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Bayesian adaptive designs for clinical trials

临床试验的贝叶斯自适应设计

A Bayesian adaptive design is proposed for a comparative two-armed clinical trial using decision-theoretic approaches. A loss function is specified, based on the cost for each patient and the costs of making incorrect decisions at the end of a trial. At each interim analysis, the decision to terminate or to continue the trial is based on the expected loss function while concurrently incorporating efficacy, futility and cost. The maximum number of interim analyses is…

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Semiparametric inference in observational duration-response studies, with duration possibly right-censored

治疗时长可能右删失的观察性时长—响应研究中的半参数推断

Once treatment is found to be effective in clinical studies, attention often focuses on optimum or efficacious treatment delivery. In treatment duration-response studies, the optimum treatment delivery refers to the treatment length that optimises the mean response. In many studies, the treatment length is often left to the discretion of an attending investigator or physician but may be abruptly terminated because of treatment-terminating events. Thus, a recommended…

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Locally-efficient robust estimation of haplotype-disease association in family-based studies

家系研究中单倍型与疾病关联的局部有效稳健估计

Modelling human genetic variation is critical to understanding the genetic basis of complex disease. The Human Genome Project has discovered millions of binary DNA sequence variants, called single nucleotide polymorphisms, and millions more may exist. As coding for proteins takes place along chromosomes, organisation of polymorphisms along each chromosome, the haplotype phase structure, may prove to be most important in discovering genetic variants associated with disease.…

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Smooth quantile ratio estimation

平滑分位数比估计

We propose a novel approach to estimating the mean difference between two highly skewed distributions. The method, which we call smooth quantile ratio estimation, smooths, over percentiles, the ratio of the quantiles of the two distributions. The method defines a large class of estimators, including the sample mean difference, the maximum likelihood estimator under log-normal samples and the L-estimator. We derive asymptotic properties such as consistency and asymptotic…

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Frequentist prediction intervals and predictive distributions

频率学派预测区间与预测分布

We consider parametric frameworks for the prediction of future values of a random variable Y, based on previously observed data X. Simple pivotal methods for obtaining calibrated prediction intervals are presented and illustrated. Frequentist predictive distributions are defined as confidence distributions, and their utility is demonstrated. A simple pivotal-based approach that produces prediction intervals and predictive distributions with well-calibrated frequentist…

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Likelihood ratio tests in curved exponential families with nuisance parameters present only under the alternative

存在仅在备择下可识别的干扰参数时,曲指数族中的似然比检验

For submodels of an exponential family, we consider likelihood ratio tests for hypotheses that render some parameters nonidentifiable. First, we establish the asymptotic equivalence between the likelihood ratio test and the score test. Secondly, the score-test representation is used to derive the asymptotic distribution of the likelihood ratio test. These results are derived for general submodels of an exponential family without assuming compactness of the parameter space.…

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Diagnostic checking for time series models with conditional heteroscedasticity estimated by the least absolute deviation approach

最小绝对偏差估计的条件异方差时间序列模型诊断

The recent paper by Peng & Yao (2003) gave an interesting extension of least absolute deviation estimation to generalised autoregressive conditional heteroscedasticity, GARCH, time series models. The asymptotic distributions of absolute residual autocorrelations and squared residual autocorrelations from the GARCH model estimated by the least absolute deviation method are derived in this paper. These results lead to two useful diagnostic tools which can be used to check…

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Nonparametric inference in multivariate mixtures

多元混合模型中的非参数推断

We consider mixture models in which the components of data vectors from any given subpopulation are statistically independent, or independent in blocks. We argue that if, under this condition of independence, we take a nonparametric view of the problem and allow the number of subpopulations to be quite general, the distributions and mixing proportions can often be estimated root-n consistently. Indeed, we show that, if the data are k-variate and there are p subpopulations,…

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Semiparametric Box–Cox power transformation models for censored survival observations

删失生存观测的半参数 Box–Cox 幂变换模型

The accelerated failure time model specifies that the logarithm of the failure time is linearly related to the covariate vector without assuming a parametric error distribution. In this paper, we consider the semiparametric Box–Cox transformation model, which includes the above regression model as a special case, to analyse possibly censored failure time observations. Inference procedures for the transformation and regression parameters are proposed via a resampling…

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A semiparametric regression cure model with current status data

This paper considers the analysis of current status data with a cured proportion in the population using a mixture model that combines a logistic regression formulation for the probability of cure with a semiparametric regression model for the time to occurrence of the event. The semiparametric regression model belongs to the flexible class of partly linear models that allows one to explore the possibly nonlinear effect of a certain covariate on the response variable. A…

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A note on composite likelihood inference and model selection

复合似然推断与模型选择札记

A composite likelihood consists of a combination of valid likelihood objects, usually related to small subsets of data. The merit of composite likelihood is to reduce the computational complexity so that it is possible to deal with large datasets and very complex models, even when the use of standard likelihood or Bayesian methods is not feasible. In this paper, we aim to suggest an integrated, general approach to inference and model selection using composite likelihood…

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Empirical likelihood analysis of the rank estimator for the censored accelerated failure time model

删失加速失效时间模型秩估计量的经验似然分析

We use the empirical likelihood method to derive a test and thus a confidence interval based on the rank estimators of the regression coefficient in the accelerated failure time model. Standard chi-squared distributions are used to calculate the p-value and to construct the confidence interval. Simulations and examples show that the chi-squared approximation to the distribution of the log empirical likelihood ratio performs well, and has some advantages over the existing…

