Confidence intervals for the difference of marginal probabilities in clustered matched-pair binary data

Although there are several available test statistics to assess the difference of marginal probabilities in clustered matched‐pair binary data, associated confidence intervals (CIs) are not readily available. Herein, the construction of corresponding CIs is proposed, and the performance of each CI is investigated. The results from Monte Carlo simulation study indicate that the proposed CIs perform well in maintaining the nominal coverage probability: for small to medium numbers of clusters, the intracluster correlation coefficient‐adjusted McNemar statistic and its associated Wald or Score CIs are preferred; however, this statistic becomes conservative when the number of clusters is larger so that alternative statistics and their associated CIs are preferred. In practice, a combination of the intracluster correlation coefficient‐adjusted McNemar statistic with an alternative statistic is recommended. To illustrate the practical application, a real clustered matched‐pair collection of data is used to illustrate testing the difference of marginal probabilities and constructing the associated CIs. Copyright © 2012 John Wiley & Sons, Ltd.     

Linear regression analysis with inequality constraints on the regression parameters via empirical likelihood

An empirical likelihood ratio test is developed for testing for or against inequality constraints on regression parameters in linear regression analysis. The proposed approach imposes no parametric model nor identically distributing assumption on the random errors. The asymptotic distribution of the proposed test statistic under null hypothesis is shown to be of chi-bar-squared type. The asymptotic power under contiguous alternatives is also briefly discussed. Moreover, an adjusted empirical likelihood method is adopted to improve the small sample size behaviour of the proposed test. Several simulation studies are carried out to assess the finite sample performance of the proposed tests. The results reveal that the proposed tests could be valuable for improving inference efficiency. A real-life example is discussed to illustrate the theoretical results.     

Analysis of binary data for the balanced one-way classification

A test is proposed for testing the equality of proportions based on the data available from a one-way classification having t treatment conditions and n binary observations per treatment. The test statistic B is a constant multiple of the F-statistic which results when the analysis of variance procedure for the one-way classification is applied to the data and, hence, is computationally simple. The statistic B from this binary analysis of variance (BIANOVA) is distributed asymptotically as a chi-square random variable. The proposed test is uniformly more powerful than either the F-test indicated above or the Pearson chi-square test; however, the attained empirical level of significance is frequently higher than for either of these competitors and usually higher than the stated level of significance for smaller values of n (say n ≤ 20).     

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.     

An Adjustment to the Bartlett's Test for Small Sample Size

The Bartlett's test (1937) for equality of variances is based on the χ² distribution approximation. This approximation deteriorates either when the sample size is small (particularly < 4) or when the population number is large. According to a simulation investigation, we find a similar varying trend for the mean differences between empirical distributions of Bartlett's statistics and their χ² approximations. By using the mean differences to represent the distribution departures, a simple adjustment approach on the Bartlett's statistic is proposed on the basis of equal mean principle. The performance before and after adjustment is extensively investigated under equal and unequal sample sizes, with number of populations varying from 3 to 100. Compared with the traditional Bartlett's statistic, the adjusted statistic is distributed more closely to χ² distribution, for homogeneity samples from normal populations. The type I error is well controlled and the power is a little higher after adjustment. In conclusion, the adjustment has good control on the type I error and higher power, and thus is recommended for small samples and large population number when underlying distribution is normal.     

A goodness-of-fit test for logistic-normal models using nonparametric smoothing method

Logistic-normal models can be applied for analysis of longitudinal binary data. The aim of this article is to propose a goodness-of-fit test using nonparametric smoothing techniques for checking the adequacy of logistic-normal models. Moreover, the leave-one-out cross-validation method for selecting the suitable bandwidth is developed. The quadratic form of the proposed test statistic based on smoothing residuals provides a global measure for checking the model with categorical and continuous covariates. The formulae of expectation and variance of the proposed statistics are derived, and their asymptotic distribution is approximated by a scaled chi-squared distribution. The power performance of the proposed test for detecting the interaction term or the squared term of continuous covariates is examined by simulation studies. A longitudinal dataset is utilized to illustrate the application of the proposed test.     

