Non-parametric tests for distributional treatment effect for randomly censored responses

Summary.  For a binary treatment ν =0, 1 and the corresponding 'potential response' Y ⁰ for the control group ( ν =0) and Y ¹ for the treatment group ( ν =1), one definition of no treatment effect is that Y ⁰ and Y ¹ follow the same distribution given a covariate vector X . Koul and Schick have provided a non-parametric test for no distributional effect when the realized response (1− ν ) Y ⁰+ ν Y ¹ is fully observed and the distribution of X is the same across the two groups. This test is thus not applicable to censored responses, nor to non-experimental (i.e. observational) studies that entail different distributions of X across the two groups. We propose ' X -matched' non-parametric tests generalizing the test of Koul and Schick following an idea of Gehan. Our tests are applicable to non-experimental data with randomly censored responses. In addition to these motivations, the tests have several advantages. First, they have the intuitive appeal of comparing all available pairs across the treatment and control groups, instead of selecting a number of matched controls (or treated) in the usual pair or multiple matching. Second, whereas most matching estimators or tests have a non-overlapping support (of X ) problem across the two groups, our tests have a built-in protection against the problem. Third, Gehan's idea allows the tests to make good use of censored observations. A simulation study is conducted, and an empirical illustration for a job training effect on the duration of unemployment is provided.     

On the Empirical Likelihood Ratio for Smooth Functions of M-functionals

It is known that the empirical likelihood ratio can be used to construct confidence regions for smooth functions of the mean, Fréchet differentiable statistical functionals and for a class of M-functionals. In this paper, we argue that this use can be extended to the class of functionals which are smooth functions of M-functionals. In particular, we find the conditions under which the empirical log-likelihood ratio for this kind of functionals admits a χ² approxima tion. Furthermore, we investigate, by simulation methods, the related approximation error in some contexts of practical interest.     

On Statistical Models for d-Dimensional Stable Processes, and Some Generalizations

In statistical models where jumps of a d -dimensional stable process ( S _t ) _{t ≥0} are observed in windows with certain asymptotic properties, and where parameters appearing in the Levy measure of S are to be estimated, we have asymptotically efficient estimators. If Poisson random measure μ on (0, ∞) × ( R ^d \{0}) with intensity dt Λ( dx ) replaces the jump measure of S , where Λ is a ε-finite measure on R ^d \{0} admitting tail parameters in a suitable sense, we specify a notion of neighbourhood which allows to treat efficiency in statistical experiments of the second type by switching to accompanying sequences of the stable process type considered first.     

Some Bounds on the Distribution of Certain Quadratic Forms in Normal Random Variables

The paper derives bounds on the distribution of the quadratic forms Z = y ^H( X Γ X ^H)⁻¹ y and W = y ^H(σ² I + X Γ X ^H)⁻¹ y , where the elements of the M × 1 vector y and the M × N matrix X are independent identically distributed (i.i.d.) complex zero mean Normal variables, Γ is some N × N diagonal matrix with positive diagonal elements, I , is the identity, σ² is a constant and ^H denotes the Hermitian transpose. The bounds are convenient for numerical work and appear to be tight for small values of M . This work has applications in digital mobile radio for a specific channel where M antennas are used to receive a signal with N interferers. Some of these applications in radio communication systems are discussed.     

Computation of the Generalized F Distribution

Exact expressions for the cumulative distribution function of a random variable of the form ( α ₁ X ₁+ α ₂ X ₂)/ Y are given where X ₁, X ₂ and Y are independent chi-squared random variables. The expressions are applied to the detection of joint outliers and Hotelling's mis-specified T ² distribution.     

Testing a Regression Model When We Have Smooth Alternatives in Mind

Goodness-of-fit tests based on residual sums of squares are standard procedures used when fitting regression models. Often we have a smooth alternative in mind, a qualitative feature that the χ²-test does not take into account. We show that the power of detecting a smooth alternative increases when we smooth the current model as well. The proposed test is shown to be able to detect any continuous local alternative tending to zero slower than n ^−1/2. Theoretical results also address minimax non-parametric hypothesis testing in Sobolev spaces. A simulation study is presented, and the procedure is applied to expenditure curve estimation.     

