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  • Obtaining evidence by a single well-powered trial or several modestly powered trials.

    abstract::There is debate whether clinical trials with suboptimal power are justified and whether results from large studies are more reliable than the (combined) results of smaller trials. We quantified the error rates for evaluations based on single conventionally powered trials (80% or 90% power) versus evaluations based on ...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280212461098

    authors: IntHout J,Ioannidis JP,Borm GF

    更新日期:2016-04-01 00:00:00

  • A Bayesian semiparametric approach with change points for spatial ordinal data.

    abstract::The change-point model has drawn much attention over the past few decades. It can accommodate the jump process, which allows for changes of the effects before and after the change point. Intellectual disability is a long-term disability that impacts performance in cognitive aspects of life and usually has its onset pr...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280212463415

    authors: Cai B,Lawson AB,McDermott S,Aelion CM

    更新日期:2016-04-01 00:00:00

  • Unconditional tests for comparing two ordered multinomials.

    abstract::We consider two exact unconditional procedures to test the difference between two multinomials with ordered categorical data. Exact unconditional procedures are compared to other approaches based on the Wilcoxon mid-rank test and the proportional odds model. We use a real example from an arthritis pain study to illust...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280212450957

    authors: Shan G,Ma C

    更新日期:2016-02-01 00:00:00

  • Modeling fecundity in the presence of a sterile fraction using a semi-parametric transformation model for grouped survival data.

    abstract::The analysis of fecundity data is challenging and requires consideration of both highly timed and interrelated biologic processes in the context of essential behaviors such as sexual intercourse during the fertile window. Understanding human fecundity is further complicated by presence of a sterile population, i.e. co...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280212438646

    authors: McLain AC,Sundaram R,Buck Louis GM

    更新日期:2016-02-01 00:00:00

  • A transformation class for spatio-temporal survival data with a cure fraction.

    abstract::We propose a hierarchical Bayesian methodology to model spatially or spatio-temporal clustered survival data with possibility of cure. A flexible continuous transformation class of survival curves indexed by a single parameter is used. This transformation model is a larger class of models containing two special cases ...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280212445658

    authors: Hurtado Rúa SM,Dey DK

    更新日期:2016-02-01 00:00:00

  • Linear time-dependent reference intervals where there is measurement error in the time variable-a parametric approach.

    abstract::This article re-examines parametric methods for the calculation of time specific reference intervals where there is measurement error present in the time covariate. Previous published work has commonly been based on the standard ordinary least squares approach, weighted where appropriate. In fact, this is an incorrect...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280211426617

    authors: Gillard J

    更新日期:2015-12-01 00:00:00

  • A comparison of power analysis methods for evaluating effects of a predictor on slopes in longitudinal designs with missing data.

    abstract::In many longitudinal studies, evaluating the effect of a binary or continuous predictor variable on the rate of change of the outcome, i.e. slope, is often of primary interest. Sample size determination of these studies, however, is complicated by the expectation that missing data will occur due to missed visits, earl...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280212437452

    authors: Wang C,Hall CB,Kim M

    更新日期:2015-12-01 00:00:00

  • Sample size calculation for treatment effects in randomized trials with fixed cluster sizes and heterogeneous intraclass correlations and variances.

    abstract::When comparing two different kinds of group therapy or two individual treatments where patients within each arm are nested within care providers, clustering of observations may occur in both arms. The arms may differ in terms of (a) the intraclass correlation, (b) the outcome variance, (c) the cluster size, and (d) th...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280214563100

    authors: Candel MJ,van Breukelen GJ

    更新日期:2015-10-01 00:00:00

  • Measuring continuous baseline covariate imbalances in clinical trial data.

    abstract::This paper presents and compares several methods of measuring continuous baseline covariate imbalance in clinical trial data. Simulations illustrate that though the t-test is an inappropriate method of assessing continuous baseline covariate imbalance, the test statistic itself is a robust measure in capturing imbalan...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280211416038

    authors: Ciolino JD,Martin RH,Zhao W,Hill MD,Jauch EC,Palesch YY

    更新日期:2015-04-01 00:00:00

  • Prospective analysis of infectious disease surveillance data using syndromic information.

    abstract::In this paper, we describe a Bayesian hierarchical Poisson model for the prospective analysis of data for infectious diseases. The proposed model consists of two components. The first component describes the behavior of disease during nonepidemic periods and the second component represents the increase in disease coun...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280214527385

    authors: Corberán-Vallet A,Lawson AB

    更新日期:2014-12-01 00:00:00

  • Controlling for localised spatio-temporal autocorrelation in long-term air pollution and health studies.

