Adaptive designs in multi-reader multi-case clinical trials of imaging devices.

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 Res

authors

Huang Z,Samuelson F,Tcheuko L,Chen W

doi

10.1177/0962280219869370

subject

Has Abstract

pub_date

2020-06-01 00:00:00

pages

1592-1611

issue

6

eissn

0962-2802

issn

1477-0334

journal_volume

29

pub_type

杂志文章
  • 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

  • A multiphase non-linear mixed effects model: An application to spirometry after lung transplantation.

    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

    authors: Rajeswaran J,Blackstone EH

    更新日期:2017-02-01 00:00:00

  • Components of variance in a group-randomized trial analysed via a random-coefficients model: the Rapid Early Action for Coronary Treatment (REACT) trial.

    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

    authors: Murray DM,Feldman HA,McGovern PG

    更新日期:2000-04-01 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

  • Inferences about population means of health care costs.

    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

    authors: Zhou XH

    更新日期:2002-08-01 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

  • 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

  • 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

  • Efficient algorithms for covariate analysis with dynamic data using nonlinear mixed-effects model.

    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

    authors: Yuan M,Zhu Z,Yang Y,Zhao M,Sasser K,Hamadeh H,Pinheiro J,Xu XS

    更新日期:2020-08-24 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

  • A goodness-of-fit test for the random-effects distribution in mixed models.

    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

    authors: Efendi A,Drikvandi R,Verbeke G,Molenberghs G

    更新日期:2017-04-01 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

  • 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

  • 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

  • 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

  • Penalized count data regression with application to hospital stay after pediatric cardiac surgery.

    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

    authors: Wang Z,Ma S,Zappitelli M,Parikh C,Wang CY,Devarajan P

    更新日期:2016-12-01 00:00:00

  • Semi-supervised identification of cancer subgroups using survival outcomes and overlapping grouping information.

    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

    authors: Wei W,Sun Z,da Silveira WA,Yu Z,Lawson A,Hardiman G,Kelemen LE,Chung D

    更新日期:2019-07-01 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

  • Extending backcalculation to analyse BSE data.

    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

    authors: Donnelly CA,Ferguson NM,Ghani AC,Anderson RM

    更新日期:2003-06-01 00:00:00

  • Regression towards the mean, historically considered.

    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

    authors: Stigler SM

    更新日期:1997-06-01 00:00:00

  • Comparing cluster-level dynamic treatment regimens using sequential, multiple assignment, randomized trials: Regression estimation and sample size considerations.

    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

    authors: NeCamp T,Kilbourne A,Almirall D

    更新日期:2017-08-01 00:00:00

  • Cluster analysis and related techniques in medical research.

    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

    authors: McLachlan GJ

    更新日期:1992-01-01 00:00:00

  • Testing hypotheses under adaptive randomization with continuous covariates in clinical trials.

    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

    authors: Li X,Zhou J,Hu F

    更新日期:2019-06-01 00:00:00

  • The impact of covariance misspecification in group-based trajectory models for longitudinal data with non-stationary covariance structure.

    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

    authors: Davies CE,Glonek GF,Giles LC

    更新日期:2017-08-01 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

  • 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

  • Designs in partially controlled studies: messages from a review.

    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

    authors: Li F,Frangakis CE

    更新日期:2005-08-01 00:00:00

  • Statistical methods for longitudinal and clustered designs with binary responses.

    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

    authors: Neuhaus JM

    更新日期:1992-01-01 00:00:00

  • Statistical methods for HIV dynamic studies in AIDS clinical trials.

    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

    authors: Wu H

    更新日期:2005-04-01 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