Likelihood Ratio Tests Under Local and Fixed Alternatives in Monotone Function Problems

Abstract.  We focus on a class of non-standard problems involving non-parametric estimation of a monotone function that is characterized by n ^1/3 rate of convergence of the maximum likelihood estimator, non-Gaussian limit distributions and the non-existence of     -regular estimators. We have shown elsewhere that under a null hypothesis of the type ψ ( z ₀) =  θ ₀ ( ψ being the monotone function of interest) in non-standard problems of the above kind, the likelihood ratio statistic has a 'universal' limit distribution that is free of the underlying parameters in the model. In this paper, we illustrate its limiting behaviour under local alternatives of the form ψ _n ( z ), where ψ _n (·) and ψ (·) vary in O ( n ^−1/3) neighbourhoods around z ₀ and ψ _n converges to ψ at rate n ^1/3 in an appropriate metric. Apart from local alternatives, we also consider the behaviour of the likelihood ratio statistic under fixed alternatives and establish the convergence in probability of an appropriately scaled version of the same to a constant involving a Kullback–Leibler distance.     

Likelihood Ratio Tests for Fixed Model Terms using Residual Maximum Likelihood

Likelihood ratio tests for fixed model terms are proposed for the analysis of linear mixed models when using residual maximum likelihood estimation. Bartlett-type adjustments, using an approximate decomposition of the data, are developed for the test statistics. A simulation study is used to compare properties of the test statistics proposed, with or without adjustment, with a Wald test. A proposed test statistic constructed by dropping fixed terms from the full fixed model is shown to give a better approximation to the asymptotic χ²-distribution than the Wald test for small data sets. Bartlett adjustment is shown to improve the χ²-approximation for the proposed tests substantially.     

On distribution-weighted partial least squares with diverging number of highly correlated predictors

Summary.  Because highly correlated data arise from many scientific fields, we investigate parameter estimation in a semiparametric regression model with diverging number of predictors that are highly correlated. For this, we first develop a distribution-weighted least squares estimator that can recover directions in the central subspace, then use the distribution-weighted least squares estimator as a seed vector and project it onto a Krylov space by partial least squares to avoid computing the inverse of the covariance of predictors. Thus, distrbution-weighted partial least squares can handle the cases with high dimensional and highly correlated predictors. Furthermore, we also suggest an iterative algorithm for obtaining a better initial value before implementing partial least squares. For theoretical investigation, we obtain strong consistency and asymptotic normality when the dimension p of predictors is of convergence rate O { n ^1/2/ log ( n )} and o ( n ^1/3) respectively where n is the sample size. When there are no other constraints on the covariance of predictors, the rates n ^1/2 and n ^1/3 are optimal. We also propose a Bayesian information criterion type of criterion to estimate the dimension of the Krylov space in the partial least squares procedure. Illustrative examples with a real data set and comprehensive simulations demonstrate that the method is robust to non-ellipticity and works well even in 'small n –large p ' problems.     

Likelihood ratio tests in linear mixed models with one variance component

Summary.  We consider the problem of testing null hypotheses that include restrictions on the variance component in a linear mixed model with one variance component and we derive the finite sample and asymptotic distribution of the likelihood ratio test and the restricted likelihood ratio test. The spectral representations of the likelihood ratio test and the restricted likelihood ratio test statistics are used as the basis of efficient simulation algorithms of their null distributions. The large sample χ ² mixture approximations using the usual asymptotic theory for a null hypothesis on the boundary of the parameter space have been shown to be poor in simulation studies. Our asymptotic calculations explain these empirical results. The theory of Self and Liang applies only to linear mixed models for which the data vector can be partitioned into a large number of independent and identically distributed subvectors. One-way analysis of variance and penalized splines models illustrate the results.     

