Patient perspectives from the BEYOND Delphi panel: burden of remaining symptoms and their fluctuations, despite treatment, in patients with generalised myasthenia gravis

Abstract

Abstract Background Patients with generalised myasthenia gravis (gMG) can experience remaining symptoms despite treatment. Our study aimed to describe how remaining symptoms and their fluctuations may affect a person’s daily life while living with gMG. Methods A modified-Delphi panel (two survey rounds) was conducted with adult patients with gMG ( n =20) from seven European countries (France, Germany, Italy, the Netherlands, Sweden, Switzerland, and the UK). A scientific advisory committee including two neurologists and three patient advocacy group representatives guided the study. Consensus was defined as ≥80% participants in agreement (rated 7‒9 on a 9-point Likert scale) on a particular statement. Results Participants receiving a range of gMG treatments highlighted the personal and variable impacts of remaining symptoms in five key domains: daily activities, social life, mental wellbeing, work and education, and future plans. Across all domains, >40% of participants declared a high impact (rating 7–9 on Likert scale), with the highest proportion of patients declaring a high impact in the mental wellbeing domain (63%). Adjustments to daily activities, evening plans, working hours or even career moves, were frequently noted impacts of remaining symptoms and fluctuations. Participants also mentioned loss of independence and fears about forthcoming symptom severity as impactful. One statement reached consensus in agreement: “if my symptoms were better controlled, I would have greater freedom to do the things I enjoy and lead a more satisfying life”. Conclusions This study is the first to investigate the specific impacts of remaining symptoms of gMG on patients’ daily lives and how fluctuations may contribute to this impact. These results demonstrate the burden of these remaining symptoms for patients, suggesting that more effective therapeutic strategies leading to more sustained disease control would likely improve patients’ daily lives.