This thesis explores the comparative efficacy and safety of a novel therapy, referred to as Treatment X, in managing IgG4-related disease (IgG4-RD), a rare immune-mediated condition. Based on the conclusions of a feasibility assessment, and due to the absence of head-to-head trials directly comparing Treatment X with rituximab (RTX)—a widely used treatment for IgG4-RD—the study employs an unanchored matching-adjusted indirect comparison (MAIC). This method uses individual patient data (IPD) from Trial Z (assessing Treatment X) and aggregate data from the Carruthers 2015 study (assessing RTX) to adjust for baseline differences in patient characteristics, including age, disease duration, and serum IgG4 levels. The analysis considers multiple scenarios to account for uncertainty in effect modifiers and prognostic factors. Results suggest that Treatment X may offer slightly superior or comparable efficacy to RTX in inducing complete remission, though findings vary across scenarios. However, RTX demonstrates a more favourable safety profile, with fewer adverse events consistently observed. Due to methodological limitations, such as small effective sample sizes and potential residual confounding, these findings are preliminary and warrant further investigation through direct comparative trials.
Αχιλλέας Μ. Ρώσιος (Wed,) studied this question.