MINIMAX ESTIMATION OF A MULTIVARIATE NORMAL MEAN UNDER A CONVEX LOSS FUNCTION

Let X = (X₁, - X_p)prime; ˜ N_p (μ, Σ) where μ= (μ₁, -, μ_p)' and Σ= diag (Σ²₁, -, Σ²_p) are both unknown and p3. Let (n_i - 2) w_i/Σ²_i! X²_ni, independent. of w_i (I ≠ j = 1, -, p). Assume that (w₁, -, w_p) and X are independent. Define W = diag (w₁, -, w_p) and ¶ X ¶²_w= X'W^-1Q^-1W^-1X where Q = diag (q₁, -,n q_p), q_i > 0, i = 1, -, p. In this paper, the minimax estimator of Berger & Bock (1976), given by δ (X, W) = [I_p - r(X, W) ¶ X ¶^-2_w Q^-1W^-1] X, is shown to be minimax relative to the convex loss (δ - μ)'[αQ + (1 - α) Σ^-1] δ - μ)/C, where C =α tr (Σ) + (1 - α)p and 0 α 1, under certain conditions on r(X, W). This generalizes the above mentioned result of Berger & Bock.     

On heteroscedastic hazards regression models: theory and application

A class of non-proportional hazards regression models is considered to have hazard specifications consisting of a power form of cross-effects on the base-line hazard function. The primary goal of these models is to deal with settings in which heterogeneous distribution shapes of survival times may be present in populations characterized by some observable covariates. Although effects of such heterogeneity can be explicitly seen through crossing cumulative hazards phenomena in k -sample problems, they are barely visible in a one-sample regression setting. Hence, heterogeneity of this kind may not be noticed and, more importantly, may result in severely misleading inference. This is because the partial likelihood approach cannot eliminate the unknown cumulative base-line hazard functions in this setting. For coherent statistical inferences, a system of martingale processes is taken as a basis with which, together with the method of sieves, an overidentified estimating equation approach is proposed. A Pearson's χ² type of goodness-of-fit testing statistic is derived as a by-product. An example with data on gastric cancer patients' survival times is analysed.     

Estimating smooth monotone functions

Many situations call for a smooth strictly monotone function f of arbitrary flexibility. The family of functions defined by the differential equation D  ² f  = w Df , where w is an unconstrained coefficient function comprises the strictly monotone twice differentiable functions. The solution to this equation is f = C ₀ + C ₁  D ⁻¹{exp( D ⁻¹ w )}, where C ₀ and C ₁ are arbitrary constants and D ⁻¹ is the partial integration operator. A basis for expanding w is suggested that permits explicit integration in the expression of f . In fitting data, it is also useful to regularize f by penalizing the integral of w ² since this is a measure of the relative curvature in f . Applications are discussed to monotone nonparametric regression, to the transformation of the dependent variable in non-linear regression and to density estimation.     

Parametric models for response-biased sampling

Suppose that subjects in a population follow the model f   ( y ^* x ^*; ) where y ^* denotes a response, x ^* denotes a vector of covariates and is the parameter to be estimated. We consider response-biased sampling, in which a subject is observed with a probability which is a function of its response. Such response-biased sampling frequently occurs in econometrics, epidemiology and survey sampling. The semiparametric maximum likelihood estimate of is derived, along with its asymptotic normality, efficiency and variance estimates. The estimate proposed can be used as a maximum partial likelihood estimate in stratified response-selective sampling. Some computation algorithms are also provided.     

A general method of constructing E(s2)-optimal supersaturated designs

There has been much recent interest in supersaturated designs and their application in factor screening experiments. Supersaturated designs have mainly been constructed by using the E ( s ²)-optimality criterion originally proposed by Booth and Cox in 1962. However, until now E ( s ²)-optimal designs have only been established with certainty for n experimental runs when the number of factors m is a multiple of n-1 , and in adjacent cases where m = q ( n -1) + r (| r | 2, q an integer). A method of constructing E ( s ²)-optimal designs is presented which allows a reasonably complete solution to be found for various numbers of runs n including n ,=8 12, 16, 20, 24, 32, 40, 48, 64.     

Effectiveness of potent antiretroviral therapy on progression of human immunodeficiency virus: Bayesian modelling and model checking via counterfactual replicates

