Camdessanché Jean-Philippe, Sacconi Sabrina, Archer Annie, Boulanger Pierre, Crochard Anne, Bertocchio Jean-Philippe, Richard Aliénor, Villy Pierre-Edouard, Solé Guilhem
Centre de Référence Maladies Neuromusculaires Rares, CHU de Saint-Étienne Service de Neurologie, Saint-Etienne, France.
Université Côte d'Azur, Système nerveux périphérique et muscle, CHU Nice, Nice, Provence-Alpes-Côte d'Azur, France.
BMJ Open. 2024 Dec 2;14(12):e088813. doi: 10.1136/bmjopen-2024-088813.
To capture patient perceptions about living with myasthenia gravis (MG) with respect to aspirations and ways to improve treatment.
Online patient survey.
Patients recruited by MG patient associations or at MG reference treatment centres.
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: 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.
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).
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.
了解重症肌无力（MG）患者对生活的看法，涉及期望以及改善治疗的方法。
在线患者调查。
由MG患者协会或MG参考治疗中心招募患者。
经医生诊断为全身性MG、在法国居住≥12个月且接受过≥1次MG治疗的成年人符合条件。291名患者打开了问卷，255名符合条件，246名完成了调查（年龄范围41 - 67岁；187名女性和59名男性）。
主要指标：对“患有这种疾病，你希望做但目前觉得困难或无法做到的事情是什么？”这一问题的自由文本回答。次要指标：对“你认为MG治疗可以做出哪些改进？”这一问题的自由文本回答。使用扎根理论和聚类分析对这些问题的回答主题进行分析。
对于疾病期望问题，238名参与者提供了617条引用，这些引用被归为45个维度和六个高级领域（身体活动、日常生活活动、心理负担、社交活动、工作/学校和其他）。最常被提及的维度是运动（82条引用）、更大的活动能力（56条引用）、不那么疲劳（46条引用）和更强的耐力（37条引用）。年龄较小、女性、近期诊断以及较差的生活质量与提及更多主题相关。对于治疗改善问题，195名参与者提供了263条引用，这些引用被归为60个维度和三个高级领域（药物特性、安全性和护理模式）。最常被提及的与治疗相关的维度是副作用更少（40条引用）、每日服药次数更少（21条引用）和消化副作用更少（20条引用）。
这些发现有助于医疗保健专业人员理解并更好地满足患者对MG生活的期望，特别是关于身体活动的重要性，以及他们对改善治疗的期望。
