On logistic regression analysis of dichotomized responses

We study the properties of treatment effect estimate in terms of odds ratio at the study end point from logistic regression model adjusting for the baseline value when the underlying continuous repeated measurements follow a multivariate normal distribution. Compared with the analysis that does not adjust for the baseline value, the adjusted analysis produces a larger treatment effect as well as a larger standard error. However, the increase in standard error is more than offset by the increase in treatment effect so that the adjusted analysis is more powerful than the unadjusted analysis for detecting the treatment effect. On the other hand, the true adjusted odds ratio implied by the normal distribution of the underlying continuous variable is a function of the baseline value and hence is unlikely to be able to be adequately represented by a single value of adjusted odds ratio from the logistic regression model. In contrast, the risk difference function derived from the logistic regression model provides a reasonable approximation to the true risk difference function implied by the normal distribution of the underlying continuous variable over the range of the baseline distribution. We show that different metrics of treatment effect have similar statistical power when evaluated at the baseline mean. Copyright © 2016 John Wiley & Sons, Ltd.