Summary.  We analyse data from a seroincident cohort of 457 homosexual men who were infected with the human immunodeficiency virus, followed within the multicentre Italian Seroconversion Study. These data include onset times to acquired immune deficiency syndrome (AIDS), longitudinal measurements of CD4⁺ T-cell counts taken on each subject during the AIDS-free period of observation and the period of administration of a highly active antiretro- viral therapy (HAART), for the subset of individuals who received it. The aim of the study is to assess the effect of HAART on the course of the disease. We analyse the data by a Bayesian model in which the sequence of longitudinal CD4⁺ cell count observations and the associated time to AIDS are jointly modelled at an individual subject's level as depending on the treatment. We discuss the inferences obtained about the efficacy of HAART, as well as modelling and computation difficulties that were encountered in the analysis. These latter motivate a model criticism stage of the analysis, in which the model specification of CD4⁺ cell count progression and of the effect of treatment are checked. Our approach to model criticism is based on the notion of a counterfactual replicate data set Z ^c . This is a data set with the same shape and size as the observed data, which we might have observed by rerunning the study in exactly the same conditions as the actual study if the treated patients had not been treated at all. We draw samples of Z ^c from a null model M ₀, which assumes absence of treatment effect, conditioning on data collected in each subject before initiation of treatment. Model checking is performed by comparing the observed data with a set of samples of Z ^c drawn from M ₀.     

Calculating power for the comparison of dependent κ-coefficients

Summary. In the psychosocial and medical sciences, some studies are designed to assess the agreement between different raters and/or different instruments. Often the same sample will be used to compare the agreement between two or more assessment methods for simplicity and to take advantage of the positive correlation of the ratings. Although sample size calculations have become an important element in the design of research projects, such methods for agreement studies are scarce. We adapt the generalized estimating equations approach for modelling dependent κ -statistics to estimate the sample size that is required for dependent agreement studies. We calculate the power based on a Wald test for the equality of two dependent κ -statistics. The Wald test statistic has a non-central χ ²-distribution with non-centrality parameter that can be estimated with minimal assumptions. The method proposed is useful for agreement studies with two raters and two instruments, and is easily extendable to multiple raters and multiple instruments. Furthermore, the method proposed allows for rater bias. Power calculations for binary ratings under various scenarios are presented. Analyses of two biomedical studies are used for illustration.     

Empirical Likelihood-based Inference in Linear Models with Missing Data

The missing response problem in linear regression is studied. An adjusted empirical likelihood approach to inference on the mean of the response variable is developed. A non-parametric version of Wilks's theorem for the adjusted empirical likelihood is proved, and the corresponding empirical likelihood confidence interval for the mean is constructed. With auxiliary information, an empirical likelihood-based estimator with asymptotic normality is defined and an adjusted empirical log-likelihood function with asymptotic χ² is derived. A simulation study is conducted to compare the adjusted empirical likelihood methods and the normal approximation methods in terms of coverage accuracies and average lengths of the confidence intervals. Based on biases and standard errors, a comparison is also made between the empirical likelihood-based estimator and related estimators by simulation. Our simulation indicates that the adjusted empirical likelihood methods perform competitively and the use of auxiliary information provides improved inferences.     

The NPMLE for Doubly Censored Current Status Data

In biostatistical applications interest often focuses on the estimation of the distribution of time T between two consecutive events. If the initial event time is observed and the subsequent event time is only known to be larger or smaller than an observed point in time, then the data is described by the well understood singly censored current status model, also known as interval censored data, case I. Jewell et al. (1994) extended this current status model by allowing the initial time to be unobserved, but with its distribution over an observed interval ' A, B ' known to be uniformly distributed; the data is referred to as doubly censored current status data. These authors used this model to handle application in AIDS partner studies focusing on the NPMLE of the distribution G of T . The model is a submodel of the current status model, but the distribution G is essentially the derivative of the distribution of interest F in the current status model. In this paper we establish that the NPMLE of G is uniformly consistent and that the resulting estimators for the n ^1/2-estimable parameters are efficient. We propose an iterative weighted pool-adjacent-violator-algorithm to compute the estimator. It is also shown that, without smoothness assumptions, the NPMLE of F converges at rate n ^−2/5 in L ²-norm while the NPMLE of F in the non-parametric current status data model converges at rate n ^−1/3 in L ²-norm, which shows that there is a substantial gain in using the submodel information.     

ON THE CONVERGENCE OF EMPIRICAL PROCESSES OF MIXING VARIABLES1

Conditions are given for the weak convergence of (t—t²)L_N(a_N^-1( t )) to a Gaussian process where v<1/2, a _N is a cdf and L _N is the normalized weighted empirical cumulative distribution function (cdf) for an α-mixing sample of random variables in R which may be non-stationary with discontinuous marginals.