Browse
Submit Data
Databases
API
Help

Dataset Information

28 Views

0 Connections

0 Citations

0 Reanalyses

0 Downloads

Omics score: 0

Power and Sample Size Calculations for Generalized Estimating Equations via Local Asymptotics.

ABSTRACT: We consider the problem of calculating power and sample size for tests based on generalized estimating equations (GEE), that arise in studies involving clustered or correlated data (e.g., longitudinal studies and sibling studies). Previous approaches approximate the power of such tests using the asymptotic behavior of the test statistics under fixed alternatives. We develop a more accurate approach in which the asymptotic behavior is studied under a sequence of local alternatives that converge to the null hypothesis at root-m rate, where m is the number of clusters. Based on this approach, explicit sample size formulae are derived for Wald and quasi-score test statistics in a variety of GEE settings. Simulation results show that in the important special case of logistic regression with exchangeable correlation structure, previous approaches can inflate the projected sample size (to obtain nominal 90% power using the Wald statistic) by over 10%, whereas the proposed approach provides an accuracy of around 2%.

SUBMITTER: Li Z

PROVIDER: S-EPMC3903421 | biostudies-literature | 2013 Jan

REPOSITORIES: biostudies-literature

ACCESS DATA

Json Xml

Publications

Power and Sample Size Calculations for Generalized Estimating Equations via Local Asymptotics.

Li Zhigang Z McKeague Ian W IW

Statistica Sinica 20130101 1

We consider the problem of calculating power and sample size for tests based on generalized estimating equations (GEE), that arise in studies involving clustered or correlated data (e.g., longitudinal studies and sibling studies). Previous approaches approximate the power of such tests using the asymptotic behavior of the test statistics under <i>fixed</i> alternatives. We develop a more accurate approach in which the asymptotic behavior is studied under a sequence of <i>local</i> alternatives t ...[more]

PMID: 24478568

Similar Datasets

Improving power in small-sample longitudinal studies when using generalized estimating equations.

Project description:Generalized estimating equations (GEE) are often used for the marginal analysis of longitudinal data. Although much work has been performed to improve the validity of GEE for the analysis of data arising from small-sample studies, little attention has been given to power in such settings. Therefore, we propose a valid GEE approach to improve power in small-sample longitudinal study settings in which the temporal spacing of outcomes is the same for each subject. Specifically, we use a modified empirical sandwich covariance matrix estimator within correlation structure selection criteria and test statistics. Use of this estimator can improve the accuracy of selection criteria and increase the degrees of freedom to be used for inference. The resulting impacts on power are demonstrated via a simulation study and application example. Copyright © 2016 John Wiley & Sons, Ltd.

| S-EPMC4965318 | biostudies-literature

Sample size and power calculations based on generalized linear mixed models with correlated binary outcomes.

Project description:The generalized linear mixed model (GLIMMIX) provides a powerful technique to model correlated outcomes with different types of distributions. The model can now be easily implemented with SAS PROC GLIMMIX in version 9.1. For binary outcomes, linearization methods of penalized quasi-likelihood (PQL) or marginal quasi-likelihood (MQL) provide relatively accurate variance estimates for fixed effects. Using GLIMMIX based on these linearization methods, we derived formulas for power and sample size calculations for longitudinal designs with attrition over time. We found that the power and sample size estimates depend on the within-subject correlation and the size of random effects. In this article, we present tables of minimum sample sizes commonly used to test hypotheses for longitudinal studies. A simulation study was used to compare the results. We also provide a Web link to the SAS macro that we developed to compute power and sample sizes for correlated binary outcomes.

| S-EPMC3737998 | biostudies-literature

Sample Size and Power Calculations for Additive Interactions.

Project description: Not available

| S-EPMC4249707 | biostudies-literature

Effect Size Guidelines, Sample Size Calculations, and Statistical Power in Gerontology.

