Comparison of correlated proportions based on paired binary data from clustered samples

In this article, we explore hypothesis testing problems related to correlated proportions from clustered matched-pair binary data. Null hypotheses of equality in proportions, homogeneity, and non-inferiority of one to another are similar testing problems of linear contrasts of correlated proportions with suitable transformation. The covariance estimators of the test statistics are based on moment estimation under the null hypotheses. We present a general framework for testing linear contrasts of the correlated proportions from clustered matched-pair data based upon a class of unbiased estimators of the proportions. The corresponding testing procedures do not impose structure assumptions on the correlation matrix and are easy to use. Simulation results suggest that the proposed method is more likely to maintain the proper significance level and to improve power than the test proposed by Obuchowski.     

Mixtures of regression models with incomplete and noisy data

The estimation of the mixtures of regression models is usually based on the normal assumption of components and maximum likelihood estimation of the normal components is sensitive to noise, outliers, or high-leverage points. Missing values are inevitable in many situations and parameter estimates could be biased if the missing values are not handled properly. In this article, we propose the mixtures of regression models for contaminated incomplete heterogeneous data. The proposed models provide robust estimates of regression coefficients varying across latent subgroups even under the presence of missing values. The methodology is illustrated through simulation studies and a real data analysis.     

A multivariate zero-inflated binomial model for the analysis of correlated proportional data

In this paper, a new multivariate zero-inflated binomial (MZIB) distribution is proposed to analyse the correlated proportional data with excessive zeros. The distributional properties of purposed model are studied. The Fisher scoring algorithm and EM algorithm are given for the computation of estimates of parameters in the proposed MZIB model with/without covariates. The score tests and the likelihood ratio tests are derived for assessing both the zero-inflation and the equality of multiple binomial probabilities in correlated proportional data. A limited simulation study is performed to evaluate the performance of derived EM algorithms for the estimation of parameters in the model with/without covariates and to compare the nominal levels and powers of both score tests and likelihood ratio tests. The whitefly data is used to illustrate the proposed methodologies.     

Testing equality of two beta binomial proportions in the presence of unequal extra-dispersion parameters

Data in the form of proportions with extra-dispersion (over/under) arise in many biomedical, epidemiological, and toxicological applications. In some situations, two samples of data in the form of proportions with extra-dispersion arise in which the problem is to test the equality of the proportions in the two groups with unspecified and possibly unequal extra-dispersion parameters. This problem is analogous to the traditional Behrens-Fisher problem in which two normal population means with possibly unequal variances are compared. To deal with this problem we develop eight tests and compare them in terms of empirical size and power, using a simulation study. Simulations show that a C(α) test based on extended quasi-likelihood estimates of the nuisance parameters holds nominal level most effectively (close to the nominal level) and it is at least as powerful as any other statistic that is not liberal. It has the simplest formula, is based on estimates of the nuisance parameters only under the null hypothesis, and is easiest to calculate. Also, it is robust in the sense that no distributional assumption is required to develop this statistic.     

Testing equality of mean vectors in a one-way MANOVA with monotone missing data

In this study, testing the equality of mean vectors in a one-way multivariate analysis of variance (MANOVA) is considered when each dataset has a monotone pattern of missing observations. The likelihood ratio test (LRT) statistic in a one-way MANOVA with monotone missing data is given. Furthermore, the modified test (MT) statistic based on likelihood ratio (LR) and the modified LRT (MLRT) statistic with monotone missing data are proposed using the decomposition of the LR and an asymptotic expansion for each decomposed LR. The accuracy of the approximation for the Chi-square distribution is investigated using a Monte Carlo simulation. Finally, an example is given to illustrate the methods.     

