Myasthenia Gravis: Reducing Outcome Measures Variability In Trials

By Lewis M. Fredane and Marta Pereira

June is ‘Myasthenia Gravis Awareness’ month, highlighting this rare autoimmune disease. Over recent decades, clinical trials for Myasthenia Gravis (MG) have advanced significantly, improving our understanding and treatment of this condition. MG, a neuromuscular disease causing muscle weakness and fatigue, affects about 150 to 200 people per million. It disrupts nerve-muscle communication due to autoantibodies blocking acetylcholine receptors.

Clinical trials have evolved from using acetylcholinesterase inhibitors to exploring monoclonal antibodies. Key challenges in MG trials include recruitment difficulties, inadequate study durations, lack of sensitive outcome measures, and variability in symptom assessment. Standardizing outcome measures, rater training, incorporating data analysis, and using electronic clinician ratings are crucial to address these issues. These methods enhance data reliability and support the development of effective MG therapies.

Uncover more information about MG and how to manage unpredictability in clinical trials by reading the article below.