Evidence map›Paper›PMID 34596912›Full record

ArticleBiometrical journal. Biometrische Zeitschrift2022

Impact of unequal cluster sizes for GEE analyses of stepped wedge cluster randomized trials with binary outcomes.

Zibo Tian, John S Preisser, Denise Esserman, Elizabeth L Turner, Paul J Rathouz, Fan Li

Abstract read
In one paragraph

Article in Biometrical journal. Biometrische Zeitschrift, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.

0numbers the graph read from it
0cells of the map it votes in
17citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

17 citing papers in PubMed.

  1. Model-robust standardization in stepped wedge cluster randomized trials.Journal of the Royal Statistical Society. Series A, (Statistics in Society) · 2026
    Article
  2. Article
  3. Article
  4. Article
  5. Article
  6. Article
  7. Article
  8. Article
  9. Article
  10. Generalizing the information content for stepped wedge designs: A marginal modeling approach.Scandinavian journal of statistics, theory and applications · 2023
    Article
  11. Article
  12. Article
  13. Review
  14. Article
  15. Review
  16. Article
  17. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

6 authors.

Zibo TianDepartment of Biostatistics, Yale University School of Public Health, New Haven, CT, USA.
John S PreisserDepartment of Biostatistics, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.ORCID 0000-0002-7869-2057
Denise EssermanDepartment of Biostatistics, Yale University School of Public Health, New Haven, CT, USA.
Elizabeth L TurnerDepartment of Biostatistics and Bioinformatics, Duke University, Durham, NC, USA.
Paul J RathouzDepartment of Population Health, The University of Texas at Austin, Austin, TX, USA.
Fan LiDepartment of Biostatistics, Yale University School of Public Health, New Haven, CT, USA.ORCID 0000-0001-6183-1893

Funding

Yale Clinical and Translational Science Award (U Component)UL1TR001863 · NCATS · YALE UNIVERSITY · PI John H. Krystal, LUCILA OHNO-MACHADO · 2016 to 2026
$102.9M
NCATS NIH HHS UL1 TR0001863NCATS NIH HHS UL1 TR001863
6 · The paper itself

Abstract

The stepped wedge (SW) design is a type of unidirectional crossover design where cluster units switch from control to intervention condition at different prespecified time points. While a convention in study planning is to assume the cluster-period sizes are identical, SW cluster randomized trials (SW-CRTs) involving repeated cross-sectional designs frequently have unequal cluster-period sizes, which can impact the efficiency of the treatment effect estimator. In this paper, we provide a comprehensive investigation of the efficiency impact of unequal cluster sizes for generalized estimating equation analyses of SW-CRTs, with a focus on binary outcomes as in the Washington State Expedited Partner Therapy trial. Several major distinctions between our work and existing work include the following: (i) we consider multilevel correlation structures in marginal models with binary outcomes; (ii) we study the implications of both the between-cluster and within-cluster imbalances in sizes; and (iii) we provide a comparison between the independence working correlation versus the true working correlation and detail the consequences of ignoring correlation estimation in SW-CRTs with unequal cluster sizes. We conclude that the working independence assumption can lead to substantial efficiency loss and a large sample size regardless of cluster-period size variability in SW-CRTs, and recommend accounting for correlations in the analysis. To improve study planning, we additionally provide a computationally efficient search algorithm to estimate the sample size in SW-CRTs accounting for unequal cluster-period sizes, and conclude by illustrating the proposed approach in the context of the Washington State study.

Indexed as

Research DesignCluster AnalysisCross-Sectional StudiesRandomized Controlled Trials as TopicSample Sizecoefficient of variationgeneralized estimating equationsintraclass correlation coefficientsrelative efficiencystepped wedge designsvariable cluster sizes

Identifiers

PMID34596912
PMCPMC9292617

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.