    abstract::Estimating the long-term health impact of air pollution using an ecological spatio-temporal study design is a challenging task, due to the presence of residual spatio-temporal autocorrelation in the health counts after adjusting for the covariate effects. This autocorrelation is commonly modelled by a set of random ef...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280214527384

    authors: Lee D,Mitchell R

    更新日期:2014-12-01 00:00:00

  • On the power of the Cochran-Armitage test for trend in the presence of misclassification.

    abstract::The Cochran-Armitage (CA) test is commonly used in both epidemiology and genetics to test for linear trend in two-way tables with a binary outcome. There has been increasing interest in the power and size of the test and in determination of sample size, especially when there is potential misclassification in the 'expo...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280211406424

    authors: Buonaccorsi JP,Laake P,Veierød MB

    更新日期:2014-06-01 00:00:00

  • Interpolation between spatial frameworks: an application of process convolution to estimating neighbourhood disease prevalence.

    abstract::Health data may be collected across one spatial framework (e.g. health provider agencies), but contrasts in health over another spatial framework (neighbourhoods) may be of policy interest. In the UK, population prevalence totals for chronic diseases are provided for populations served by general practitioner practice...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280212447150

    authors: Congdon P

    更新日期:2014-04-01 00:00:00

  • Joint latent class models for longitudinal and time-to-event data: a review.

    abstract::Most statistical developments in the joint modelling area have focused on the shared random-effect models that include characteristics of the longitudinal marker as predictors in the model for the time-to-event. A less well-known approach is the joint latent class model which consists in assuming that a latent class s...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章,评审

    doi:10.1177/0962280212445839

    authors: Proust-Lima C,Séne M,Taylor JM,Jacqmin-Gadda H

    更新日期:2014-02-01 00:00:00

  • Bayesian sample size calculation for estimation of the difference between two binomial proportions.

    abstract::In this study, we discuss a decision theoretic or fully Bayesian approach to the sample size question in clinical trials with binary responses. Data are assumed to come from two binomial distributions. A Dirichlet distribution is assumed to describe prior knowledge of the two success probabilities p1 and p2. The param...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280211399562

    authors: Pezeshk H,Nematollahi N,Maroufy V,Marriott P,Gittins J

    更新日期:2013-12-01 00:00:00

  • The cross-validated AUC for MCP-logistic regression with high-dimensional data.

    abstract::We propose a cross-validated area under the receiving operator characteristic (ROC) curve (CV-AUC) criterion for tuning parameter selection for penalized methods in sparse, high-dimensional logistic regression models. We use this criterion in combination with the minimax concave penalty (MCP) method for variable selec...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280211428385

    authors: Jiang D,Huang J,Zhang Y

    更新日期:2013-10-01 00:00:00

  • Controlling false positive selections in high-dimensional regression and causal inference.

    abstract::Guarding against false positive selections is important in many applications. We discuss methods based on subsampling and sample splitting for controlling the expected number of false positives and assigning p-values. They are generic and especially useful for high-dimensional settings. We review encouraging results f...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280211428371

    authors: Bühlmann P,Rütimann P,Kalisch M

    更新日期:2013-10-01 00:00:00

  • Predicting brain activity using a Bayesian spatial model.

    abstract::Increasing the clinical applicability of functional neuroimaging technology is an emerging objective, e.g. for diagnostic and treatment purposes. We propose a novel Bayesian spatial hierarchical framework for predicting follow-up neural activity based on an individual's baseline functional neuroimaging data. Our appro...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280212448972

    authors: Derado G,Bowman FD,Zhang L,Alzheimer's Disease Neuroimaging Initiative Investigators.

    更新日期:2013-08-01 00:00:00

  • Projections of cancer mortality risks using spatio-temporal P-spline models.

    abstract::Cancer mortality risk estimates are essential for planning resource allocation and designing and evaluating cancer prevention and management strategies. However, mortality figures generally become available after a few years, making necessary to develop reliable procedures to provide current and near future mortality ...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280212446366

    authors: Ugarte MD,Goicoa T,Etxeberria J,Militino AF

    更新日期:2012-10-01 00:00:00

  • Everything all right in method comparison studies?

    abstract::Researchers and clinicians often need to know whether a new method of measurement is equivalent to an established one that is already in use. For this problem, the estimation of limits of agreement advocated by Bland and Altman is a widely used solution. However, this approach ignores two vital issues in method compar...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280210379365

    authors: Alanen E

    更新日期:2012-08-01 00:00:00

  • Propensity scores: from naive enthusiasm to intuitive understanding.

