recommendations for the analysis of individually randomised controlled trials with clustering in one arm a case of continuous outcomes

Flight et al BMC Medical Research Methodology (2016) 16:165 DOI 10.1186/s12874-016-0249-5 RESEARCH ARTICLE Open Access Recommendations for the analysis of individually randomised controlled trials with clustering in one arm – a case of continuous outcomes Laura Flight1 , Annabel Allison2 , Munyaradzi Dimairo1 , Ellen Lee1 , Laura Mandefield1 and Stephen J Walters1* Abstract Background: In an individually randomised controlled trial where the treatment is delivered by a health professional it seems likely that the effectiveness of the treatment, independent of any treatment effect, could depend on the skill, training or even enthusiasm of the health professional delivering it This may then lead to a potential clustering of the outcomes for patients treated by the same health professional, but similar clustering may not occur in the control arm Using four case studies, we aim to provide practical guidance and recommendations for the analysis of trials with some element of clustering in one arm Methods: Five approaches to the analysis of outcomes from an individually randomised controlled trial with clustering in one arm are identified in the literature Some of these methods are applied to four case studies of completed randomised controlled trials with clustering in one arm with sample sizes ranging from 56 to 539 Results are obtained using the statistical packages R and Stata and summarised using a forest plot Results: The intra-cluster correlation coefficient (ICC) for each of the case studies was small (