This thesis critically evaluates the current clinical research landscape in autoimmune bullous diseases (AIBDs), a heterogeneous group of rare blistering disorders caused by autoantibodies targeted against adhesion molecules of the skin and/or mucous membranes. AIBDs are classified into pemphigoid diseases, characterized by subepidermal blistering due to autoantibodies against various structural proteins of hemidesmosomes, and pemphigus diseases, which are intraepidermal blistering disorders driven by autoantibodies targeting the desmosomal cadherins desmoglein (DSG) 1 and/or DSG 3. Common pemphigoid subtypes include bullous pemphigoid (BP) and mucous membrane pemphigoid (MMP), while pemphigus mainly comprises pemphigus vulgaris (PV) and pemphigus foliaceus (PF).
Despite decades of clinical trials and extensive preclinical evidence, the development of targeted therapies for AIBDs has remained limited. This limited progress underscores the need for a critical evaluation of the current clinical research landscape in AIBDs. This thesis provides a comprehensive analysis of clinical research in AIBDs, with particular emphasis on outcome measurement, challenges in trial implementation, and the evaluation of novel therapeutic approaches.
