Robust inference for the stepped wedge design.

Abstract:

:Stepped wedge designed trials are a type of cluster-randomized study in which the intervention is introduced to each cluster in a random order over time. This design is often used to assess the effect of a new intervention as it is rolled out across a series of clinics or communities. Based on a permutation argument, we derive a closed-form expression for an estimate of the intervention effect, along with its standard error, for a stepped wedge design trial. We show that these estimates are robust to misspecification of both the mean and covariance structure of the underlying data-generating mechanism, thereby providing a robust approach to inference for the intervention effect in stepped wedge designs. We use simulations to evaluate the type 1 error and power of the proposed estimate and to compare the performance of the proposed estimate to the optimal estimate when the correct model specification is known. The limitations, possible extensions, and open problems regarding the method are discussed.

journal_name

Biometrics

journal_title

Biometrics

authors

Hughes JP,Heagerty PJ,Xia F,Ren Y

doi

10.1111/biom.13106

subject

Has Abstract

pub_date

2020-03-01 00:00:00

pages

119-130

issue

1

eissn

0006-341X

issn

1541-0420

journal_volume

76

pub_type

杂志文章
  • Estimation of individual genetic effects from binary observations on relatives applied to a family history of respiratory illnesses and chronic lung disease of newborns.

    abstract::This paper considers methods for estimating the relationship between a binary response Y and the genetic effects responsible for a second binary trait Z. The responses Y are observed only for target individuals, and the responses Z are observed only for the relatives of these targets. The analysis consists of two part...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.0006-341x.2000.00808.x

    authors: Houwing-Duistermaat JJ,van Houwelingen HC,de Winter JP

    更新日期:2000-09-01 00:00:00

  • Response-adaptive regression for longitudinal data.

    abstract::We propose a response-adaptive model for functional linear regression, which is adapted to sparsely sampled longitudinal responses. Our method aims at predicting response trajectories and models the regression relationship by directly conditioning the sparse and irregular observations of the response on the predictor,...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.1541-0420.2010.01518.x

    authors: Wu S,Müller HG

    更新日期:2011-09-01 00:00:00

  • Statistical inference for serial dilution assay data.

    abstract::Serial dilution assays are widely employed for estimating substance concentrations and minimum inhibitory concentrations. The Poisson-Bernoulli model for such assays is appropriate for count data but not for continuous measurements that are encountered in applications involving substance concentrations. This paper pre...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.0006-341x.1999.01215.x

    authors: Lee ML,Whitmore GA

    更新日期:1999-12-01 00:00:00

  • Bayesian modeling of multiple lesion onset and growth from interval-censored data.

    abstract::In studying rates of occurrence and progression of lesions (or tumors), it is typically not possible to obtain exact onset times for each lesion. Instead, data consist of the number of lesions that reach a detectable size between screening examinations, along with measures of the size/severity of individual lesions at...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.0006-341X.2004.00217.x

    authors: Dunson DB,Holloman C,Calder C,Gunn LH

    更新日期:2004-09-01 00:00:00

  • One-sided sequential stopping boundaries for clinical trials: a decision-theoretic approach.

    abstract::We address one-sided stopping rules for clinical trials, or more generally, drug development programs, from a decision-theoretic point of view. If efficacy results are sufficiently negative then the trial will be stopped. But regardless of how positive the efficacy results are, the trial will continue in order to demo...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:

    authors: Berry DA,Ho CH

    更新日期:1988-03-01 00:00:00

  • Unbalanced regression analysis with residuals having a covariance structure of intra-class form.

    abstract::Let Yi be an ni X 1 vector of observations, Xi an ni X p matrix of known values, and beta an unknown p X 1 with the structure Yi = Xi beta + epsilon i, where the covariance matrix of epsilon i is of intra-class form, that is Cov (epsilon i) = sigma2[(1 - rho) Ii + rho e i e i'] where Ii is the ni X ni identity matrix ...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:

    authors: Wiorkowski JJ

    更新日期:1975-09-01 00:00:00

  • A comparison of several point estimators of the odds ratio in a single 2 x 2 contingency table.

    abstract::The relative performance of the unconditioned maximum likelihood estimators (UMLEs), conditional MLEs (CMLEs), and Jewell-type estimators of the odds ratio (OR) and its logarithm were investigated in sets of single 2 x 2 contingency tables. The tables were generated by complete enumeration of all possible cell frequen...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:

    authors: Walter SD,Cook RJ

    更新日期:1991-09-01 00:00:00

  • Maximum likelihood estimation in dynamical models of HIV.

    abstract::The study of dynamical models of HIV infection, based on a system of nonlinear ordinary differential equations (ODE), has considerably improved the knowledge of its pathogenesis. While the first models used simplified ODE systems and analyzed each patient separately, recent works dealt with inference in non-simplified...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.1541-0420.2007.00812.x

    authors: Guedj J,Thiébaut R,Commenges D

    更新日期:2007-12-01 00:00:00

  • A note on the conditional approach to interval estimation in the calibration problem.

