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  • Interval estimation of a population mean using existing knowledge or data on effect sizes.

    abstract::Bayes or empirical Bayes methods to improve inferential accuracy for a population mean has been widely adopted in medical research. As the joint prior distribution of both the mean and variance parameters can be difficult to specify or estimate, most of these methods have relied on certain level of simplifications of ...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280218773537

    authors: Shen C

    更新日期:2019-06-01 00:00:00

  • A new approach to hierarchical data analysis: Targeted maximum likelihood estimation for the causal effect of a cluster-level exposure.

    abstract::We often seek to estimate the impact of an exposure naturally occurring or randomly assigned at the cluster-level. For example, the literature on neighborhood determinants of health continues to grow. Likewise, community randomized trials are applied to learn about real-world implementation, sustainability, and popula...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280218774936

    authors: Balzer LB,Zheng W,van der Laan MJ,Petersen ML

    更新日期:2019-06-01 00:00:00

  • Shared parameter models for joint analysis of longitudinal and survival data with left truncation due to delayed entry - Applications to cystic fibrosis.

    abstract::Many longitudinal studies observe time to occurrence of a clinical event such as death, while also collecting serial measurements of one or more biomarkers that are predictive of the event, or are surrogate outcomes of interest. Joint modeling can be used to examine the relationship between the biomarker and the event...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280218764193

    authors: Schluchter MD,Piccorelli AV

    更新日期:2019-05-01 00:00:00

  • Measuring diagnostic accuracy for biomarkers under tree-ordering.

    abstract::In the field of diagnostic studies for tree or umbrella ordering, under which the marker measurement for one class is lower or higher than those for the rest unordered classes, there exist a few diagnostic measures such as the naive AUC ( NAUC), the umbrella volume ( UV), and the recently proposed TAUC, i.e. area unde...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280218755810

    authors: Feng Y,Tian L

    更新日期:2019-05-01 00:00:00

  • Separating variability in healthcare practice patterns from random error.

    abstract::Improving the quality of care that patients receive is a major focus of clinical research, particularly in the setting of cardiovascular hospitalization. Quality improvement studies seek to estimate and visualize the degree of variability in dichotomous treatment patterns and outcomes across different providers, where...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280217754230

    authors: Thomas LE,Schulte PJ

    更新日期:2019-04-01 00:00:00

  • A mixed-effects, spatially varying coefficients model with application to multi-resolution functional magnetic resonance imaging data.

    abstract::Spatial resolution plays an important role in functional magnetic resonance imaging studies as the signal-to-noise ratio increases linearly with voxel volume. In scientific studies, where functional magnetic resonance imaging is widely used, the standard spatial resolution typically used is relatively low which ensure...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280217752378

    authors: Liu Z,Bartsch AJ,Berrocal VJ,Johnson TD

    更新日期:2019-04-01 00:00:00

  • A test of inflated zeros for Poisson regression models.

    abstract::Excessive zeros are common in practice and may cause overdispersion and invalidate inference when fitting Poisson regression models. There is a large body of literature on zero-inflated Poisson models. However, methods for testing whether there are excessive zeros are less well developed. The Vuong test comparing a Po...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280217749991

    authors: He H,Zhang H,Ye P,Tang W

    更新日期:2019-04-01 00:00:00

  • Quantile residual lifetime regression with functional principal component analysis of longitudinal data for dynamic prediction.

    abstract::Optimal therapeutic decisions can be made according to disease prognosis, where the residual lifetime is extensively used because of its straightforward interpretation and formula. To predict the residual lifetime in a dynamic manner, a longitudinal biomarker that is repeatedly measured during the post-baseline follow...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280217753466

    authors: Lin X,Li R,Yan F,Lu T,Huang X

    更新日期:2019-04-01 00:00:00

  • Modelling breast cancer tumour growth for a stable disease population.

    abstract::Statistical models of breast cancer tumour progression have been used to further our knowledge of the natural history of breast cancer, to evaluate mammography screening in terms of mortality, to estimate overdiagnosis, and to estimate the impact of lead-time bias when comparing survival times between screen detected ...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280217734583

    authors: Isheden G,Humphreys K

    更新日期:2019-03-01 00:00:00

  • Power and sample size for multivariate logistic modeling of unmatched case-control studies.

    abstract::Sample size calculations are needed to design and assess the feasibility of case-control studies. Although such calculations are readily available for simple case-control designs and univariate analyses, there is limited theory and software for multivariate unconditional logistic analysis of case-control data. Here we...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280217737157

    authors: Gail MH,Haneuse S

    更新日期:2019-03-01 00:00:00

  • Adjustment for treatment changes in epilepsy trials: A comparison of causal methods for time-to-event outcomes.