Project description:Background and Objectives:Researchers typically use Cohen's guidelines of Pearson's r = .10, .30, and .50, and Cohen's d = 0.20, 0.50, and 0.80 to interpret observed effect sizes as small, medium, or large, respectively. However, these guidelines were not based on quantitative estimates and are only recommended if field-specific estimates are unknown. This study investigated the distribution of effect sizes in both individual differences research and group differences research in gerontology to provide estimates of effect sizes in the field. Research Design and Methods:Effect sizes (Pearson's r, Cohen's d, and Hedges' g) were extracted from meta-analyses published in 10 top-ranked gerontology journals. The 25th, 50th, and 75th percentile ranks were calculated for Pearson's r (individual differences) and Cohen's d or Hedges' g (group differences) values as indicators of small, medium, and large effects. A priori power analyses were conducted for sample size calculations given the observed effect size estimates. Results:Effect sizes of Pearson's r = .12, .20, and .32 for individual differences research and Hedges' g = 0.16, 0.38, and 0.76 for group differences research were interpreted as small, medium, and large effects in gerontology. Discussion and Implications:Cohen's guidelines appear to overestimate effect sizes in gerontology. Researchers are encouraged to use Pearson's r = .10, .20, and .30, and Cohen's d or Hedges' g = 0.15, 0.40, and 0.75 to interpret small, medium, and large effects in gerontology, and recruit larger samples.

| S-EPMC6736231 | biostudies-literature

Power considerations for generalized estimating equations analyses of four-level cluster randomized trials.

Project description:In this article, we develop methods for sample size and power calculations in four-level intervention studies when intervention assignment is carried out at any level, with a particular focus on cluster randomized trials (CRTs). CRTs involving four levels are becoming popular in healthcare research, where the effects are measured, for example, from evaluations (level 1) within participants (level 2) in divisions (level 3) that are nested in clusters (level 4). In such multilevel CRTs, we consider three types of intraclass correlations between different evaluations to account for such clustering: that of the same participant, that of different participants from the same division, and that of different participants from different divisions in the same cluster. Assuming arbitrary link and variance functions, with the proposed correlation structure as the true correlation structure, closed-form sample size formulas for randomization carried out at any level (including individually randomized trials within a four-level clustered structure) are derived based on the generalized estimating equations approach using the model-based variance and using the sandwich variance with an independence working correlation matrix. We demonstrate that empirical power corresponds well with that predicted by the proposed method for as few as eight clusters, when data are analyzed using the matrix-adjusted estimating equations for the correlation parameters with a bias-corrected sandwich variance estimator, under both balanced and unbalanced designs.

| S-EPMC9574475 | biostudies-literature

Power calculation for analyses of cross-sectional stepped-wedge cluster randomized trials with binary outcomes via generalized estimating equations.

Project description:Power calculation for stepped-wedge cluster randomized trials (SW-CRTs) presents unique challenges, beyond those of standard parallel cluster randomized trials, due to the need to consider temporal within cluster correlations and background period effects. To date, power calculation methods specific to SW-CRTs have primarily been developed under a linear model. When the outcome is binary, the use of a linear model corresponds to assessing a prevalence difference; yet trial analysis often employs a nonlinear link function. We propose power calculation methods for cross-sectional SW-CRTs under a logistic model fitted by generalized estimating equations. Firstly, under an exchangeable correlation structure, we show the power based on a logistic model is lower than that from assuming a linear model in the absence of period effects. We then evaluate the impact of background prevalence changes over time on power. To allow the correlation among outcomes in the same cluster to change over time and with treatment status, we generalize the methods to more complex correlation structures. Our simulation studies demonstrate that the proposed power calculation methods perform well with the model-based variance under the true correlation structure and reveal that a working independence structure can result in substantial efficiency loss, while a working exchangeable structure performs well even when the underlying correlation structure deviates from exchangeable. An extension to our methods accounts for variable cluster sizes and reveals that unequal cluster sizes have a modest impact on power. We illustrate the approaches by application to a quality of care improvement trial for acute coronary syndrome.

| S-EPMC8900518 | biostudies-literature

PGS: penalized generalized estimating equations with grid search for epigenome-wide multiple measurement study

Project description:Motivation: The need of identifying genetic determinants that disclose the heritability of complex traits as well as pinpoint causative genetic effects in some complex diseases has propelled large-scale, systematic fashion bred epigenome-wide studies. With the very dynamic nature of epigenome and much reduced cost in microarray and sequencing experiments, more and more researchers are now studying epigenomic at multiple time points, i.e. multiple measurement and/or longitudinal study design. However, the popular site-by-site multiple testing as commonly used in genome-wide studies may impair the value of multiple measurements because it leads to underpowered analyses by ignoring the inherent dependent structure, which has called for new methods to address the statistical challenges. Results: We have proposed a penalized regression model incorporating with grid search method (PGS), for analyzing epigenome-wide multiple measurement data. The development of this analytical framework was motivated by a real-world micro RNA dataset. Comparisons between PGS and the site-by-site testing approach reveal that PGS provides smaller phenotype prediction errors and higher enrichment of phenotype-related biological pathway than the site-by-site testing approach. Our approach is also useful for epigenome-wide data of DNA methylation and histone modifications.

