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IntroductionMyasthenia gravis (MG) is a rare, chronic, autoimmune disease, mediated by immunoglobulin G antibodies, which causes debilitating muscle weakness. As with most rare diseases, there is little patient-reported data with which to understand and address patient needs. This study explores the impact of MG in the real world from the patient perspective.Methods and analysisThis is a 2-year prospective, observational, digital, longitudinal study of adults with MG, resident in the following countries: the USA, Japan, Germany, France, the UK, Italy, Spain, Canada and Belgium. The planned sample size is 2000. Recruitment will be community based, via patient advocacy groups, social media and word of mouth. Participants will use a smartphone application (app) to check eligibility, provide consent and contribute data. Planned data entry is as follows: (1) personal profile on enrollment—covering demographics, MG characteristics and previous care; (2) monthly event tracker—current treatments, healthcare visits, treatment-related adverse events, productivity losses; (3) monthly selection of validated generic and disease-specific patient-reported outcomes instruments: EQ-5D-5L, Myasthenia Gravis Activities of Daily Living, Myasthenia Gravis Quality of Life 15-item revised scale, Hospital Anxiety and Depression Scale and Health Utilities Index III. Analyses are planned for when the study has been running in most countries for approximately 6, 12, 18 and 24 months.Ethics and disseminationThe study protocol has been reviewed and granted ethics approval by Salus IRB for participants resident in the following countries: Germany, the UK and the US. Local ethics approval is being sought for the following study countries: Belgium, Canada, France, Italy, Japan and Spain. Study results will be communicated to the public and participants via conference presentations and journal publications, as well as regular email, social media and in-application communication.Trial registration numberNCT04176211.

The Myasthenia Gravis Activities of Living (MG‐ADL) scale is an 8‐item patient‐reported scale that measures myasthenia gravis (MG) symptoms and functional status. The objective of the current review is to summarize the psychometric properties of the MG‐ADL and published evidence of MG‐ADL use. A targeted literature review for published studies of the MG‐ADL was conducted using a database and gray literature search. A total of 48 publications and 35 clinical trials were included. Studies indicated that the MG‐ADL is a reliable and valid measure that has been used as an outcome in clinical trials and observational studies to measure MG symptoms and response to treatment. While most often used as a secondary endpoint in clinical trials, its use as a primary endpoint has increased in recent years. The most common MG‐ADL endpoint is change in MG‐ADL score from baseline, although there has been an increase in the analysis of a responder threshold using the MG‐ADL. A new concept of minimal symptom expression (MSE) has emerged more recently. Duration of treatment effect is another important construct that is being increasingly evaluated using the MG‐ADL. The use of the MG‐ADL as a primary endpoint in clinical trials and in responder threshold analyses to indicate treatment improvement has increased in recent years. MSE using the MG‐ADL shows promise in helping to determine success of treatment and may be the aspirational goal of MG treatment for the future once validated, particularly given the evolving treatment landscape in MG.

Objectives: To understand the economic and humanistic burden attributed to myasthenia gravis (MG). Methods: We conducted a literature search (January 2009 to April 2019) in the biomedical database Embase (including Medline), focusing on English language observational studies and economic models evaluating patients with MG in the United States. Key search terms focused on costs, healthcare resource utilization, and quality of life (QoL). Results: A total of 18 studies were identified that included economic or humanistic burden data. Median length of hospitalization in patients with MG ranged from 2 days to 8 days, with costs per admission, using National Inpatient Sample data, ranging from a median of $16,000 in 1 study to a mean of nearly $99,000 in another analysis. Patient factors including MG crisis, presence of respiratory failure, and complex medication regimens (including multiple immunosuppressive and conventional therapies) worsened the burden, increasing length of stay from a median of 4 -6 days to 6 -10 days. Costs of admission increased from $16,000 to $26,600 for patients hospitalized for MG (not MG crisis), up to almost $54,000 for patients experiencing an MG crisis and receiving plasma exchange. Among patients treated with multiple immunosuppressants, presence of continued functional disability contributed to a worse QoL. One study found that QoL was worse in women than men, but the difference was eliminated for women who had undergone a thymectomy. Conclusions: The economic and humanistic burden associated with MG is considerable, particularly for patients with severe complications, including MG crisis. Understanding the risk factors associated with MG-related complications, and the potential impact on treatment modalities, is crucial to improve the medical management of these patients. Additionally, there is a clear unmet need in this patient population for new treatment options to improve disease course and prevent complications.

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