|Title||Comparison of adaptive treatment strategies based on longitudinal outcomes in sequential multiple assignment randomized trials.|
|Publication Type||Journal Article|
|Year of Publication||2017|
|Date Published||2017 Feb 10|
|Keywords||Antipsychotic Agents, Data Interpretation, Statistical, Humans, Linear Models, Longitudinal Studies, Models, Statistical, Randomized Controlled Trials as Topic, Regression Analysis, Schizophrenia, Statistics as Topic, Treatment Outcome|
In sequential multiple assignment randomized trials, longitudinal outcomes may be the most important outcomes of interest because this type of trials is usually conducted in areas of chronic diseases or conditions. We propose to use a weighted generalized estimating equation (GEE) approach to analyzing data from such type of trials for comparing two adaptive treatment strategies based on generalized linear models. Although the randomization probabilities are known, we consider estimated weights in which the randomization probabilities are replaced by their empirical estimates and prove that the resulting weighted GEE estimator is more efficient than the estimators with true weights. The variance of the weighted GEE estimator is estimated by an empirical sandwich estimator. The time variable in the model can be linear, piecewise linear, or more complicated forms. This provides more flexibility that is important because, in the adaptive treatment setting, the treatment changes over time and, hence, a single linear trend over the whole period of study may not be practical. Simulation results show that the weighted GEE estimators of regression coefficients are consistent regardless of the specification of the correlation structure of the longitudinal outcomes. The weighted GEE method is then applied in analyzing data from the Clinical Antipsychotic Trials of Intervention Effectiveness. Copyright © 2016 John Wiley & Sons, Ltd.
|Alternate Journal||Stat Med|
|Original Publication||Comparison of adaptive treatment strategies based on longitudinal outcomes in sequential multiple assignment randomized trials.|
|PubMed Central ID||PMC5209271|
|Grant List||P01 CA142538 / CA / NCI NIH HHS / United States|
Comparison of adaptive treatment strategies based on longitudinal outcomes in sequential multiple assignment randomized trials.