    abstract::Estimation of the effect of a binary exposure on an outcome in the presence of confounding is often carried out via outcome regression modelling. An alternative approach is to use propensity score methodology. The propensity score is the conditional probability of receiving the exposure given the observed covariates a...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280210394483

    authors: Williamson E,Morley R,Lucas A,Carpenter J

    更新日期:2012-06-01 00:00:00

  • Multiplicity adjustments in trials with two correlated comparisons of interest.

    abstract::Clinical trials investigating the efficacy of two or more doses of an experimental treatment compared to a single reference arm are not uncommon. In such situations, if each dose is compared to the reference arm using an un-adjusted significance level, consideration of the Type I familywise error is likely to be requi...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280210378943

    authors: Fernandes N,Stone A

    更新日期:2011-12-01 00:00:00

  • A frequentist approach to estimating the force of infection for a respiratory disease using repeated measurement data from a birth cohort.

    abstract::This article aims to develop a probability-based model involving the use of direct likelihood formulation and generalised linear modelling (GLM) approaches useful in estimating important disease parameters from longitudinal or repeated measurement data. The current application is based on infection with respiratory sy...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280210385749

    authors: Mwambi H,Ramroop S,White Lj,Okiro E,Nokes Dj,Shkedy Z,Molenberghs G

    更新日期:2011-10-01 00:00:00

  • Inferences about a linear combination of proportions.

    abstract::Statistical methods for carrying out asymptotic inferences (tests or confidence intervals) relative to one or two independent binomial proportions are very frequent. However, inferences about a linear combination of K independent proportions L = Σβ(i)p(i) (in which the first two are special cases) have had very little...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280209347953

    authors: Martín Andrés A,Alvarez Hernández M,Herranz Tejedor I

    更新日期:2011-08-01 00:00:00

  • Estimating the personal cure rate of cancer patients using population-based grouped cancer survival data.

    abstract::Cancer patients are subject to multiple competing risks of death and may die from causes other than the cancer diagnosed. The probability of not dying from the cancer diagnosed, which is one of the patients' main concerns, is sometimes called the 'personal cure' rate. Two approaches of modelling competing-risk surviva...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280209347046

    authors: Binbing Yu,Tiwari RC,Feuer EJ

    更新日期:2011-06-01 00:00:00

  • A kernel-based spatio-temporal surveillance system for monitoring influenza-like illness incidence.

    abstract::The threat of pandemics has made influenza surveillance systems a priority in epidemiology services around the world. The emergence of A-H1N1 influenza has required accurate surveillance systems in order to undertake specific actions only when and where they are necessary. In that sense, the main goal of this article ...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280210370265

    authors: Martinez-Beneito MA,Botella-Rocamora P,Zurriaga O

    更新日期:2011-04-01 00:00:00

  • Multi-state Markov models in cancer screening evaluation: a brief review and case study.

    abstract::This work presents a brief overview of Markov models in cancer screening evaluation and focuses on two specific models. A three-state model was first proposed to estimate jointly the sensitivity of the screening procedure and the average duration in the preclinical phase, i.e. the period when the cancer is asymptomati...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章,评审

    doi:10.1177/0962280209359848

    authors: Uhry Z,Hédelin G,Colonna M,Asselain B,Arveux P,Rogel A,Exbrayat C,Guldenfels C,Courtial I,Soler-Michel P,Molinié F,Eilstein D,Duffy SW

    更新日期:2010-10-01 00:00:00

  • Latent mixture models for multivariate and longitudinal outcomes.

    abstract::Repeated measures and multivariate outcomes are an increasingly common feature of trials. Their joint analysis by means of random effects and latent variable models is appealing but patterns of heterogeneity in outcome profile may not conform to standard multivariate normal assumptions. In addition, there is much inte...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章,评审

    doi:10.1177/0962280209105016

    authors: Pickles A,Croudace T

    更新日期:2010-06-01 00:00:00

  • Pseudo-observations in survival analysis.

    abstract::We review recent work on the application of pseudo-observations in survival and event history analysis. This includes regression models for parameters like the survival function in a single point, the restricted mean survival time and transition or state occupation probabilities in multi-state models, e.g. the competi...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章,评审

    doi:10.1177/0962280209105020

    authors: Andersen PK,Perme MP

    更新日期:2010-02-01 00:00:00

  • Stochastic models of sequence evolution including insertion-deletion events.

    abstract::Comparison of sequences that have descended from a common ancestor based on an explicit stochastic model of substitutions, insertions and deletions has risen to prominence in the last decade. Making statements about the positions of insertions-deletions (abbr. indels) is central in sequence and genome analysis and is ...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280208099500

    authors: Miklós I,Novák A,Satija R,Lyngsø R,Hein J

    更新日期:2009-10-01 00:00:00

  • Re-weighted inference about hepatitis C virus-infected communities when analysing diagnosed patients referred to liver clinics.

