Hypothesis test for paired samples in the presence of missing data

Missing data are present in almost all statistical analysis. In simple paired design tests, when some subject has one of the involved variables missing in the so-called partially overlapping samples scheme, it is usually discarded for the analysis. The lack of consistency between the information reported in the univariate and multivariate analysis is, perhaps, the main consequence. Although the randomness on the missing mechanism (missingness completely at random) is an usual and needed assumption for this particular situation, missing data presence could lead to serious inconsistencies on the reported conclusions. In this paper, the authors develop a simple and direct procedure which allows using the whole available information in order to perform paired tests. In particular, the proposed methodology is applied to check the equality among the means from two paired samples. In addition, the use of two different resampling techniques is also explored. Finally, real-world data are analysed.     

Robust location estimation with missing data

In a missing data setting, we have a sample in which a vector of explanatory variables ${\bf x}_i$ is observed for every subject i, while scalar responses $y_i$ are missing by happenstance on some individuals. In this work we propose robust estimators of the distribution of the responses assuming missing at random (MAR) data, under a semiparametric regression model. Our approach allows the consistent estimation of any weakly continuous functional of the response's distribution. In particular, strongly consistent estimators of any continuous location functional, such as the median, L‐functionals and M‐functionals, are proposed. A robust fit for the regression model combined with the robust properties of the location functional gives rise to a robust recipe for estimating the location parameter. Robustness is quantified through the breakdown point of the proposed procedure. The asymptotic distribution of the location estimators is also derived. The proofs of the theorems are presented in Supplementary Material available online. The Canadian Journal of Statistics 41: 111–132; 2013 © 2012 Statistical Society of Canada     

A structured framework for assessing sensitivity to missing data assumptions in longitudinal clinical trials

The objective of this research was to demonstrate a framework for drawing inference from sensitivity analyses of incomplete longitudinal clinical trial data via a re‐analysis of data from a confirmatory clinical trial in depression. A likelihood‐based approach that assumed missing at random (MAR) was the primary analysis. Robustness to departure from MAR was assessed by comparing the primary result to those from a series of analyses that employed varying missing not at random (MNAR) assumptions (selection models, pattern mixture models and shared parameter models) and to MAR methods that used inclusive models. The key sensitivity analysis used multiple imputation assuming that after dropout the trajectory of drug‐treated patients was that of placebo treated patients with a similar outcome history (placebo multiple imputation). This result was used as the worst reasonable case to define the lower limit of plausible values for the treatment contrast. The endpoint contrast from the primary analysis was ? 2.79 (p = .013). In placebo multiple imputation, the result was ? 2.17. Results from the other sensitivity analyses ranged from ? 2.21 to ? 3.87 and were symmetrically distributed around the primary result. Hence, no clear evidence of bias from missing not at random data was found. In the worst reasonable case scenario, the treatment effect was 80% of the magnitude of the primary result. Therefore, it was concluded that a treatment effect existed. The structured sensitivity framework of using a worst reasonable case result based on a controlled imputation approach with transparent and debatable assumptions supplemented a series of plausible alternative models under varying assumptions was useful in this specific situation and holds promise as a generally useful framework. Copyright © 2012 John Wiley & Sons, Ltd.     

On the recoding of continuous and bounded indexes to a binomial form: an application to quality-of-life scores

The statistical analysis of patient-reported outcomes (PROs) as endpoints has shown to be of great practical relevance. The resulting scores or indexes from the questionnaires used to measure PROs could be treated as continuous or ordinal. The goal of this study is to propose and evaluate a recoding process of the scores, so that they can be treated as binomial outcomes and, therefore, analyzed using logistic regression with random effects. The general methodology of recoding is based on the observable values of the scores. In order to obtain an optimal recoding, the evaluation of the recoding method is tested for different values of the parameters of the binomial distribution and different probability distributions of the random effects. We illustrate, evaluate and validate the proposed method of recoding with the Short Form-36 (SF-36) Survey and real data. The optimal recoding approach is very useful and flexible. Moreover, it has a natural interpretation, not only for ordinal scores, but also for questionnaires with many dimensions and different profiles, where a common method of analysis is desired, such as the SF-36.     

Non-negative estimation of variance components in heteroscedastic one-way random-effects ANOVA models

There is a considerable amount of literature dealing with inference about the parameters in a heteroscedastic one-way random-effects ANOVA model. In this paper, we primarily address the problem of improved quadratic estimation of the random-effect variance component. It turns out that such estimators with a smaller mean squared error compared with some standard unbiased quadratic estimators exist under quite general conditions. Improved estimators of the error variance components are also established.     

Design-based random permutation models with auxiliary information

We extend the random permutation model to obtain the best linear unbiased estimator of a finite population mean accounting for auxiliary variables under simple random sampling without replacement (SRS) or stratified SRS. The proposed method provides a systematic design-based justification for well-known results involving common estimators derived under minimal assumptions that do not require specification of a functional relationship between the response and the auxiliary variables.     

Mean estimation with data missing at random for functional covariables

In a missing-data setting, we want to estimate the mean of a scalar outcome, based on a sample in which an explanatory variable is observed for every subject while responses are missing by happenstance for some of them. We consider two kinds of estimates of the mean response when the explanatory variable is functional. One is based on the average of the predicted values and the second one is a functional adaptation of the Horvitz–Thompson estimator. We show that the infinite dimensionality of the problem does not affect the rates of convergence by stating that the estimates are root-n consistent, under missing at random (MAR) assumption. These asymptotic features are completed by simulated experiments illustrating the easiness of implementation and the good behaviour on finite sample sizes of the method. This is the first paper emphasizing that the insensitiveness of averaged estimates, well known in multivariate non-parametric statistics, remains true for an infinite-dimensional covariable. In this sense, this work opens the way for various other results of this kind in functional data analysis.     

Random translation of discrete order statistics

We investigate the existence and uniqueness of a discrete parent distribution supported on the integers whose order statistics are related by a random translation. We also provide some examples using the constructive method that we propose.     

A class of integral operators generated by random variables

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Complete convergence for arrays of rowwise negatively superadditive-dependent random variables and its applications

In this paper, the Rosenthal-type maximal inequalities and Kolmogorov-type exponential inequality for negatively superadditive-dependent (NSD) random variables are presented. By using these inequalities, we study the complete convergence for arrays of rowwise NSD random variables. As applications, the Baum–Katz-type result for arrays of rowwise NSD random variables and the complete consistency for the estimator of nonparametric regression model based on NSD errors are obtained. Our results extend and improve the corresponding ones of Chen et al. [On complete convergence for arrays of rowwise negatively associated random variables. Theory Probab Appl. 2007;52(2):393–397] for arrays of rowwise negatively associated random variables to the case of arrays of rowwise NSD random variables.