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Weighted least absolute deviations estimation for an AR(1) process with ARCH(1) errors

ARCH(1) 误差下 AR(1) 过程的加权最小绝对偏差估计

The weighted least absolute deviations estimator is studied for an AR(1) process with ARCH(1) errors εt. Unlike for the quasi maximum likelihood estimator, the estimator's, limiting distribution is shown to be normal even when E(εt4) = ∞. Furthermore, the estimator can be applied to examine the symmetry of the density of εt and to estimate the quantity E(log |α + λ½ εt|), which are of crucial importance for conducting asymptotic inference for quasi maximum likelihood…

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Mean estimating equation approach to analysing cluster-correlated data with nonignorable cluster sizes

用均值估计方程分析簇大小不可忽略的簇相关数据

Most methods for analysing cluster-correlated biological data implicitly assume the ignorability of cluster sizes. When this assumption fails, the resulting inferences may be asymptotically invalid. Hoffman et al. (2001) proposed a simple but computationally intensive method, based on a large number of within-cluster resamples and associated separate estimating equations, that leads to asymptotically valid inferences whether the cluster sizes are ignorable or not. We study a…

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Some nonregular designs from the Nordstrom–Robinson code and their statistical properties

由 Nordstrom–Robinson 码构造的非正规设计及其统计性质

The Nordstrom–Robinson code is a well-known nonlinear code in coding theory. This paper explores the statistical properties of this nonlinear code. Many nonregular designs with 32, 64, 128 and 256 runs and 7–16 factors are derived from it. It is shown that these nonregular designs are better than regular designs of the same size in terms of resolution, aberration and projectivity. Furthermore, many of these nonregular designs are shown to have generalised minimum aberration…

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Direction estimation in single-index regressions

单指标回归中的方向估计

We propose a general dimension-reduction method that combines the ideas of likelihood, correlation, inverse regression and information theory. We do not require that the dependence be confined to particular conditional moments, nor do we place restrictions on the predictors or on the regression that are necessary for methods like ordinary least squares and sliced-inverse regression. Although we focus on single-index regressions, the underlying idea is applicable more…

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On the identification of path analysis models with one hidden variable

含一个隐藏变量的路径分析模型的识别

We study criteria for identifiability of path analysis models with one hidden variable. We first derive sufficient criteria for identification of models in which marginalisation is carried out over the hidden variable. The sufficient criteria are based on the structure of the directed acyclic graph associated with the path analysis model and can be derived from the graph. We treat further the identification of models when the hidden variable is conditioned on and establish…

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A Monte Carlo method for computing the marginal likelihood in nondecomposable Gaussian graphical models

非可分解高斯图模型边际似然的蒙特卡洛计算

A centred Gaussian model that is Markov with respect to an undirected graph G is characterised by the parameter set of its precision matrices which is the cone M+(G) of positive definite matrices with entries corresponding to the missing edges of G constrained to be equal to zero. In a Bayesian framework, the conjugate family for the precision parameter is the distribution with Wishart density with respect to the Lebesgue measure restricted to M+(G). We call this…

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Variable selection for multivariate failure time data

多元失效时间数据的变量选择

In this paper, we proposed a penalised pseudo-partial likelihood method for variable selection with multivariate failure time data with a growing number of regression coefficients. Under certain regularity conditions, we show the consistency and asymptotic normality of the penalised likelihood estimators. We further demonstrate that, for certain penalty functions with proper choices of regularisation parameters, the resulting estimator can correctly identify the true model,…

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Additive hazards Markov regression models illustrated with bone marrow transplant data

加性风险 Markov 回归模型及骨髓移植数据示例

When there are covariate effects to be considered, multi-state survival analysis is dominated either by parametric Markov regression models or by semiparametric Markov regression models using Cox's (1972) proportional hazards models for transition intensities between the states. The purpose of this research work is to study alternatives to Cox's model in a general finite-state Markov process setting. We shall look at two alternative models, Aalen's (1989) nonparametric…

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Empirical-likelihood-based semiparametric inference for the treatment effect in the two-sample problem with censoring

删失两样本问题中治疗效应的经验似然半参数推断

To compare two samples of censored data, we propose a unified semiparametric inference for the parameter of interest when the model for one sample is parametric and that for the other is nonparametric. The parameter of interest may represent, for example, a comparison of means, or survival probabilities. The confidence interval derived from the semiparametric inference, which is based on the empirical likelihood principle, improves its counterpart constructed from the common…

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Expected lengths of confidence intervals based on empirical discrepancy statistics

基于经验差异统计量的置信区间期望长度

We consider a very general class of empirical discrepancy statistics that includes the Cressie–Read discrepancy statistics and, in particular, the empirical likelihood ratio statistic. Higher-order asymptotics for expected lengths of associated confidence intervals are investigated. An explicit formula is worked out and its use for comparative purposes is discussed. It is seen that the empirical likelihood ratio statistic, which enjoys interesting second-order power…

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Monte Carlo conditioning on a sufficient statistic

给定充分统计量的蒙特卡洛条件化

In this paper we derive general formulae suitable for Monte Carlo computation of conditional expectations of functions of a random vector given a sufficient statistic. The problem of direct sampling from the conditional distribution is considered in particular. It is shown that this can be done by a simple parameter adjustment of the original statistical model, provided the model has a certain pivotal structure. A connection with a classical problem regarding fiducial and…

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