Goodness-of-fit Tests for GEE with Correlated Binary Data

The marginal logistic regression, in combination with GEE, is an increasingly important method in dealing with correlated binary data. As for independent binary data, when the number of possible combinations of the covariate values in a logistic regression model is much larger than the sample size, such as when the logistic model contains at least one continuous covariate, many existing chi-square goodness-of-fit tests either are not applicable or have some serious drawbacks. In this paper two residual based normal goodness-of-fit test statistics are proposed: the Pearson chi-square and an unweighted sum of residual squares. Easy-to-calculate approximations to the mean and variance of either statistic are also given. Their performance, in terms of both size and power, was satisfactory in our simulation studies. For illustration we apply them to a real data set.     

Empirical phi-divergence test statistics for testing simple and composite null hypotheses

The main purpose of this paper is to introduce first a new family of empirical test statistics for testing a simple null hypothesis when the vector of parameters of interest is defined through a specific set of unbiased estimating functions. This family of test statistics is based on a distance between two probability vectors, with the first probability vector obtained by maximizing the empirical likelihood (EL) on the vector of parameters, and the second vector defined from the fixed vector of parameters under the simple null hypothesis. The distance considered for this purpose is the phi-divergence measure. The asymptotic distribution is then derived for this family of test statistics. The proposed methodology is illustrated through the well-known data of Newcomb's measurements on the passage time for light. A simulation study is carried out to compare its performance with that of the EL ratio test when confidence intervals are constructed based on the respective statistics for small sample sizes. The results suggest that the ‘empirical modified likelihood ratio test statistic’ provides a competitive alternative to the EL ratio test statistic, and is also more robust than the EL ratio test statistic in the presence of contamination in the data. Finally, we propose empirical phi-divergence test statistics for testing a composite null hypothesis and present some asymptotic as well as simulation results for evaluating the performance of these test procedures.     

Empirical Likelihood Based Synthetic Data Method for Censored Regression Analysis

In this article, we propose a new empirical likelihood method for linear regression analysis with a right censored response variable. The method is based on the synthetic data approach for censored linear regression analysis. A log-empirical likelihood ratio test statistic for the entire regression coefficients vector is developed and we show that it converges to a standard chi-squared distribution. The proposed method can also be used to make inferences about linear combinations of the regression coefficients. Moreover, the proposed empirical likelihood ratio provides a way to combine different normal equations derived from various synthetic response variables. Maximizing this empirical likelihood ratio yields a maximum empirical likelihood estimator which is asymptotically equivalent to the solution of the estimating equation that are optimal linear combination of the original normal equations. It improves the estimation efficiency. The method is illustrated by some Monte Carlo simulation studies as well as a real example.     

An extensive power evaluation of a novel two-sample density-based empirical likelihood ratio test for paired data with an application to a treatment study of attention-deficit/hyperactivity disorder and severe mood dysregulation

In many case-control studies, it is common to utilize paired data when treatments are being evaluated. In this article, we propose and examine an efficient distribution-free test to compare two independent samples, where each is based on paired observations. We extend and modify the density-based empirical likelihood ratio test presented by Gurevich and Vexler [7] to formulate an appropriate parametric likelihood ratio test statistic corresponding to the hypothesis of our interest and then to approximate the test statistic nonparametrically. We conduct an extensive Monte Carlo study to evaluate the proposed test. The results of the performed simulation study demonstrate the robustness of the proposed test with respect to values of test parameters. Furthermore, an extensive power analysis via Monte Carlo simulations confirms that the proposed method outperforms the classical and general procedures in most cases related to a wide class of alternatives. An application to a real paired data study illustrates that the proposed test can be efficiently implemented in practice.     

