SPOON: an observational, cross-sectional study of perceptions and expectations of adults with generalised myasthenia gravis in France

Abstract

Objectives: To capture patient perceptions about living with myasthenia gravis (MG) with respect to aspirations and ways to improve treatment.

Design: Online patient survey.

Setting: Patients recruited by MG patient associations or at MG reference treatment centres.

Participants: Adults with physician-diagnosed generalised MG, living in France for ≥12 months, who had received ≥1 MG treatment were eligible. 291 patients opened the questionnaire, 255 were eligible and 246 completed the survey (age range 41-67 years; 187 women and 59 men).

Primary and secondary outcome measures: Primary: free-text response to the question 'Living with your disease, what would you like to do that you currently find difficult or impossible to do?' Secondary: free-text response to the question 'What improvements do you think could be made to treatments for MG?' Themes from replies to these questions were analysed using grounded theory and cluster analysis.

Results: For the disease aspirations question, 617 citations were provided by 238 participants, which were grouped into 45 dimensions and six high-level domains (physical activity, activities of daily living, psychological burden, social activities, work/school and other). The most frequently cited dimensions were sport (82 citations), greater mobility (56 citations), being less tired (46 citations) and greater endurance (37 citations). Younger age, female gender, recent diagnosis and poorer quality of life were associated with citing more themes. For the treatment amelioration question, 263 citations were provided by 195 participants, which were grouped into 60 dimensions and three high-level domains (medication characteristics, safety and care paradigm). The most cited treatment-related dimensions were fewer side effects (40 citations), fewer daily medication intakes (21 citations) and fewer digestive side effects (20 citations).

Conclusions: These findings could help healthcare professionals to understand and better address patients' aspirations about living with MG, notably concerning the importance of physical activity, and their expectations for improved treatments.

Keywords: Neuromuscular disease; Patient Reported Outcome Measures; Patient Satisfaction; Quality of Life.

Figure 1. Qualitative analysis. (A) Domains and dimensions of unfulfilled aspirations about disease—’In your life with MG, what would you like to do that, at the moment, you have difficulty doing or can’t do?’. The analysis was performed on the 238 participants who answered this question. Domains are represented by the inner branches, with the width of the branches corresponding to the number of citations. Dimensions are represented by the circles at the end of the branches, with the size of the circle corresponding to the number of citations. The actual number of citations for each dimension is indicated outside the circles. (B) Domains and dimensions of ways to improve treatment—’In your opinion, how could myasthenia treatments be improved?’. The analysis was performed on the 195 participants who answered this question. Domains are represented by the inner branches, with the width of the branches corresponding to the number of citations. Dimensions are represented by the circles at the end of the branches, with the size of the circle corresponding to the number of citations. The actual number of citations for each dimension is indicated outside the circles.

Figure 2. Poisson regression model: variables associated with the number of citations. HADS: Hospital Anxiety and Depression Scale; MG-ADL: MG activities of daily living profile.