Progressive multi-state models for informatively incomplete longitudinal data

Progressive multi-state models provide a convenient framework for characterizing chronic disease processes where the states represent the degree of damage resulting from the disease. Incomplete data often arise in studies of such processes, and standard methods of analysis can lead to biased parameter estimates when observation of data is response-dependent. This paper describes a joint analysis useful for fitting progressive multi-state models to data arising in longitudinal studies in such settings. Likelihood based methods are described and parameters are shown to be identifiable. An EM algorithm is described for parameter estimation, and variance estimation is carried out using the Louis’ method. Simulation studies demonstrate that the proposed method works well in practice under a variety of settings. An application to data from a smoking prevention study illustrates the utility of the method.     

Analysis of proportions for clustered binary data in the presence of unequal dispersions

This paper investigates the test procedures for testing the homogeneity of the proportions in the analysis of clustered binary data in the context of unequal dispersions across the treatment groups. We introduce a simple test procedure based on adjusted proportions using a sandwich estimator of the variance of the proportion estimators obtained by the generalized estimating equations approach of Zeger and Liang (1986) [Biometrics 42, 121-130]. We also extend the exiting test procedures of testing the hypothesis of proportions in this context. These test procedures are then compared, by simulations, in terms of size and power. Moreover, we derive the score test for testing the homogeneity of the dispersion parameters among several groups of clustered binary data. An illustrative application of the recommended test procedures is also presented.     

Testing homogeneity of difference of two proportions for stratified correlated paired binary data

In ophthalmologic or otolaryngologic study, each subject may contribute paired organs measurements to the analysis. A number of statistical methods have been proposed on bilateral correlated data. In practice, it is important to detect confounding effect by treatment interaction, since ignoring confounding effect may lead to unreliable conclusion. Therefore, stratified data analysis can be considered to adjust the effect of confounder on statistical inference. In this article, we investigate and derive three test procedures for testing homogeneity of difference of two proportions for stratified correlated paired binary data in the basis of equal correlation model assumption. The performance of proposed test procedures is examined through Monte Carlo simulation. The simulation results show that the Score test is usually robust on type I error control with high power, and therefore is recommended among the three methods. One example from otolaryngologic study is given to illustrate the three test procedures.     

A stochastic approximation algorithm for maximum-likelihood estimation with incomplete data

We propose a new stochastic approximation (SA) algorithm for maximum-likelihood estimation (MLE) in the incomplete-data setting. This algorithm is most useful for problems when the EM algorithm is not possible due to an intractable E-step or M-step. Compared to other algorithm that have been proposed for intractable EM problems, such as the MCEM algorithm of Wei and Tanner (1990), our proposed algorithm appears more generally applicable and efficient. The approach we adopt is inspired by the Robbins-Monro (1951) stochastic approximation procedure, and we show that the proposed algorithm can be used to solve some of the long-standing problems in computing an MLE with incomplete data. We prove that in general O(n) simulation steps are required in computing the MLE with the SA algorithm and O(n log n) simulation steps are required in computing the MLE using the MCEM and/or the MCNR algorithm, where n is the sample size of the observations. Examples include computing the MLE in the nonlinear error-in-variable model and nonlinear regression model with random effects.     

Testing the equality means of several log-normal distributions

Researches propose various methods for comparing the means of two log-normal distributions. Some of these methods have been recently extended to test the equality means of several log-normal populations. Investigations show that none of the established methods is satisfactory. In this article, we provide three methods based on the computational approach test, which is a parametric bootstrap approach, for testing the means of several log-normal distributions. Further, we compare our methods with the existing methods through Monte Carlo simulation. The numerical results show that the Type I errors of these procedures are satisfactory regardless of the sample size, number of populations, and the true parameters. Finally, we explain the considered methods by real examples.     

A unified sequential test procedure for simultaneous testing the equality of several binomial proportions to a specified standard

In this article, we propose a unified sequentially rejective test procedure for testing simultaneously the equality of several independent binomial proportions to a specified standard. The proposed test procedure is general enough to include some well-known multiple testing procedures such as the Ordinary Bonferroni procedure, Hochberg procedure and Rom procedure. It involves multiple tests of significance based on the simple binomial tests (exact or approximate) which can be easily found in many elementary standard statistics textbooks. Unlike the traditional Chi-square test of the overall hypothesis, the procedure can identify the subset of the binomial proportions, which are different from the prespecified standard with the control of the familywise type I error rate. Moreover, the power computation of the procedure is provided and the procedure is illustrated by two real examples from an ecological study and a carcinogenicity study.     