    abstract::In the calibration problem, the need to construct a confidence interval to estimate the unknown chi 0 arises when the null hypothesis of zero slope is rejected. Otherwise, the resulting confidence interval will be infinite to reflect the fact that the slope of the regression line may be zero. Under the condition of re...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:

    authors: Lee JJ

    更新日期:1991-12-01 00:00:00

  • Latent variable models for longitudinal data with multiple continuous outcomes.

    abstract::Multiple outcomes are often used to properly characterize an effect of interest. This paper proposes a latent variable model for the situation where repeated measures over time are obtained on each outcome. These outcomes are assumed to measure an underlying quantity of main interest from different perspectives. We re...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.0006-341x.2000.01047.x

    authors: Roy J,Lin X

    更新日期:2000-12-01 00:00:00

  • Outlier reduction by an option-3 measurement scheme.

    abstract::Detecting changes in longitudinal data is important in medical research. However, the existence of measurement outliers can cause an unexpected increase in the false alarm rate in claiming changes. To reduce the outliers, a new method has been developed. In this scheme, two measures are initially taken and, if they ar...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:

    authors: Namgung YY,Yang MC

    更新日期:1994-03-01 00:00:00

  • Using empirical likelihood to combine data: application to food risk assessment.

    abstract::This article introduces an original methodology based on empirical likelihood, which aims at combining different food contamination and consumption surveys to provide risk managers with a risk measure, taking into account all the available information. This risk index is defined as the probability that exposure to a c...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.1541-0420.2008.01051.x

    authors: Crépet A,Harari-Kermadec H,Tressou J

    更新日期:2009-03-01 00:00:00

  • Two-group Poisson-Dirichlet mixtures for multiple testing.

    abstract::The simultaneous testing of multiple hypotheses is common to the analysis of high-dimensional data sets. The two-group model, first proposed by Efron, identifies significant comparisons by allocating observations to a mixture of an empirical null and an alternative distribution. In the Bayesian nonparametrics literatu...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/biom.13314

    authors: Denti F,Guindani M,Leisen F,Lijoi A,Wadsworth WD,Vannucci M

    更新日期:2020-06-14 00:00:00

  • Generalized estimating equation model for binary outcomes with missing covariates.

    abstract::This paper presents an approach to handling missing covariates in the generalized estimating equation (GEE) model for binary outcomes when the probability of missingness depends on the observed outcomes and covariates. The proposed method is to replace the missing quantities in the estimating function with consistent ...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:

    authors: Xie F,Paik MC

    更新日期:1997-12-01 00:00:00

  • Maximum likelihood estimation for N-mixture models.

    abstract::The focus of this article is on the nature of the likelihood associated with N-mixture models for repeated count data. It is shown that the infinite sum embedded in the likelihood associated with the Poisson mixing distribution can be expressed in terms of a hypergeometric function and, thence, in closed form. The res...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/biom.12521

    authors: Haines LM

    更新日期:2016-12-01 00:00:00

  • Semiparametric estimation of the covariate-specific ROC curve in presence of ignorable verification bias.

    abstract::Covariate-specific receiver operating characteristic (ROC) curves are often used to evaluate the classification accuracy of a medical diagnostic test or a biomarker, when the accuracy of the test is associated with certain covariates. In many large-scale screening tests, the gold standard is subject to missingness due...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.1541-0420.2011.01562.x

    authors: Liu D,Zhou XH

    更新日期:2011-09-01 00:00:00

  • Extension of the rank sum test for clustered data: two-group comparisons with group membership defined at the subunit level.

    abstract::The Wilcoxon rank sum test is widely used for two-group comparisons for nonnormal data. An assumption of this test is independence of sampling units both between and within groups. In ophthalmology, data are often collected on two eyes of an individual, which are highly correlated. In ophthalmological clinical trials,...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.1541-0420.2006.00582.x

    authors: Rosner B,Glynn RJ,Lee ML

    更新日期:2006-12-01 00:00:00

  • Multimodal neuroimaging data integration and pathway analysis.

    abstract::With advancements in technology, the collection of multiple types of measurements on a common set of subjects is becoming routine in science. Some notable examples include multimodal neuroimaging studies for the simultaneous investigation of brain structure and function and multi-omics studies for combining genetic an...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/biom.13351

    authors: Zhao Y,Li L,Caffo BS

    更新日期:2020-08-13 00:00:00

  • Bayesian experimental design for nonlinear mixed-effects models with application to HIV dynamics.

    abstract::Bayesian experimental design is investigated for Bayesian analysis of nonlinear mixed-effects models. Existence of the posterior risk for parameter estimation is shown. When the same prior distribution is used for both design and inference, existence of the preposterior risk for design is also proven. If the prior dis...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.0006-341X.2004.00148.x

    authors: Han C,Chaloner K

    更新日期:2004-03-01 00:00:00

  • Bayesian latent multi-state modeling for nonequidistant longitudinal electronic health records.

