Characterization and optimization of myasthenia gravis care

Myasthenia gravis (MG; OrphaCode 391490; ICD-10 G70.0) is a chronic autoimmune disease affecting neuromuscular signal transmission, with an estimated prevalence of 15 to 25 per 100.000. Disease severity varies from milder forms with potential for long-term remission to severe life-threatening disease.

Clinical management is still mainly based on empirical evidence, with a scarcity of reliable clinical predictors or biomarkers for disease sub-form stratification and long-term outcomes. As a consequence, treatment practices vary both within and between countries.

We will here perform a nationwide registry linkage study in Sweden to establish more precise prevalence/incidence rates and candidate clinical predictors of long-term outcomes, also including patient-reported outcomes and epidemiological questionnaire data.

These findings will be corroborated, extended and cross-compared in large and well-characterized cohorts from 5 countries across Europe reflecting different geographic areas, patient populations and treatment practices.

Finally, using advanced immunological methods we will identify novel immunological markers for MG sub-form characterization and disease severity prediction to be validated in a multicenter prospective, observational study of new onset MG.

The knowledge thus gained has the potential to change treatment practices with currently available interventions and to be informative for future intervention studies.