Testing equality of two normal covariance matrices with monotone missing data

Abstract

The problem of testing equality of two multivariate normal covariance matrices is considered. Assuming that the incomplete data are of monotone pattern, a quantity similar to the Likelihood Ratio Test Statistic is proposed. A satisfactory approximation to the distribution of the quantity is derived. Hypothesis testing based on the approximate distribution is outlined. The merits of the test are investigated using Monte Carlo simulation. Monte Carlo studies indicate that the test is very satisfactory even for moderately small samples. The proposed methods are illustrated using an example.     

Testing equality of correlation coefficients for paired binary data from multiple groups

Paired binary data arise naturally when paired body parts are investigated in clinical trials. One of the widely used models for dealing with this kind of data is the equal correlation coefficients model. Before using this model, it is necessary to test whether the correlation coefficients in each group are actually equal. In this paper, three test statistics (likelihood ratio test, Wald-type test, and Score test) are derived for this purpose. The simulation results show that the Score test statistic maintains type I error rate and has satisfactory power, and therefore is recommended among the three methods. The likelihood ratio test is over conservative in most cases, and the Wald-type statistic is not robust with respect to empirical type I error. Three real examples, including a multi-centre Phase II double-blind placebo randomized controlled trial, are given to illustrate the three proposed test statistics.     

Tests for homogeneity of risk differences in stratified design with correlated bilateral data

ABSTRACT

Correlated bilateral data arise from stratified studies involving paired body organs in a subject. When it is desirable to conduct inference on the scale of risk difference, one needs first to assess the assumption of homogeneity in risk differences across strata. For testing homogeneity of risk differences, we herein propose eight methods derived respectively from weighted-least-squares (WLS), the Mantel-Haenszel (MH) estimator, the WLS method in combination with inverse hyperbolic tangent transformation, and the test statistics based on their log-transformation, the modified Score test statistic and Likelihood ratio test statistic. Simulation results showed that four of the tests perform well in general, with the tests based on the WLS method and inverse hyperbolic tangent transformation always performing satisfactorily even under small sample size designs. The methods are illustrated with a dataset.     

Causal inference for recurrent events data with all-or-none compliance

ABSTRACT

In this article, causal inference in randomized studies with recurrent events data and all-or-none compliance is considered. We use the counting process to analyze the recurrent events data and propose a causal proportional intensity model. The maximum likelihood approach is adopted to estimate the parameters of the proposed causal model. To overcome the computational difficulties created by the mixture structure of the problem, we develop an expectation-maximization (EM) algorithm. The resulting estimators are shown to be consistent and asymptotically normal. We further estimate the complier average causal effect (CACE), which is defined as the difference of the average numbers of recurrence between treatment and control groups within the complier class. The corresponding inferential procedures are established. Some simulation studies are conducted to assess the finite sample performance of the proposed approach.     

One-way analysis of proportions for misclassified binomial data

We consider data with a nominal grouping variable and a binary response variable. The grouping variable is measured without error, but the response variable is measured using a fallible device subject to misclassification. To achieve model identifiability, we use the double-sampling scheme which requires obtaining a subsample of the original data or another independent sample. This sample is then classified by both the fallible device and another infallible device regarding the response variable. We propose two Wald tests for testing the association between the two variables and illustrate the test using traffic data. The Type-I error rate and power of the tests are examined using simulations and a modified Wald test is recommended.     