2017-12-21 | GSE55950 | GEO

Sample size and power calculations for open cohort longitudinal cluster randomized trials.

Project description:When calculating sample size or power for stepped wedge or other types of longitudinal cluster randomized trials, it is critical that the planned sampling structure be accurately specified. One common assumption is that participants will provide measurements in each trial period, that is, a closed cohort, and another is that each participant provides only one measurement during the course of the trial. However some studies have an "open cohort" sampling structure, where participants may provide measurements in variable numbers of periods. To date, sample size calculations for longitudinal cluster randomized trials have not accommodated open cohorts. Feldman and McKinlay (1994) provided some guidance, stating that the participant-level autocorrelation could be varied to account for the degree of overlap in different periods of the study, but did not indicate precisely how to do so. We present sample size and power formulas that allow for open cohorts and discuss the impact of the degree of "openness" on sample size and power. We consider designs where the number of participants in each cluster will be maintained throughout the trial, but individual participants may provide differing numbers of measurements. Our results are a unification of closed cohort and repeated cross-sectional sample results of Hooper et al (2016), and indicate precisely how participant autocorrelation of Feldman and McKinlay should be varied to account for an open cohort sampling structure. We discuss different types of open cohort sampling schemes and how open cohort sampling structure impacts on power in the presence of decaying within-cluster correlations and autoregressive participant-level errors.

| S-EPMC7217159 | biostudies-literature

TENSOR GENERALIZED ESTIMATING EQUATIONS FOR LONGITUDINAL IMAGING ANALYSIS.

Project description:Longitudinal neuroimaging studies are becoming increasingly prevalent, where brain images are collected on multiple subjects at multiple time points. Analyses of such data are scientifically important, but also challenging. Brain images are in the form of multidimensional arrays, or tensors, which are characterized by both ultrahigh dimensionality and a complex structure. Longitudinally repeated images and induced temporal correlations add a further layer of complexity. Despite some recent efforts, there exist very few solutions for longitudinal imaging analyses. In response to the increasing need to analyze longitudinal imaging data, we propose several tensor generalized estimating equations (GEEs). The proposed GEE approach accounts for intra-subject correlation, and an imposed low-rank structure on the coefficient tensor effectively reduces the dimensionality. We also propose a scalable estimation algorithm, establish the asymptotic properties of the solution to the tensor GEEs, and investigate sparsity regularization for the purpose of region selection. We demonstrate the proposed method using simulations and by analyzing a real data set from the Alzheimer's Disease Neuroimaging Initiative.

| S-EPMC7286574 | biostudies-literature

Power/sample size calculations for assessing correlates of risk in clinical efficacy trials.

Project description:In a randomized controlled clinical trial that assesses treatment efficacy, a common objective is to assess the association of a measured biomarker response endpoint with the primary study endpoint in the active treatment group, using a case-cohort, case-control, or two-phase sampling design. Methods for power and sample size calculations for such biomarker association analyses typically do not account for the level of treatment efficacy, precluding interpretation of the biomarker association results in terms of biomarker effect modification of treatment efficacy, with detriment that the power calculations may tacitly and inadvertently assume that the treatment harms some study participants. We develop power and sample size methods accounting for this issue, and the methods also account for inter-individual variability of the biomarker that is not biologically relevant (e.g., due to technical measurement error). We focus on a binary study endpoint and on a biomarker subject to measurement error that is normally distributed or categorical with two or three levels. We illustrate the methods with preventive HIV vaccine efficacy trials and include an R package implementing the methods. Copyright © 2016 John Wiley & Sons, Ltd.

| S-EPMC4965346 | biostudies-literature

OmicsDI is part of the ELIXIR infrastructure

OmicsDI is an Elixir interoperability service. Learn more ›

Tweets

OmicsDI Databases

PRIDE
PeptideAtlas
MassIVE
JPOST Repository
Physiome Model Repository

EGA
EVA
ENA
LINCS
PAXDB
Cell Collective

MetaboLights
Metabolomics Workbench
MetabolomeExpress
GNPS
BioModels
FAIRDOMHub

ArrayExpress
dbGaP
ExpressionAtlas
GEO
NODE

Information

Databases
Help
API
Contact us
Code on GitHub
Terms of use
Submit Data