Multi-Arm Randomized Control Trials in Inflammatory Bowel Disease: A Literature Review and an Illustration of Methods for Analysis
Abstract
This thesis aimed to review the literature on multiple-arm randomized control trials in inflammatory bowel disease (IBD) and to illustrate how to analyze these trials, focusing on appropriately controlling the type 1 error rates. The literature review found 247 trials published from the inception of each database to April 2014, of which 122 (49%) trials were multiple-arm trials and of those, 59 (48%) trials were on ulcerative colitis and 63 (52%) on Crohn’s disease. A published assessment tool was adopted to assess whether controlling of Type I error rates was needed. Despite the common use of this trial design and the need for multiple comparison procedures (MCPs) based on the assessment tool, only 20% of trials applied any MCPs. Failing to apply appropriate procedures may have inflated the Type I error rates, yielding false positive results. Data from a multiple-arm trial evaluating the efficacy of MLN02 (vedolizumab) was used as a case study to present how to implement common MCPs. Assessment tools regarding MCPs are helpful to have readily available to improve the scientific rigour of such research. Future research into the utility of online resources that guide multiple-arm trial design and analysis within IBD could promote such tools.