    abstract::Large amounts of longitudinal health records are now available for dynamic monitoring of the underlying processes governing the observations. However, the health status progression across time is not typically observed directly: records are observed only when a subject interacts with the system, yielding irregular and...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/biom.13261

    authors: Luo Y,Stephens DA,Verma A,Buckeridge DL

    更新日期:2020-03-11 00:00:00

  • Variance estimation for systematic designs in spatial surveys.

    abstract::In spatial surveys for estimating the density of objects in a survey region, systematic designs will generally yield lower variance than random designs. However, estimating the systematic variance is well known to be a difficult problem. Existing methods tend to overestimate the variance, so although the variance is g...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.1541-0420.2011.01604.x

    authors: Fewster RM

    更新日期:2011-12-01 00:00:00

  • Multivariate survival analysis using piecewise gamma frailty.

    abstract::In this note we propose a frailty model called piecewise gamma frailty for correlated survival data with random effects having a nested structure. In frailty models, a dependence function defined as a hazard ratio of one member given the failure time of another member in a unit is determined by the distributional assu...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:

    authors: Paik MC,Tsai WY,Ottman R

    更新日期:1994-12-01 00:00:00

  • Ranked set sampling with unequal samples.

    abstract::A ranked set sampling procedure with unequal samples (RSSU) is proposed and used to estimate the population mean. This estimator is then compared with the estimators based on the ranked set sampling (RSS) and median ranked set sampling (MRSS) procedures. It is shown that the relative precisions of the estimator based ...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.0006-341x.2001.00957.x

    authors: Bhoj DS

    更新日期:2001-09-01 00:00:00

  • Assessing effects of cholera vaccination in the presence of interference.

    abstract::Interference occurs when the treatment of one person affects the outcome of another. For example, in infectious diseases, whether one individual is vaccinated may affect whether another individual becomes infected or develops disease. Quantifying such indirect (or spillover) effects of vaccination could have important...

    journal_title:Biometrics

    pub_type: 杂志文章,随机对照试验

    doi:10.1111/biom.12184

    authors: Perez-Heydrich C,Hudgens MG,Halloran ME,Clemens JD,Ali M,Emch ME

    更新日期:2014-09-01 00:00:00

  • Estimating overdispersion in sparse multinomial data.

    abstract::Multinomial data arise in many areas of the life sciences, such as mark-recapture studies and phylogenetics, and will often by overdispersed, with the variance being higher than predicted by a multinomial model. The quasi-likelihood approach to modeling this overdispersion involves the assumption that the variance is ...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/biom.13194

    authors: Afroz F,Parry M,Fletcher D

    更新日期:2020-09-01 00:00:00

  • A Bayesian approach to dose-response assessment and synergy and its application to in vitro dose-response studies.

    abstract::In this article, we propose a Bayesian approach to dose-response assessment and the assessment of synergy between two combined agents. We consider the case of an in vitro ovarian cancer research study aimed at investigating the antiproliferative activities of four agents, alone and paired, in two human ovarian cancer ...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.1541-0420.2010.01403.x

    authors: Hennessey VG,Rosner GL,Bast RC Jr,Chen MY

    更新日期:2010-12-01 00:00:00

  • Some scale estimators and lack-of-fit tests for the censored two-sample accelerated life model.

    abstract::Some new scale estimators for the censored two-sample accelerated life model are introduced. They are zeros of some integrated weighted difference between the two cumulative hazard estimators. These estimators are asymptotically normal. The weight is chosen to result in estimators whose asymptotic variances do not inv...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:

    authors: Yang S

    更新日期:1998-09-01 00:00:00

  • Connecting the latent multinomial.

    abstract::Link et al. (2010, Biometrics 66, 178-185) define a general framework for analyzing capture-recapture data with potential misidentifications. In this framework, the observed vector of counts, y, is considered as a linear function of a vector of latent counts, x, such that y=Ax, with x assumed to follow a multinomial d...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/biom.12333

    authors: Schofield MR,Bonner SJ

    更新日期:2015-12-01 00:00:00

  • Optimally weighted L(2) distance for functional data.

    abstract::Many techniques of functional data analysis require choosing a measure of distance between functions, with the most common choice being L2 distance. In this article we show that using a weighted L2 distance, with a judiciously chosen weight function, can improve the performance of various statistical methods for funct...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/biom.12161

    authors: Chen H,Reiss PT,Tarpey T

    更新日期:2014-09-01 00:00:00

  • A new criterion for confounder selection.

    abstract::We propose a new criterion for confounder selection when the underlying causal structure is unknown and only limited knowledge is available. We assume all covariates being considered are pretreatment variables and that for each covariate it is known (i) whether the covariate is a cause of treatment, and (ii) whether t...

    journal_title:Biometrics

    pub_type: 杂志文章

    doi:10.1111/j.1541-0420.2011.01619.x

    authors: VanderWeele TJ,Shpitser I

    更新日期:2011-12-01 00:00:00