    abstract:BACKGROUND:When trials are subject to departures from randomised treatment, simple statistical methods that aim to estimate treatment efficacy, such as per protocol or as treated analyses, typically introduce selection bias. More appropriate methods to adjust for departure from randomised treatment are rarely employed,...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280217735560

    authors: Dodd S,Williamson P,White IR

    更新日期:2019-03-01 00:00:00

  • A stochastic estimation procedure for intermittently-observed semi-Markov multistate models with back transitions.

    abstract::Multistate models provide an important method for analyzing a wide range of life history processes including disease progression and patient recovery following medical intervention. Panel data consisting of the states occupied by an individual at a series of discrete time points are often used to estimate transition i...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280217736342

    authors: Aralis H,Brookmeyer R

    更新日期:2019-03-01 00:00:00

  • Interpretation of mixed models and marginal models with cohort attrition due to death and drop-out.

    abstract::Mixed models estimated by maximum likelihood and marginal models estimated by generalized estimating equations are the standard methods for the analysis of longitudinal data. However, their use is highly debated when attrition may be due to death. While some authors consider that mixed model estimates are interpretabl...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280217723675

    authors: Rouanet A,Helmer C,Dartigues JF,Jacqmin-Gadda H

    更新日期:2019-02-01 00:00:00

  • Survival forests for data with dependent censoring.

    abstract::Tree-based methods are very powerful and popular tools for analysing survival data with right-censoring. The existing methods assume that the true time-to-event and the censoring times are independent given the covariates. We propose different ways to build survival forests when dependent censoring is suspected, by us...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280217727314

    authors: Moradian H,Larocque D,Bellavance F

    更新日期:2019-02-01 00:00:00

  • Flexible Bayesian P-splines for smoothing age-specific spatio-temporal mortality patterns.

    abstract::In this paper age-space-time models based on one and two-dimensional P-splines with B-spline bases are proposed for smoothing mortality rates, where both fixed relative scale and scale invariant two-dimensional penalties are examined. Model fitting and inference are carried out using integrated nested Laplace approxim...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280217726802

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

    更新日期:2019-02-01 00:00:00

  • A unified approach for assessing heterogeneity in age-period-cohort model parameters using random effects.

    abstract::Age-period-cohort models are a popular tool for studying population-level rates; for example, trends in cancer incidence and mortality. Age-period-cohort models decompose observed trends into age effects that correlate with natural history, period effects that reveal factors impacting all ages simultaneously (e.g. inn...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280217713033

    authors: Chernyavskiy P,Little MP,Rosenberg PS

    更新日期:2019-01-01 00:00:00

  • A robust imputation method for missing responses and covariates in sample selection models.

    abstract::Sample selection arises when the outcome of interest is partially observed in a study. Although sophisticated statistical methods in the parametric and non-parametric framework have been proposed to solve this problem, it is yet unclear how to deal with selectively missing covariate data using simple multiple imputati...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280217715663

    authors: Ogundimu EO,Collins GS

    更新日期:2019-01-01 00:00:00

  • Meta-analysis without study-specific variance information: Heterogeneity case.

    abstract::The random effects model in meta-analysis is a standard statistical tool often used to analyze the effect sizes of the quantity of interest if there is heterogeneity between studies. In the special case considered here, meta-analytic data contain only the sample means in two treatment arms and the sample sizes, but no...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280217718867

    authors: Sangnawakij P,Böhning D,Niwitpong SA,Adams S,Stanton M,Holling H

    更新日期:2019-01-01 00:00:00

  • Letter to the editor: Fitting truncated normal distributions.

    abstract::I comment here on a recent paper in this journal, on the fitting of truncated normal distributions by the EM algorithm. I show that the fitting of such distributions by direct numerical maximization of likelihood (rather than EM) is straightforward, contrary to an assertion made by the authors of that paper. ...

    journal_title:Statistical methods in medical research

    pub_type: 评论,信件

    doi:10.1177/0962280217712089

    authors: MacDonald IL

    更新日期:2018-12-01 00:00:00

  • Expected p-values in light of an ROC curve analysis applied to optimal multiple testing procedures.

    abstract::Many statistical studies report p-values for inferential purposes. In several scenarios, the stochastic aspect of p-values is neglected, which may contribute to drawing wrong conclusions in real data experiments. The stochastic nature of p-values makes their use to examine the performance of given testing procedures o...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280217704451

    authors: Vexler A,Yu J,Zhao Y,Hutson AD,Gurevich G

    更新日期:2018-12-01 00:00:00

  • Multilevel growth curve models that incorporate a random coefficient model for the level 1 variance function.

