IDEAS home Printed from https://ideas.repec.org/a/bpj/ijbist/v7y2011i1n27.html
   My bibliography  Save this article

Relative Risk Estimation in Cluster Randomized Trials: A Comparison of Generalized Estimating Equation Methods

Author

Listed:
  • Yelland Lisa N
  • Salter Amy B
  • Ryan Philip

Abstract

Relative risks have become a popular measure of treatment effect for binary outcomes in randomized controlled trials (RCTs). Relative risks can be estimated directly using log binomial regression but the model may fail to converge. Alternative methods are available for estimating relative risks but these have generally only been evaluated for independent data. As some of these methods are now being applied in cluster RCTs, investigation of their performance in this context is needed. We compare log binomial regression and three alternative methods (expanded logistic regression, log Poisson regression and log normal regression) for estimating relative risks in cluster RCTs. Clustering is taken into account using generalized estimating equations (GEEs) with an independence or exchangeable working correlation structure. The results of our large simulation study show that the log binomial GEE generally performs well for clustered data but suffers from convergence problems, as expected. Both the log Poisson GEE and log normal GEE have advantages in certain settings in terms of type I error, bias and coverage. The expanded logistic GEE can perform poorly and is sensitive to the chosen working correlation structure. Conclusions about the effectiveness of treatment often differ depending on the method used, highlighting the need to pre-specify an analysis approach. We recommend pre-specifying that either the log Poisson GEE or log normal GEE will be used in the event that the log binomial GEE fails to converge.

Suggested Citation

  • Yelland Lisa N & Salter Amy B & Ryan Philip, 2011. "Relative Risk Estimation in Cluster Randomized Trials: A Comparison of Generalized Estimating Equation Methods," The International Journal of Biostatistics, De Gruyter, vol. 7(1), pages 1-26, May.
    • Handle: RePEc:bpj:ijbist:v:7:y:2011:i:1:n:27
    DOI: 10.2202/1557-4679.1323
    as

    Download full text from publisher

    File URL: https://doi.org/10.2202/1557-4679.1323
    Download Restriction: For access to full text, subscription to the journal or payment for the individual article is required.
    File URL: https://libkey.io/10.2202/1557-4679.1323?utm_source=ideas
    LibKey link: if access is restricted and if your library uses this service, LibKey will redirect you to where you can use your library subscription to access this item
    ---><---

    As the access to this document is restricted, you may want to search for a different version of it.

    References listed on IDEAS

    as
    1. Lloyd A. Mancl & Timothy A. DeRouen, 2001. "A Covariance Estimator for GEE with Improved Small‐Sample Properties," Biometrics, The International Biometric Society, vol. 57(1), pages 126-134, March.
    2. Bing Lu & John S. Preisser & Bahjat F. Qaqish & Chirayath Suchindran & Shrikant I. Bangdiwala & Mark Wolfson, 2007. "A Comparison of Two Bias-Corrected Covariance Estimators for Generalized Estimating Equations," Biometrics, The International Biometric Society, vol. 63(3), pages 935-941, September.
    3. Hammill, Bradley G. & Preisser, John S., 2006. "A SAS/IML software program for GEE and regression diagnostics," Computational Statistics & Data Analysis, Elsevier, vol. 51(2), pages 1197-1212, November.
    4. Yelland Lisa N & Salter Amy B & Ryan Philip, 2011. "Relative Risk Estimation in Randomized Controlled Trials: A Comparison of Methods for Independent Observations," The International Journal of Biostatistics, De Gruyter, vol. 7(1), pages 1-31, January.
    Full references (including those not matched with items on IDEAS)

    Citations

    Citations are extracted by the CitEc Project, subscribe to its RSS feed for this item.
    as


    Cited by:

    1. Francis L. Huang, 2022. "Analyzing Cross-Sectionally Clustered Data Using Generalized Estimating Equations," Journal of Educational and Behavioral Statistics, , vol. 47(1), pages 101-125, February.

