Identifying a patient-centered outcome measure for a comparative effectiveness treatment trial in myasthenia gravis.

Introduction/aim Data regarding the comparative effectiveness of myasthenia gravis (MG) treatments is not available. We used patient input to identify a patient-centered outcome measure (PCOM) for PROMISE-MG, a comparative effectiveness trial of myasthenia gravis (MG) treatments. Methods 1. A questionnaire survey was administered to 58 people with MG at the patient meeting of the Myasthenia Gravis Foundation of America (MGFA), evaluating the impact of MG-related symptoms and MG treatments on patients' lives. 2. An online focus group of 13 patients with MG was conducted. 3. A potential outcome measure was selected. 4. The selected PCOM was evaluated by patients to assess how completely and accurately it captured their experiences with MG. Results The patient survey found that limb weakness had the most impact on patients' lives. Weight gain, mood swings, insomnia and diarrhea were the most bothersome treatment side effects. Avoiding hospitalization was very important. Focus group participants reported fatigue as one of the most bothersome symptoms and differentiated it from myasthenic weakness. They defined an ideal treatment as having minimal or no side effects and an 80% improvement in symptoms. Discussion Based on patient input, the Myasthenia Gravis- quality of life 15-revised scale (MG-QOL15r), a validated patient-reported outcome measure (PRO) was selected as the primary PCOM for PROMISE-MG. Avoiding hospitalization and having minimal-to-no treatment adverse effects, were selected as additional outcome measures. The patient centeredness of a PRO depends on the context of a study: PROs should be evaluated for appropriateness as a PCOM for every study. This article is protected by copyright. All rights reserved.