    abstract::To project national hepatitis C virus (HCV) burden, unbiased estimation of HCV progression to liver cirrhosis is required for the whole community of HCV-infected individuals. However, widely varying estimates of progression rates to cirrhosis have been produced. This disparity is partly associated with the statistical...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280208094688

    authors: Fu B,Tom BD,Bird SM

    更新日期:2009-06-01 00:00:00

  • Stratified and randomized play-the-winner rule.

    abstract::In this paper, a new allocation rule for treatment assignments in sequential clinical trials is proposed. The stratified and randomized play-the-winner rule (SRPWR) is an extension of the randomized play-the-winner rule to more than two treatments. It is applicable to cases where the probabilities of success of a trea...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280207081606

    authors: Liang Y,Carriere KC

    更新日期:2008-12-01 00:00:00

  • Practical issues arising in an exploratory analysis evaluating progression-free survival as a surrogate endpoint for overall survival in advanced colorectal cancer.

    abstract::This paper is based on a conference presentation in which several authors presented results from analyses of the same dataset concerning the evaluation of progression-free survival (PFS) as a surrogate endpoint for overall survival in advanced colorectal cancer clinical trials. In evaluating a potential surrogate endp...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280207081860

    authors: Hughes MD

    更新日期:2008-10-01 00:00:00

  • Relative efficiency of unequal cluster sizes for variance component estimation in cluster randomized and multicentre trials.

    abstract::Cluster randomized and multicentre trials evaluate the effect of a treatment on persons nested within clusters, for instance patients within clinics or pupils within schools. Although equal sample sizes per cluster are generally optimal for parameter estimation, they are rarely feasible. This paper addresses the relat...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280206079018

    authors: van Breukelen GJ,Candel MJ,Berger MP

    更新日期:2008-08-01 00:00:00

  • Evaluation of change in CD4+ cell counts in AIDS clinical trials.

    abstract::To evaluate the antiretroviral activity of antiretroviral agents and to compare the effects of two different antiretroviral agents, we propose a non-parametric mixed-effects model to investigate change of CD4+ counts. The proposed model and methods are applied to analyse the data from PACTG345 study. Population and in...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280206075524

    authors: Liang H

    更新日期:2008-04-01 00:00:00

  • Random-effects models for multivariate repeated measures.

    abstract::Mixed models are widely used for the analysis of one repeatedly measured outcome. If more than one outcome is present, a mixed model can be used for each one. These separate models can be tied together into a multivariate mixed model by specifying a joint distribution for their random effects. This strategy has been u...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280206075305

    authors: Fieuws S,Verbeke G,Molenberghs G

    更新日期:2007-10-01 00:00:00

  • Applying artificial neural networks to the diagnosis of organic dyspepsia.

    abstract:BACKGROUND:Dyspepsia diagnoses and treatment decisions are made in situations in which multiple factors must be taken into account. Evolving from neuro-biological insights, artificial neural networks (ANNs) can employ multiple factors in resolving medical prediction, classification, pattern recognition, and pattern com...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280206071839

    authors: García-Altés A,Santín D,Barenys M

    更新日期:2007-08-01 00:00:00

  • Evaluation of software for multiple imputation of semi-continuous data.

    abstract::It is now widely accepted that multiple imputation (MI) methods properly handle the uncertainty of missing data over single imputation methods. Several standard statistical software packages, such as SAS, R and STATA, have standard procedures or user-written programs to perform MI. The performance of these packages is...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280206074464

    authors: Yu LM,Burton A,Rivero-Arias O

    更新日期:2007-06-01 00:00:00

  • Optimal vaccination schemes for epidemics among a population of households, with application to variola minor in Brazil.

    abstract::This paper is concerned with stochastic models for the spread of an epidemic among a community of households, in which individuals mix uniformly within households and, in addition, uniformly at a much lower rate within the population at large. This two-level mixing structure has important implications for the threshol...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280206071643

    authors: Ball F,Lyne O

    更新日期:2006-10-01 00:00:00

  • Checking linearity of non-parametric component in partially linear models with an application in systemic inflammatory response syndrome study.

    abstract::Two tests are proposed for checking the linearity of nonparametric function in partially linear models. The first one is based on a Crámer-von Mises statistic. This test can detect the local alternative converging to the null at the parametric rate 1/square root n. A bootstrap resample technique is provided to calcula...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1191/0962280206sm440oa

    authors: Liang H

    更新日期:2006-06-01 00:00:00

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