Factors that influence reading and comprehension of branching instructions in self–administered questionnaires

Summary: In this paper we examine the tendency for branching instructions to be ignored, misread, or otherwise not appropriately followed so that item nonresponse occurs for follow–up questions. The potential influence on branching errors of seven features of question complexity are examined, including high number of question words, high number of answer categories, last categories branch, all categories branch, write–in responses, location at the bottom of a page, and high distance between the answer box and branching instruction. A logistic regression analysis revealed that question complexity had a tendency to increase certain errors, but not others.* A more detailed version of this paper with additional analysis and discussion is available at http://www.sesrc.wsu.edu/dillman/. The opinions expressed here are those of the authors, not necessarily of the institutions where they presently work or the U.S. Census Bureau, which provided financial support for the collection of these data. We would like to thank Aref Dajani and Yves Thibaudeau for their advice on the analysis used in this paper.     

Bayesian Survival Analysis Using Bernstein Polynomials

Abstract.  Bayesian survival analysis of right-censored survival data is studied using priors on Bernstein polynomials and Markov chain Monte Carlo methods. These priors easily take into consideration geometric information like convexity or initial guess on the cumulative hazard functions, select only smooth functions, can have large enough support, and can be easily specified and generated. Certain frequentist asymptotic properties of the posterior distribution are established. Simulation studies indicate that these Bayes methods are quite satisfactory.     

Assessing accuracy of a continuous screening test in the presence of verification bias

Summary.  In studies to assess the accuracy of a screening test, often definitive disease assessment is too invasive or expensive to be ascertained on all the study subjects. Although it may be more ethical or cost effective to ascertain the true disease status with a higher rate in study subjects where the screening test or additional information is suggestive of disease, estimates of accuracy can be biased in a study with such a design. This bias is known as verification bias. Verification bias correction methods that accommodate screening tests with binary or ordinal responses have been developed; however, no verification bias correction methods exist for tests with continuous results. We propose and compare imputation and reweighting bias-corrected estimators of true and false positive rates, receiver operating characteristic curves and area under the receiver operating characteristic curve for continuous tests. Distribution theory and simulation studies are used to compare the proposed estimators with respect to bias, relative efficiency and robustness to model misspecification. The bias correction estimators proposed are applied to data from a study of screening tests for neonatal hearing loss.     

Generalized additive models for location, scale and shape

Summary.  A general class of statistical models for a univariate response variable is presented which we call the generalized additive model for location, scale and shape (GAMLSS). The model assumes independent observations of the response variable y given the parameters, the explanatory variables and the values of the random effects. The distribution for the response variable in the GAMLSS can be selected from a very general family of distributions including highly skew or kurtotic continuous and discrete distributions. The systematic part of the model is expanded to allow modelling not only of the mean (or location) but also of the other parameters of the distribution of y , as parametric and/or additive nonparametric (smooth) functions of explanatory variables and/or random-effects terms. Maximum (penalized) likelihood estimation is used to fit the (non)parametric models. A Newton–Raphson or Fisher scoring algorithm is used to maximize the (penalized) likelihood. The additive terms in the model are fitted by using a backfitting algorithm. Censored data are easily incorporated into the framework. Five data sets from different fields of application are analysed to emphasize the generality of the GAMLSS class of models.     

Bayesian model selection for join point regression with application to age-adjusted cancer rates

Summary.  The method of Bayesian model selection for join point regression models is developed. Given a set of K +1 join point models M ₀,  M ₁, …,  M _K with 0, 1, …,  K join points respec-tively, the posterior distributions of the parameters and competing models M _k are computed by Markov chain Monte Carlo simulations. The Bayes information criterion BIC is used to select the model M _k with the smallest value of BIC as the best model. Another approach based on the Bayes factor selects the model M _k with the largest posterior probability as the best model when the prior distribution of M _k is discrete uniform. Both methods are applied to analyse the observed US cancer incidence rates for some selected cancer sites. The graphs of the join point models fitted to the data are produced by using the methods proposed and compared with the method of Kim and co-workers that is based on a series of permutation tests. The analyses show that the Bayes factor is sensitive to the prior specification of the variance σ ², and that the model which is selected by BIC fits the data as well as the model that is selected by the permutation test and has the advantage of producing the posterior distribution for the join points. The Bayesian join point model and model selection method that are presented here will be integrated in the National Cancer Institute's join point software ( http://www.srab.cancer.gov/joinpoint/ ) and will be available to the public.     

Estimation of non-parametric regression for dasometric measures

The aim of this paper is to describe a simulation procedure to compare parametric regression against a non-parametric regression method, for different functions and sets of information. The proposed methodology improves lack of fit at the edges of the regression curves, and an acceptable result is obtained for the no-parametric estimation in all studied cases. Larger differences appear at the edges of the estimation. The results are applied to the study of dasometric variables, which do not fulfil the normality hypothesis needed for parametric estimation. The kernel regression shows the relationship between the studied variables, which would not be detected with more rigid parametric models.     

Pooling multivariate data under W, LR and LM tests

Two independent random samples are drawn from two multivariate normal populations with mean vectors μ1 and μ2 and a common variance-covariance matrix Σ. Ahmed and Saleh (1990) considered preliminary test maximum likelihood estimator (PMLTE) for estimating μ1 based on the Hotelling's T _N ², when it is suspected that μ1=μ2. In this paper, the PTMLE based on the Wald (W), Likelihood Ratio (LR) and Lagrangian Multiplier (LM) tests are considered. Using the quadratic risk function, the conditions of superiority of the proposed estimator for departure parameter are derived. A max-min rule for the size of the preliminary test of significance is presented. It is demonstrated that the PTMLE based on W test produces the highest minimum guaranteed efficiencies compared to UMLE among the three test procedures.     

Effects of unmeasured heterogeneity in the linear transformation model for censored data

We investigate the effect of unobserved heterogeneity in the context of the linear transformation model for censored survival data in the clinical trials setting. The unobserved heterogeneity is represented by a frailty term, with unknown distribution, in the linear transformation model. The bias of the estimate under the assumption of no unobserved heterogeneity when it truly is present is obtained. We also derive the asymptotic relative efficiency of the estimate of treatment effect under the incorrect assumption of no unobserved heterogeneity. Additionally we investigate the loss of power for clinical trials that are designed assuming the model without frailty when, in fact, the model with frailty is true. Numerical studies under a proportional odds model show that the loss of efficiency and the loss of power can be substantial when the heterogeneity, as embodied by a frailty, is ignored. An erratum to this article can be found at     

A computationally simple bivariate survival estimator for efficacy and safety

Both treatment efficacy and safety are typically the primary endpoints in Phase II, and even in some Phase III, clinical trials. Efficacy is frequently measured by time to response, death, or some other milestone event and thus is a continuous, possibly censored, outcome. Safety, however, is frequently measured on a discrete scale; in Eastern Cooperative Oncology Group clinical trial E2290, it was measured as the number of weekly rounds of chemotherapy that were tolerable to colorectal cancer patients. For the joint analysis of efficacy and safety, we propose a non-parametric, computationally simple estimator for the bivariate survival function when one time-to-event is continuous, one is discrete, and both are subject to right-censoring. The bivariate censoring times may depend on each other, but they are assumed to be independent of both event times. We derive a closed-form covariance estimator for the survivor function which allows for inference to be based on any of several possible statistics of interest. In addition, we derive its covariance with respect to calendar time of analysis, allowing for its use in sequential studies.