Empirical Likelihood Method for General Additive-Multiplicative Hazard Models

An empirical likelihood-based inferential procedure is developed for a class of general additive-multiplicative hazard models. The proposed log-empirical likelihood ratio test statistic for the parameter vector is shown to have a chi-squared limiting distribution. The result can be used to make inference about the entire parameter vector as well as any linear combination of it. The asymptotic power of the proposed test statistic under contiguous alternatives is discussed. The method is illustrated by extensive simulation studies and a real example.     

部分函数型线性变系数模型的序列相关检验

部分函数型线性变系数模型(PFLVCM)是近几年出现的一个比较灵活、应用广泛的新模型。在实际应用中,搜集到的经济和金融数据往往存在序列相关性。如果不考虑数据间的相关性直接对其进行建模,会影响模型中参数估计的精度和有效性。本文主要研究了PFLVCM中误差的序列相关性的检验问题,基于经验似然,把标量时间序列数据相关性检验的方法拓展到函数型数据中,提出了经验对数似然比检验统计量,并在零假设下得到了检验统计量的近似分布。通过蒙特卡洛数值模拟说明该统计量在有限样本下有良好的水平和功效。最后,把该方法用于检验美国商业用电消费数据是否有序列相关性,证明该统计量的有效性和实用性。     

Power of the adjusted Q statistic to evaluate heterogeneity in meta-analyses of cluster randomized trials

Because of its simplicity, the Q statistic is frequently used to test the heterogeneity of the estimated intervention effect in meta-analyses of individually randomized trials. However, it is inappropriate to apply it directly to the meta-analyses of cluster randomized trials without taking clustering effects into account. We consider the properties of the adjusted Q statistic for testing heterogeneity in the meta-analyses of cluster randomized trials with binary outcomes. We also derive an analytic expression for the power of this statistic to detect heterogeneity in meta-analyses, which can be useful when planning a meta-analysis. A simulation study is used to assess the performance of the adjusted Q statistic, in terms of its Type I error rate and power. The simulation results are compared to that obtained from the proposed formula. It is found that the adjusted Q statistic has a Type I error rate close to the nominal level of 5%, as compared to the unadjusted Q statistic commonly used to test for heterogeneity in the meta-analyses of individually randomized trials with an inflated Type I error rate. Data from a meta-analysis of four cluster randomized trials are used to illustrate the procedures.     

Methods for testing subblock patterns

A number of statistical tests have been recommended over the last twenty years for assessing the randomness of long binary strings used in cryptographic algorithms. Several of these tests include methods of examining subblock patterns. These tests are the uniformity test, the universal test and the repetition test. The effectiveness of these tests are compared based on the subblock length, the limitations on data requirements, and on their power in detecting deviations from randomness. Due to the complexity of the test statistics, the power functions are estimated by simulation methods. The results show that for small subblocks the uniformity test is more powerful than the universal test, and that there is some doubt about the parameters of the hypothesised distribution for the universal test statistic. For larger subblocks the results show that the repetition test is the most effective test, since it requires far less data than either of the other two tests and is an efficient test in detecting deviations from randomness in binary strings.     

Empirical Likelihood Inference of the Partial Linear Isotonic Errors-in-variables Regression Models with Missing Data

This article is concerned with statistical inference of the partial linear isotonic regression model missing response and measurement errors in covariates. We proposed an empirical likelihood ratio test statistics and show that it has a limiting weighted chi-square distribution. An adjusted empirical likelihood ratio statistic, which is shown to have a limiting standard central chi-square distribution, is then proposed further. A maximum empirical likelihood estimator is also developed. A simulation study is conducted to examine the finite-sample property of proposed procedure.     

Empirical Likelihood-based Inference for Stationary-ergodicity of the Generalized Random Coefficient Autoregressive Model

In this article, we consider the application of the empirical likelihood method to the generalized random coefficient autoregressive (GRCA) model. When the order of the model is 1, we derive an empirical likelihood ratio test statistic to test the stationary-ergodicity. Some simulation studies are also conducted to investigate the finite sample performances of the proposed test.     

