Patient perspectives on treatment outcomes and priorities in autoimmune bullous diseases: an exploratory survey among Dutch patients.

Introduction Autoimmune bullous diseases (AIBDs), comprising pemphigoid and pemphigus diseases, have seen limited therapeutic advances beyond rituximab for pemphigus vulgaris. As novel therapies are evaluated in clinical trials, well-defined, uniform and relevant outcomes with patient involvement are essential. To date, however, patient-reported outcomes remain underrepresented, leaving uncertainty about whether trial results genuinely reflect patients' expectations. This study examines perspectives of patients with AIBD on treatment outcomes and priorities to ensure that future research focuses on what matters most to them. Methods A cross-sectional study was conducted among Dutch patients with AIBD between October 2023 and January 2024, using a self-developed questionnaire with both closed- and open-ended questions to assess patient perspectives on treatment outcomes and priorities, key factors in choosing a treatment, and indicators of treatment success. Results Regarding skin and/or mucous membrane complaints, 'the formation of new blisters and wounds' emerged as the most important complaint a treatment should address for both pemphigoid (43%) and pemphigus (86%) patients. In open-ended questions, patients with pemphigoid most frequently prioritized 'pruritus' (44%), while patients with pemphigus emphasized 'pain' (39%). Most important concerns regarding physical and daily functioning were 'vision problems' (20%), 'sleep disturbances' (20%), and 'self-care difficulties' (23%) for patients with pemphigoid, whereas patients with pemphigus most commonly cited 'eating and/or swallowing difficulties' (57%) and 'daily activity limitations' (41%). Regarding emotional/psychological functioning, both subgroups prioritized 'anxiety and/or worry' as most important concern (pemphigoid: 28%, pemphigus: 43%). Side effects were identified as the most important factor in choosing a treatment (pemphigoid: 41%, pemphigus: 39%). The absence of one or more symptoms and clinical signs (i.e., 'no blisters') was mentioned as the most important indicator of treatment success (pemphigoid: 88%, pemphigus: 91%), although minimal clinical signs (i.e., 'minimal blisters') were also considered acceptable (pemphigoid: 25%, pemphigus: 13%). Conclusion Patients with pemphigoid and pemphigus exhibit some distinct treatment priorities, reflecting their distinct pathomechanisms. Nonetheless, both subgroups consistently prioritize not only the resolution of disease-specific clinical signs but also preservation of physical and psychological well-being, underscoring the need for more holistic and patient-centred outcome measurement to ensure the establishment of meaningful, AIBD subgroup specific treatment outcomes.