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 readers and cases contribute to the uncertainty of the estimated diagnostic performance, which is often measured by the area under the ROC curve (AUC). Due to limited prior information, sizing of such a study is often unreliable. It is desired to adaptively resize the study toward a target power after an interim analysis. Although adaptive methods are available in clinical trials where only the patient sample is sized, such methodologies have not been established for MRMC studies. The challenge lies in the fact that there is a correlation structure in MRMC data and the sizing involves both readers and cases. We develop adaptive MRMC design methodologies to enable study resizing. In particular, we resize the study and adjust the critical value for hypothesis testing simultaneously after an interim analysis to achieve a target power and control the type I error rate in comparing AUCs of two modalities. Analytical results have been derived. Simulations show that the type I error rate is controlled close to the nominal level and the power is adjusted toward the target value under a variety of simulation conditions. We demonstrate the use of our methods in a real-world application comparing two imaging modalities for breast cancer detection.
journal_name
Stat Methods Med Resjournal_title
Statistical methods in medical researchauthors
Huang Z,Samuelson F,Tcheuko L,Chen Wdoi
10.1177/0962280219869370subject
Has Abstractpub_date
2020-06-01 00:00:00pages
1592-1611issue
6eissn
0962-2802issn
1477-0334journal_volume
29pub_type
杂志文章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::In medical sciences, we often encounter longitudinal temporal relationships that are non-linear in nature. The influence of risk factors may also change across longitudinal follow-up. A system of multiphase non-linear mixed effects model is presented to model temporal patterns of longitudinal continuous measurements, ...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280214537255
更新日期:2017-02-01 00:00:00
abstract::Rapid Early Action for Coronary Treatment (REACT) was a multisite trial testing a community intervention to reduce the delay between onset of symptoms of acute myocardial infarction (MI) and patients' arrival at a hospital emergency department. The study employed a group-randomized trial design, with ten communities r...
journal_title:Statistical methods in medical research
pub_type: 临床试验,杂志文章,随机对照试验
doi:10.1177/096228020000900204
更新日期:2000-04-01 00:00:00
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
更新日期:2018-11-01 00:00:00
abstract::The analysis of health care costs is complicated by the skewed and heteroscedastic nature of their distribution with possibly additional zero values. Statistical methods that do not adjust for these features can lead to incorrect conclusions. This paper reviews recent developments in statistical methods for the analys...
journal_title:Statistical methods in medical research
pub_type: 杂志文章,评审
doi:10.1191/0962280202sm290ra
更新日期:2002-08-01 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
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
更新日期:2014-04-01 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::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::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
更新日期:2014-06-01 00:00:00
abstract::In this paper, we develop a simple diagnostic test for the random-effects distribution in mixed models. The test is based on the gradient function, a graphical tool proposed by Verbeke and Molenberghs to check the impact of assumptions about the random-effects distribution in mixed models on inferences. Inference is c...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280214564721
更新日期:2017-04-01 00:00:00
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
更新日期:2011-06-01 00:00:00
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
更新日期:2018-02-01 00:00:00
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
更新日期:2018-10-01 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::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::Identification of cancer patient subgroups using high throughput genomic data is of critical importance to clinicians and scientists because it can offer opportunities for more personalized treatment and overlapping treatments of cancers. In spite of tremendous efforts, this problem still remains challenging because o...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280217752980
更新日期:2019-07-01 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::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::The simple yet subtle concept of regression towards the mean is reviewed historically. Verbal, geometric, and mathematical expressions of the concept date to the discoverer of the concept, Francis Galton. That discovery and subsequent understanding (and misunderstanding) of the concept are surveyed. ...
journal_title:Statistical methods in medical research
pub_type: 传,历史文章,杂志文章,评审
doi:10.1177/096228029700600202
更新日期:1997-06-01 00:00:00
abstract::Cluster-level dynamic treatment regimens can be used to guide sequential treatment decision-making at the cluster level in order to improve outcomes at the individual or patient-level. In a cluster-level dynamic treatment regimen, the treatment is potentially adapted and re-adapted over time based on changes in the cl...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280217708654
更新日期:2017-08-01 00:00:00
abstract::In this paper we review methods of cluster analysis in the context of classifying patients on the basis of clinical and/or laboratory type observations. Both hierarchical and non-hierarchical methods of clustering are considered, although the emphasis is on the latter type, with particular attention devoted to the mix...
journal_title:Statistical methods in medical research
pub_type: 杂志文章,评审
doi:10.1177/096228029200100103
更新日期:1992-01-01 00:00:00
abstract::Covariate-adaptive designs are widely used to balance covariates and maintain randomization in clinical trials. Adaptive designs for discrete covariates and their asymptotic properties have been well studied in the literature. However, important continuous covariates are often involved in clinical studies. Simply disc...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280218770231
更新日期:2019-06-01 00:00:00
abstract::One purpose of a longitudinal study is to gain a better understanding of how an outcome of interest changes among a given population over time. In what follows, a trajectory will be taken to mean the series of measurements of the outcome variable for an individual. Group-based trajectory modelling methods seek to iden...
journal_title:Statistical methods in medical research
pub_type: 杂志文章
doi:10.1177/0962280215598806
更新日期:2017-08-01 00:00:00
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
更新日期:2019-04-01 00:00:00
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
更新日期:2018-06-01 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::Dependent binary response data arise frequently in practice due to repeated measurements in longitudinal studies or to subsampling primary sampling units as in fields such as teratology and ophthalmology. Several classes of approaches have recently been proposed to analyse such repeated binary outcome data. The differ...
journal_title:Statistical methods in medical research
pub_type: 杂志文章,评审
doi:10.1177/096228029200100303
更新日期:1992-01-01 00:00:00
abstract::Studies of HIV dynamics in AIDS research are very important for understanding pathogenesis of HIV infection and for assessing the potency of antiviral therapies. Since the viral dynamic results from clinical data were first published by Ho et al. and Wei et al., the study of HIV-1 dynamics in vivo has drawn a great at...
journal_title:Statistical methods in medical research
pub_type: 杂志文章,评审
doi:10.1191/0962280205sm390oa
更新日期:2005-04-01 00:00:00
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
更新日期:2018-03-01 00:00:00