Abstract
In the examples of the first chapters, the outcome variable of the RCT was continuous. In this chapter, the analysis of data from RCTs with a dichotomous outcome is discussed. In general, the same principles can be used for RCTs with a dichotomous outcome than for RCTs with a continuous outcome. The difference is that for dichotomous outcomes, logistic models must be used instead of linear models. However, when there is more than one follow-up measurement, instead of a logistic mixed model analysis, it is advised to use logistic generalized estimating equations to deal with the dependency of the observations and to estimate an appropriate treatment effect. It is also argued that an adjustment for the baseline value is often not necessary, because at baseline mostly all subjects have the same value (e.g., they all have a certain disease). Finally, the problem of non-collapsibility is discussed.
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References
Albert, P. S. (1999). Longitudinal data analysis (repeated measures) in clinical trials. Statistics in Medicine, 18, 1707–1732.
Apeldoorn, A. T., Ostelo, R. W., van Helvoirt, H., Fritz, J. M., Knol, D. L., van Tulder, M., & de Vet, H. (2012). A randomized controlled trial on the effectiveness of a classification-based system for subacute and chronic low back pain. Spine, 37, 1347–1356.
Bellamy, S. L., Gibberd, R., Hancock, L., Howley, P., Kennedy, B., Klar, N., Lipsitz, S., & Ryan, L. (2009). Analysis of dichotomous outcome data for community intervention studies. Statistical Methods in Medical Research, 9, 135.
Goldstein, H. (2003). Multilevel statistical models (3nd ed.). Edward Arnold.
Greenland, S., & Robins, J. M. (2009). Identifiability, exchangeability and confounding revisited. Epidemiologic Perspectives & Innovations, 6, 4.
Heo, M., & Leon, A. C. (2005). Comparison of statistical methods for analysis of clustered binary outcomes. Statistics in Medicine, 24, 911–923.
Hernan, M. A., Clayton, D., & Keiding, N. (2011). The Simpson’s paradox unravelled. International Journal of Epidemiology, 40, 780–785.
Hu, F. B., Goldberg, J., Hedeker, D., Flay, B. R., & Pentz, M. (1998). Comparison of population-averaged and subject-specific approaches for analyzing repeated binary outcomes. American Journal of Epidemiology, 147, 694–703.
Hubbard, A. E., Ahern, J., Fleischer, N. L., van der Laan, M., Lippman, S. A., Jewell, N., Bruckner, T., & Satariano, W. A. (2010). To GEE or not to GEE. Comparing population average and mixed models for estimating the associations between neighborhood risk factors and health. Epidemiology, 21, 467–474.
Kim, H.-Y., Preisser, J. S., Rozier, R. G., & Valiyaparambi, J. V. (2006). Multilevel analysis of group randomized trials with binary data. Community Dentistry and Oral Epidemiology, 34, 241–251.
Laird, N. M., & Ware, J. H. (1982). Random effects models for longitudinal data. Biometrics, 38, 963–974.
Lesaffre, E., & Spiessens, B. (2001). On the effect of the number of quadrature points in a logistic random-effects model: An example. Applied Statistics, 50, 325–335.
Liang, K., & Zeger, S. L. (1986). Longitudinal data analysis using generalized linear models. Biometrika, 73, 45–51.
Little, R. J. A. (1995). Modelling the drop-out mechanism repeated measures studies. Journal of the American Statistical Association, 90, 1112–1121.
Liu, Q., & Pierce, D. A. (1994). A note on Gauss–Hermite quadrature. Biometrika, 81, 624–629.
Newman, S. C. (2004). Commonalities in the classical, collapsibility and counterfactual concepts in confounding. Journal of Clinical Epidemiology, 57, 325–329.
Omar, R. Z., Wright, E. M., Turner, R. M., & Thompson, S. G. (1999). Analysing repeated measurements data: A practical comparison of methods. Statistics in Medicine, 18, 1587–1603.
Rabe-Hesketh, S., & Skrondal, A. (2001). Parameterisation of multivariate random effects models for categorical data. Biometrics, 57, 1256–1264.
Subramanian, S. V. (2004). The relevance of multilevel statistical methods for identifying causal neighbourhood effects. Social Science & Medicine, 58, 1961–1967.
ten Have, T. R., Ratcliffe, S. J., Reboussin, B. A., & Miller, M. E. (2004). Deviations from the population-average cluster-specific relationship for clustered binary data. Statistical Methods in Medical Research, 13, 3–16.
Twisk, J. W. R. (2013). Applied longitudinal data analysis for epidemiology (2nd ed.). Cambridge University Press.
Twisk, J. W. R., de Vente, W., Apeldoorn, A. T., & de Boer, M. (2017). Should we use logistic mixed model analysis for the effect estimation in a longitudinal RCT with a dichotomous outcome variable? Epidemiology, Biostatistics and Public Health, 14(3), 1–8.
Warmerdam, L., van Straten, A., Twisk, J., Riper, H., & Cuijpers, P. (2008). Internet-based treatment for adults with depressive symptoms: Randomized controlled trial. Journal of Medical Internet Research, 10, e44.
Zeger, S. L., & Liang, K.-Y. (1986). Longitudinal data analysis for discrete and continuous outcomes. Biometrics, 42, 121–130.
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Twisk, J.W.R. (2021). Dichotomous Outcomes. In: Analysis of Data from Randomized Controlled Trials. Springer, Cham. https://doi.org/10.1007/978-3-030-81865-4_8
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