    abstract::Aim To present a flexible model for repeated measures longitudinal growth data within individuals that allows trends over time to incorporate individual-specific random effects. These may reflect the timing of growth events and characterise within-individual variability which can be modelled as a function of age. Subj...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280217706728

    authors: Goldstein H,Leckie G,Charlton C,Tilling K,Browne WJ

    更新日期:2018-11-01 00:00:00

  • Optimal quantile level selection for disease classification and biomarker discovery with application to electrocardiogram data.

    abstract::Classification with a large number of predictors and biomarker discovery become increasingly important in biological and medical research. This paper focuses on performing classification of cardiovascular diseases based on electrocardiogram analysis which deals with many variables and a lot of measurements within vari...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280217699996

    authors: Zhou Y,Huang R,Yu S,Ma Y

    更新日期:2018-11-01 00:00:00

  • A composite likelihood approach to predict the sex of the baby.

    abstract::Couples with diseases associated with the sexual chromosomes, as well as families in countries where the desire for a male is extreme, are interested in influencing the sex of the baby. We propose an original composite likelihood approach to analyse the relation between sex of the newborn and timing of the intercourse...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280217702415

    authors: Tiberi S,Scarpa B,Sartori N

    更新日期:2018-11-01 00:00:00

  • Model selection in multivariate semiparametric regression.

    abstract::Variable selection in semiparametric mixed models for longitudinal data remains a challenge, especially in the presence of multiple correlated outcomes. In this paper, we propose a model selection procedure that simultaneously selects fixed and random effects using a maximum penalized likelihood method with the adapti...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280217690769

    authors: Li Z,Liu H,Tu W

    更新日期:2018-10-01 00:00:00

  • Estimating marginal and incremental effects in the analysis of medical expenditure panel data using marginalized two-part random-effects generalized Gamma models: Evidence from China healthcare cost data.

    abstract::Conditional two-part random-effects models have been proposed for the analysis of healthcare cost panel data that contain both zero costs from the non-users of healthcare facilities and positive costs from the users. These models have been extended to accommodate more flexible data structures when using the generalize...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280217690770

    authors: Zhang B,Liu W,Hu Y

    更新日期:2018-10-01 00:00:00

  • An ad hoc method for dual adjusting for measurement errors and nonresponse bias for estimating prevalence in survey data: Application to Iranian mental health survey on any illicit drug use.

    abstract::Purpose The prevalence estimates of binary variables in sample surveys are often subject to two systematic errors: measurement error and nonresponse bias. A multiple-bias analysis is essential to adjust for both biases. Methods In this paper, we linked the latent class log-linear and proxy pattern-mixture models to ad...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280217690939

    authors: Khalagi K,Mansournia MA,Motevalian SA,Nourijelyani K,Rahimi-Movaghar A,Bakhtiyari M

    更新日期:2018-10-01 00:00:00

  • armDNA: A functional beta model for detecting age-related genomewide DNA methylation marks.

    abstract::DNA methylation has been shown to play an important role in many complex diseases. The rapid development of high-throughput DNA methylation scan technologies provides great opportunities for genomewide DNA methylation-disease association studies. As methylation is a dynamic process involving time, it is quite plausibl...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280216683571

    authors: Wang C,Shen Q,Du L,Xu J,Zhang H

    更新日期:2018-09-01 00:00:00

  • Fitting mechanistic epidemic models to data: A comparison of simple Markov chain Monte Carlo approaches.

    abstract::Simple mechanistic epidemic models are widely used for forecasting and parameter estimation of infectious diseases based on noisy case reporting data. Despite the widespread application of models to emerging infectious diseases, we know little about the comparative performance of standard computational-statistical fra...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280217747054

    authors: Li M,Dushoff J,Bolker BM

    更新日期:2018-07-01 00:00:00

  • The asymptotic maximal procedure for subject randomization in clinical trials.

    abstract::The maximal procedure is a restricted randomization method that maximizes the number of feasible allocation sequences under the constraints of the maximum tolerated imbalance and the allocation sequence length. It assigns an equal probability to all feasible sequences. However, its implementation is not easy due to th...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280216677107

    authors: Zhao W,Berger VW,Yu Z

    更新日期:2018-07-01 00:00:00

  • Measurement error, time lag, unmeasured confounding: Considerations for longitudinal estimation of the effect of a mediator in randomised clinical trials.

    abstract::Clinical trials are expensive and time-consuming and so should also be used to study how treatments work, allowing for the evaluation of theoretical treatment models and refinement and improvement of treatments. These treatment processes can be studied using mediation analysis. Randomised treatment makes some of the a...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280216666111

    authors: Goldsmith KA,Chalder T,White PD,Sharpe M,Pickles A

    更新日期:2018-06-01 00:00:00

  • Time-dependent efficacy of longitudinal biomarker for clinical endpoint.

