Myasthenia gravis (MG) is a rare, chronic disease mediated by immunoglobulin G antibodies that causes crippling muscle weakness. As with most rare diseases, there is little self-reported data to understand and meet their needs.
This study explores the impact of MG on the day-to-day lives of patients. This prospective, observational, digital longitudinal study, lasting 2 years, will concern adults with MG residing in the following countries: the United States, Japan, Germany, France, the United Kingdom, ‘Italy, Spain, Canada and Belgium. The expected sample size is 2000. Recruitment will be community based, through patient advocacy groups, social media and word of mouth. Participants will use a smartphone app to verify eligibility, provide consent and provide data. The planned data entry is as follows:
- personal profile on demographics covering enrollment, characteristics of MG and previous care;
- monitoring of monthly events, current treatments, medical visits, treatment-related adverse events, productivity losses;
- monthly selection of validated instruments of generic and disease-specific patient-reported outcomes: EQ-5D-5L, Myasthenia Gravis Activities of Daily Living, Myasthenia Gravis Quality of Life revised 15 item scale, Hospital Anxiety and Depression Scale and Health Utilities Index III.
Analyzes are scheduled after the study has started in most countries about 6, 12, 18 and 24 months ago.The results of the study will be communicated to the public and participants through conference presentations and journal publications, as well as through regular email, social media and in-app communication.
Plus d’infos sur cet essai : NCT04176211.
