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 interest in both allowing for and identifying sub-groups of patients who vary in treatment responsiveness. We review methods based on discrete random effects distributions and mixture models for application in this field.
journal_name
Stat Methods Med Resjournal_title
Statistical methods in medical researchauthors
Pickles A,Croudace Tdoi
10.1177/0962280209105016subject
Has Abstractpub_date
2010-06-01 00:00:00pages
271-89issue
3eissn
0962-2802issn
1477-0334pii
0962280209105016journal_volume
19pub_type
杂志文章,评审abstract::Several epidemiological parameters have been introduced for quantifying the population impact of a certain exposure on morbidity on a population level, termed 'attributable risk' (AR). Of these definitions, the AR as suggested by Levin in 1953 or some algebraic transformations of it are most commonly used. A structure...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/096228020101000305
更新日期:2001-06-01 00:00:00
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
更新日期:2014-02-01 00:00:00
abstract::Dependent censoring arises in biomedical studies when the survival outcome of interest is censored by competing risks. In survival data with microarray gene expressions, gene selection based on the univariate Cox regression analyses has been used extensively in medical research, which however, is only valid under the ...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280214533378
更新日期:2016-12-01 00:00:00
abstract::We review the origins of backcalculation (or back projection) methods developed for the analysis of AIDS (acquired immunodeficiency syndrome) incidence data. These techniques have been used extensively for >15 years to deconvolute clinical case incidence, given knowledge of the incubation period distribution, to obtai...
journal_title:Statistical methods in medical research
pub_type: 杂志文章,评审
doi:10.1191/0962280203sm337ra
更新日期:2003-06-01 00:00:00
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
更新日期:2018-05-01 00:00:00
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
更新日期:2014-12-01 00:00:00
abstract::Evaluation of medical imaging devices often involves clinical studies where multiple readers (MR) read images of multiple cases (MC) for a clinical task, which are often called MRMC studies. In addition to sizing patient cases as is required in most clinical trials, MRMC studies also require sizing readers, since both...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280219869370
更新日期:2020-06-01 00:00:00
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
更新日期:2008-12-01 00:00:00
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
更新日期:2018-12-01 00:00:00
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
更新日期:2019-06-01 00:00:00
abstract::The ability to evaluate effects of factors on outcomes is increasingly important for studies that control some but not all of the factors. Although important advances have been made in methods of analysis for such partially controlled studies, work on designs has been limited. To help understand why, we review the mai...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1191/0962280205sm405oa
更新日期:2005-08-01 00:00:00
abstract::Measurement error is a serious problem in the analysis of epidemiological data. In the past 20 years, a large number of methods for the correction of measurement error have been developed. While at the beginning mostly methods for cohort studies were considered, recently more attention has been paid to case-control st...
journal_title:Statistical methods in medical research
pub_type: 杂志文章,评审
doi:10.1177/096228020000900504
更新日期:2000-10-01 00:00:00
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
更新日期:2009-10-01 00:00:00
abstract::Regression models are frequently used to model the functional relationship between an interesting outcome parameter and one or more potentially relevant explanatory variables. Objectives can be to set up as a prognostic model, for example, or an estimation model for a certain parameter of interest. Determining half-li...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280213502403
更新日期:2016-10-01 00:00:00
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
更新日期:2007-08-01 00:00:00
abstract::This review provides an overview of forensic inference from genetic markers. Because the judge and jurors are charged with decision-making, the forensic expert's job is to provide a useful summary of the evidence to the court. Hence, this review focuses on the likelihood ratio as a means of summarizing the genetic dat...
journal_title:Statistical methods in medical research
pub_type: 杂志文章,评审
doi:10.1177/096228029300200304
更新日期:1993-01-01 00:00:00
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
更新日期:2019-02-01 00:00:00
abstract::In recent years, disease mapping studies have become a routine application within geographical epidemiology and are typically analysed within a Bayesian hierarchical model formulation. A variety of model formulations for the latent level have been proposed but all come with inherent issues. In the classical BYM (Besag...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280216660421
更新日期:2016-08-01 00:00:00
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
更新日期:2013-08-01 00:00:00
abstract::Propensity score methods are common for estimating a binary treatment effect when treatment assignment is not randomized. When exposure is measured on an ordinal scale (i.e. low-medium-high), however, propensity score inference requires extensions which have received limited attention. Estimands of possible interest w...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280214560046
更新日期:2017-04-01 00:00:00
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
更新日期:2011-08-01 00:00:00
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journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280213484401
更新日期:2016-08-01 00:00:00
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journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280212437452
更新日期:2015-12-01 00:00:00
abstract::Joint models for recurrent and terminal events have not been yet developed for clustered data. The goals of our study are to develop a statistical framework for modelling clustered recurrent and terminal events and to perform dynamic predictions of the terminal event in family studies. We propose a joint nested frailt...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280219863076
更新日期:2020-05-01 00:00:00
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
更新日期:2019-03-01 00:00:00
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
更新日期:2018-11-01 00:00:00
abstract::Nonlinear mixed-effects modeling is one of the most popular tools for analyzing repeated measurement data, particularly for applications in the biomedical fields. Multiple integration and nonlinear optimization are the two major challenges for likelihood-based methods in nonlinear mixed-effects modeling. To solve thes...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280220949898
更新日期:2020-08-24 00:00:00
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
更新日期:2015-12-01 00:00:00
abstract::Pediatric cardiac surgery may lead to poor outcomes such as acute kidney injury (AKI) and prolonged hospital length of stay (LOS). Plasma and urine biomarkers may help with early identification and prediction of these adverse clinical outcomes. In a recent multi-center study, 311 children undergoing cardiac surgery we...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280214530608
更新日期:2016-12-01 00:00:00
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
更新日期:2019-03-01 00:00:00