    abstract::Joint modelling of longitudinal biomarker and event-time processes has gained its popularity in recent years as they yield more accurate and precise estimates. Considering this modelling framework, a new methodology for evaluating the time-dependent efficacy of a longitudinal biomarker for clinical endpoint is propose...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280216673084

    authors: Kolamunnage-Dona R,Williamson PR

    更新日期:2018-06-01 00:00:00

  • Efficient Monte Carlo evaluation of resampling-based hypothesis tests with applications to genetic epidemiology.

    abstract::Monte Carlo evaluation of resampling-based tests is often conducted in statistical analysis. However, this procedure is generally computationally intensive. The pooling resampling-based method has been developed to reduce the computational burden but the validity of the method has not been studied before. In this arti...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280216661876

    authors: Fung WK,Yu K,Yang Y,Zhou JY

    更新日期:2018-05-01 00:00:00

  • Analysis of phase II methodologies for single-arm clinical trials with multiple endpoints in rare cancers: An example in Ewing's sarcoma.

    abstract::Trials run in either rare diseases, such as rare cancers, or rare sub-populations of common diseases are challenging in terms of identifying, recruiting and treating sufficient patients in a sensible period. Treatments for rare diseases are often designed for other disease areas and then later proposed as possible tre...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280216662070

    authors: Dutton P,Love SB,Billingham L,Hassan AB

    更新日期:2018-05-01 00:00:00

  • Inferential tools in penalized logistic regression for small and sparse data: A comparative study.

    abstract::This paper focuses on inferential tools in the logistic regression model fitted by the Firth penalized likelihood. In this context, the Likelihood Ratio statistic is often reported to be the preferred choice as compared to the 'traditional' Wald statistic. In this work, we consider and discuss a wider range of test st...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280216661213

    authors: Siino M,Fasola S,Muggeo VM

    更新日期:2018-05-01 00:00:00

  • Semiparametric models for multilevel overdispersed count data with extra zeros.

    abstract::This study proposes semiparametric models for analysis of hierarchical count data containing excess zeros and overdispersion simultaneously. The methods discussed in this paper handle nonlinear covariate effects through flexible semiparametric multilevel regression techniques. This is performed by providing a comprehe...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280216657376

    authors: Mahmoodi M,Moghimbeigi A,Mohammad K,Faradmal J

    更新日期:2018-04-01 00:00:00

  • Joint modeling of longitudinal zero-inflated count and time-to-event data: A Bayesian perspective.

    abstract::Longitudinal zero-inflated count data are encountered frequently in substance-use research when assessing the effects of covariates and risk factors on outcomes. Often, both the time to a terminal event such as death or dropout and repeated measure count responses are collected for each subject. In this setting, the l...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280216659312

    authors: Zhu H,DeSantis SM,Luo S

    更新日期:2018-04-01 00:00:00

  • Bayesian variable selection in the accelerated failure time model with an application to the surveillance, epidemiology, and end results breast cancer data.

    abstract::Accelerated failure time model is a popular model to analyze censored time-to-event data. Analysis of this model without assuming any parametric distribution for the model error is challenging, and the model complexity is enhanced in the presence of large number of covariates. We developed a nonparametric Bayesian met...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280215626947

    authors: Zhang Z,Sinha S,Maiti T,Shipp E

    更新日期:2018-04-01 00:00:00

  • A measure of association for ordered categorical data in population-based studies.

    abstract::Ordinal classification scales are commonly used to define a patient's disease status in screening and diagnostic tests such as mammography. Challenges arise in agreement studies when evaluating the association between many raters' classifications of patients' disease or health status when an ordered categorical scale ...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280216643347

    authors: Nelson KP,Edwards D

    更新日期:2018-03-01 00:00:00

  • Bayesian nonparametric inference for the three-class Youden index and its associated optimal cutoff points.

    abstract::The three-class Youden index serves both as a measure of medical test accuracy and a criterion to choose the optimal pair of cutoff values for classifying subjects into three ordinal disease categories (e.g. no disease, mild disease, advanced disease). We present a Bayesian nonparametric approach for estimating the th...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280217742538

    authors: Carvalho VI,Branscum AJ

    更新日期:2018-03-01 00:00:00

  • Correcting for dependent censoring in routine outcome monitoring data by applying the inverse probability censoring weighted estimator.

    abstract::Censored data make survival analysis more complicated because exact event times are not observed. Statistical methodology developed to account for censored observations assumes that patients' withdrawal from a study is independent of the event of interest. However, in practice, some covariates might be associated to b...

    journal_title:Statistical methods in medical research

    pub_type: 杂志文章

    doi:10.1177/0962280216628900

    authors: Willems S,Schat A,van Noorden MS,Fiocco M

    更新日期:2018-02-01 00:00:00

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