A profile likelihood method for normal mixture with unequal variance

It is well known that the normal mixture with unequal variance has unbounded likelihood and thus the corresponding global maximum likelihood estimator (MLE) is undefined. One of the commonly used solutions is to put a constraint on the parameter space so that the likelihood is bounded and then one can run the EM algorithm on this constrained parameter space to find the constrained global MLE. However, choosing the constraint parameter is a difficult issue and in many cases different choices may give different constrained global MLE. In this article, we propose a profile log likelihood method and a graphical way to find the maximum interior mode. Based on our proposed method, we can also see how the constraint parameter, used in the constrained EM algorithm, affects the constrained global MLE. Using two simulation examples and a real data application, we demonstrate the success of our new method in solving the unboundness of the mixture likelihood and locating the maximum interior mode.     

Missing covariates in generalized linear models when the missing data mechanism is non-ignorable

We propose a method for estimating parameters in generalized linear models with missing covariates and a non-ignorable missing data mechanism. We use a multinomial model for the missing data indicators and propose a joint distribution for them which can be written as a sequence of one-dimensional conditional distributions, with each one-dimensional conditional distribution consisting of a logistic regression. We allow the covariates to be either categorical or continuous. The joint covariate distribution is also modelled via a sequence of one-dimensional conditional distributions, and the response variable is assumed to be completely observed. We derive the E- and M-steps of the EM algorithm with non-ignorable missing covariate data. For categorical covariates, we derive a closed form expression for the E- and M-steps of the EM algorithm for obtaining the maximum likelihood estimates (MLEs). For continuous covariates, we use a Monte Carlo version of the EM algorithm to obtain the MLEs via the Gibbs sampler. Computational techniques for Gibbs sampling are proposed and implemented. The parametric form of the assumed missing data mechanism itself is not `testable' from the data, and thus the non-ignorable modelling considered here can be viewed as a sensitivity analysis concerning a more complicated model. Therefore, although a model may have `passed' the tests for a certain missing data mechanism, this does not mean that we have captured, even approximately, the correct missing data mechanism. Hence, model checking for the missing data mechanism and sensitivity analyses play an important role in this problem and are discussed in detail. Several simulations are given to demonstrate the methodology. In addition, a real data set from a melanoma cancer clinical trial is presented to illustrate the methods proposed.     

An EM and a Stochastic Version of the EM Algorithm for Nonparametric Hidden Semi-Markov Models

The Hidden semi-Markov models (HSMMs) were introduced to overcome the constraint of a geometric sojourn time distribution for the different hidden states in the classical hidden Markov models. Several variations of HSMMs were proposed that model the sojourn times by a parametric or a nonparametric family of distributions. In this article, we concentrate our interest on the nonparametric case where the duration distributions are attached to transitions and not to states as in most of the published papers in HSMMs. Therefore, it is worth noticing that here we treat the underlying hidden semi-Markov chain in its general probabilistic structure. In that case, Barbu and Limnios (2008 Barbu , V. , Limnios , N. ( 2008 ). Semi-Markov Chains and Hidden Semi-Markov Models Toward Applications: Their Use in Reliability and DNA Analysis . New York : Springer . [Google Scholar]) proposed an Expectation–Maximization (EM) algorithm in order to estimate the semi-Markov kernel and the emission probabilities that characterize the dynamics of the model. In this article, we consider an improved version of Barbu and Limnios' EM algorithm which is faster than the original one. Moreover, we propose a stochastic version of the EM algorithm that achieves comparable estimates with the EM algorithm in less execution time. Some numerical examples are provided which illustrate the efficient performance of the proposed algorithms.     

Semiparametric estimation of the cumulative incidence function under competing risks and left-truncated sampling

In this article, we propose semiparametric methods to estimate the cumulative incidence function of two dependent competing risks for left-truncated and right-censored data. The proposed method is based on work by Huang and Wang (1995). We extend previous model by allowing for a general parametric truncation distribution and a third competing risk before recruitment. Based on work by Vardi (1989), several iterative algorithms are proposed to obtain the semiparametric estimates of cumulative incidence functions. The asymptotic properties of the semiparametric estimators are derived. Simulation results show that a semiparametric approach assuming the parametric truncation distribution is correctly specified produces estimates with smaller mean squared error than those obtained in a fully nonparametric model.