    Most related items

    These are the items that most often cite the same works as this one and are cited by the same works as this one.
    1. Steven Teerenstra & Bing Lu & John S. Preisser & Theo van Achterberg & George F. Borm, 2010. "Sample Size Considerations for GEE Analyses of Three-Level Cluster Randomized Trials," Biometrics, The International Biometric Society, vol. 66(4), pages 1230-1237, December.
    2. Jamie Perin & John S. Preisser, 2017. "Alternating logistic regressions with improved finite sample properties," Biometrics, The International Biometric Society, vol. 73(2), pages 696-705, June.
    3. Tyler J. VanderWeele, 2020. "Optimal approximate conversions of odds ratios and hazard ratios to risk ratios," Biometrics, The International Biometric Society, vol. 76(3), pages 746-752, September.
    4. Vens, Maren & Ziegler, Andreas, 2012. "Generalized estimating equations and regression diagnostics for longitudinal controlled clinical trials: A case study," Computational Statistics & Data Analysis, Elsevier, vol. 56(5), pages 1232-1242.
    5. Ji-Hyun Lee & Michael J Schell & Richard Roetzheim, 2009. "Analysis of Group Randomized Trials with Multiple Binary Endpoints and Small Number of Groups," PLOS ONE, Public Library of Science, vol. 4(10), pages 1-9, October.
    6. A. Colin Cameron & Jonah B. Gelbach & Douglas L. Miller, 2008. "Bootstrap-Based Improvements for Inference with Clustered Errors," The Review of Economics and Statistics, MIT Press, vol. 90(3), pages 414-427, August.
    7. Westgate, Philip M., 2013. "A bias-corrected covariance estimator for improved inference when using an unstructured correlation with quadratic inference functions," Statistics & Probability Letters, Elsevier, vol. 83(6), pages 1553-1558.
    8. You-Gan Wang & Yuning Zhao, 2007. "A Modified Pseudolikelihood Approach for Analysis of Longitudinal Data," Biometrics, The International Biometric Society, vol. 63(3), pages 681-689, September.
    9. Haiyan Wang & Michael Akritas, 2010. "Inference from heteroscedastic functional data," Journal of Nonparametric Statistics, Taylor & Francis Journals, vol. 22(2), pages 149-168.
    10. Paniagua, Victoria, 2022. "When clients vote for brokers: How elections improve public goods provision in urban slums," World Development, Elsevier, vol. 158(C).
    11. Galea, Manuel & de Castro, Mário, 2017. "Robust inference in a linear functional model with replications using the t distribution," Journal of Multivariate Analysis, Elsevier, vol. 160(C), pages 134-145.
    12. Michael Eichinger & Tatiana Görig & Sabine Georg & Dorle Hoffmann & Diana Sonntag & Heike Philippi & Jochem König & Michael S. Urschitz & Freia De Bock, 2022. "Evaluation of a Complex Intervention to Strengthen Participation-Centred Care for Children with Special Healthcare Needs: Protocol of the Stepped Wedge Cluster Randomised PART-CHILD Trial," IJERPH, MDPI, vol. 19(24), pages 1-18, December.
    13. Fan, Chunpeng & Zhang, Donghui, 2014. "Wald-type rank tests: A GEE approach," Computational Statistics & Data Analysis, Elsevier, vol. 74(C), pages 1-16.
    14. Francis L. Huang, 2022. "Analyzing Cross-Sectionally Clustered Data Using Generalized Estimating Equations," Journal of Educational and Behavioral Statistics, , vol. 47(1), pages 101-125, February.
    15. Masahiko Gosho & Hisashi Noma & Kazushi Maruo, 2021. "Practical Review and Comparison of Modified Covariance Estimators for Linear Mixed Models in Small‐sample Longitudinal Studies with Missing Data," International Statistical Review, International Statistical Institute, vol. 89(3), pages 550-572, December.
    16. Michael P. Fay & Barry I. Graubard, 2001. "Small-Sample Adjustments for Wald-Type Tests Using Sandwich Estimators," Biometrics, The International Biometric Society, vol. 57(4), pages 1198-1206, December.
    17. Saskia Litière & Ariel Alonso & Geert Molenberghs, 2007. "Type I and Type II Error Under Random-Effects Misspecification in Generalized Linear Mixed Models," Biometrics, The International Biometric Society, vol. 63(4), pages 1038-1044, December.
    18. Diana Miglioretti & Patrick Heagerty, 2004. "Marginal Modeling of Multilevel Binary Data with Time-Varying Covariates," UW Biostatistics Working Paper Series 1050, Berkeley Electronic Press.
    19. Cheng, Guang & Yu, Zhuqing & Huang, Jianhua Z., 2013. "The cluster bootstrap consistency in generalized estimating equations," Journal of Multivariate Analysis, Elsevier, vol. 115(C), pages 33-47.
    20. Bing Lu & John S. Preisser & Bahjat F. Qaqish & Chirayath Suchindran & Shrikant I. Bangdiwala & Mark Wolfson, 2007. "A Comparison of Two Bias-Corrected Covariance Estimators for Generalized Estimating Equations," Biometrics, The International Biometric Society, vol. 63(3), pages 935-941, September.

    Corrections

    All material on this site has been provided by the respective publishers and authors. You can help correct errors and omissions. When requesting a correction, please mention this item's handle: RePEc:bpj:ijbist:v:7:y:2011:i:1:n:27. See general information about how to correct material in RePEc.

    If you have authored this item and are not yet registered with RePEc, we encourage you to do it here. This allows to link your profile to this item. It also allows you to accept potential citations to this item that we are uncertain about.

    If CitEc recognized a bibliographic reference but did not link an item in RePEc to it, you can help with this form .

    If you know of missing items citing this one, you can help us creating those links by adding the relevant references in the same way as above, for each refering item. If you are a registered author of this item, you may also want to check the "citations" tab in your RePEc Author Service profile, as there may be some citations waiting for confirmation.

    For technical questions regarding this item, or to correct its authors, title, abstract, bibliographic or download information, contact: Peter Golla (email available below). General contact details of provider: https://www.degruyter.com .

    Please note that corrections may take a couple of weeks to filter through the various RePEc services.

    IDEAS is a RePEc service. RePEc uses bibliographic data supplied by the respective publishers.