Resampling Methods for Testing a Semiparametric Random Censorship Model

This paper presents a goodness-of-fit test for a semiparametric random censorship model proposed by Dikta (1998 ). The test statistic is derived from a model-based process which is asymptotically Gaussian. In addition to test consistency, the proposed test can detect local alternatives distinct n ^-1/2 from the null hypothesis. Due to the intractability of the asymptotic null distribution of the test statistic, we turn to two resampling approximations. We first use the well-known bootstrap method to approximate critical values of the test. We then introduce a so-called random symmetrization method for carrying out the test. Both methods perform very well with a sample of moderate size. A simulation study shows that the latter possesses better empirical powers and sizes for small samples.     

Nonparametric Covariate-Adjusted Association Tests Based on the Generalized Kendall's Tau()

Identifying the risk factors for comorbidity is important in psychiatric research. Empirically, studies have shown that testing multiple, correlated traits simultaneously is more powerful than testing a single trait at a time in association analysis. Furthermore, for complex diseases, especially mental illnesses and behavioral disorders, the traits are often recorded in different scales such as dichotomous, ordinal and quantitative. In the absence of covariates, nonparametric association tests have been developed for multiple complex traits to study comorbidity. However, genetic studies generally contain measurements of some covariates that may affect the relationship between the risk factors of major interest (such as genes) and the outcomes. While it is relatively easy to adjust these covariates in a parametric model for quantitative traits, it is challenging for multiple complex traits with possibly different scales. In this article, we propose a nonparametric test for multiple complex traits that can adjust for covariate effects. The test aims to achieve an optimal scheme of adjustment by using a maximum statistic calculated from multiple adjusted test statistics. We derive the asymptotic null distribution of the maximum test statistic, and also propose a resampling approach, both of which can be used to assess the significance of our test. Simulations are conducted to compare the type I error and power of the nonparametric adjusted test to the unadjusted test and other existing adjusted tests. The empirical results suggest that our proposed test increases the power through adjustment for covariates when there exist environmental effects, and is more robust to model misspecifications than some existing parametric adjusted tests. We further demonstrate the advantage of our test by analyzing a data set on genetics of alcoholism.     

Nonparametric two-sample tests for dispersion differences based on placements

Two different two-sample tests for dispersion differences based on placement statistics are proposed. The means and variances of the test statistics are derived, and asymptotic normality is established for both. Variants of the proposed tests based on reversing the X and Y labels in the test statistic calculations are shown to have different small-sample properties; for both pairs of tests, one member of the pair will be resolving, the other nonresolving. The proposed tests are similar in spirit to the dispersion tests of both Mood and Hollander; comparative simulation results for these four tests are given. For small sample sizes, the powers of the proposed tests are approximately equal to the powers of the tests of both Mood and Hollander for samples from the normal, Cauchy and exponential distributions. The one-sample limiting distributions are also provided, yielding useful approximations to the exact tests when one sample is much larger than the other. A bootstrap test may alternatively be performed. The proposed test statistics may be used with lightly censored data by substituting Kaplan-Meier estimates for the empirical distribution functions.     

Testing a block exchangeable covariance matrix

Testing hypotheses about the structure of a covariance matrix for doubly multivariate data is often considered in the literature. In this paper the Rao's score test (RST) is derived to test the block exchangeable covariance matrix or block compound symmetry (BCS) covariance structure under the assumption of multivariate normality. It is shown that the empirical distribution of the RST statistic under the null hypothesis is independent of the true values of the mean and the matrix components of a BCS structure. A significant advantage of the RST is that it can be performed for small samples, even smaller than the dimension of the data, where the likelihood ratio test (LRT) cannot be used, and it outperforms the standard LRT in a number of contexts. Simulation studies are performed for the sample size consideration, and for the estimation of the empirical quantiles of the null distribution of the test statistic. The RST procedure is illustrated on a real data set from the medical studies.