the interval [-1, 1] is presented for both types of agreement. The conditioning argument described facilitates exact inference. The proposed methodology is demonstrated by way of an example involving hypothetical data chosen for illustrative purposes, and data from a National Health Survey Study (Rogot and Goldberg 1966).     

A comparison of regression models for ordinal data in an analysis of transplanted-kidney function

Three regression models for ordinal data, those of Fienberg, McCullagh, and Anderson, are applied to an analysis of kidney function among transplant recipients. The conclusions arising from each model are presented and contrasted.     

The versatility of multi-state models for the analysis of longitudinal data with unobservable features

Multi-state models provide a convenient statistical framework for a wide variety of medical applications characterized by multiple events and longitudinal data. We illustrate this through four examples. The potential value of the incorporation of unobserved or partially observed states is highlighted. In addition, joint modelling of multiple processes is illustrated with application to potentially informative loss to follow-up, mis-measured or missclassified data and causal inference.     

Partially hidden multi-state modelling of a prolonged disease state defined by a composite outcome

Lifetime Data Analysis - For rheumatic diseases, Minimal Disease Activity (MDA) is usually defined as a composite outcome which is a function of several individual outcomes describing symptoms or...     

Analysis of longitudinal data with drop-out: objectives, assumptions and a proposal

Summary. The problem of analysing longitudinal data that are complicated by possibly informative drop-out has received considerable attention in the statistical literature. Most researchers have concentrated on either methodology or application, but we begin this paper by arguing that more attention could be given to study objectives and to the relevant targets for inference. Next we summarize a variety of approaches that have been suggested for dealing with drop-out. A long-standing concern in this subject area is that all methods require untestable assumptions. We discuss circumstances in which we are willing to make such assumptions and we propose a new and computationally efficient modelling and analysis procedure for these situations. We assume a dynamic linear model for the expected increments of a constructed variable, under which subject-specific random effects follow a martingale process in the absence of drop-out. Informal diagnostic procedures to assess the tenability of the assumption are proposed. The paper is completed by simulations and a comparison of our method and several alternatives in the analysis of data from a trial into the treatment of schizophrenia, in which approximately 50% of recruited subjects dropped out before the final scheduled measurement time.     

Mixture models in survival analysis: Are they worth the risk?

There has been a recurring interest in models for survival data which hypothesize subpopulations of individuals highly susceptible to some type of adverse event. Other individuals are assumed to be at much less risk. Most commonly, in clinical trials, these models attempt to estimate the fraction of patients cured of disease. The use of such models is examined, and the likelihood function is advocated as an informative inference tool.     

An overview of risk-adjusted charts

Summary.  The paper provides an overview of risk-adjusted charts, with examples based on two data sets: the first consisting of outcomes following cardiac surgery and patient factors contributing to the Parsonnet score; the second being age–sex-adjusted death-rates per year under a single general practitioner. Charts presented include the cumulative sum (CUSUM), resetting sequential probability ratio test, the sets method and Shewhart chart. Comparisons between the charts are made. Estimation of the process parameter and two-sided charts are also discussed. The CUSUM is found to be the least efficient, under the average run length (ARL) criterion, of the resetting sequential probability ratio test class of charts, but the ARL criterion is thought not to be sensible for comparisons within that class. An empirical comparison of the sets method and CUSUM, for binary data, shows that the sets method is more efficient when the in-control ARL is small and more efficient for a slightly larger range of in-control ARLs when the change in parameter being tested for is larger. The Shewart p -chart is found to be less efficient than the CUSUM even when the change in parameter being tested for is large.