This research project is in progress. PCORI will post the research findings on this page within 90 days after the results are final.
What is the research about?
Myasthenia gravis, or MG, is a rare disease of the immune system that can weaken certain muscles. MG can cause droopy eyelids and trouble swallowing, chewing, talking, and breathing. There is no cure for MG, but treatment can help relieve symptoms. Many patients benefit from immunosuppressive medicines, which change how the immune system functions. But doctors don’t know how medicines compare for relieving symptoms of MG.
In this study, the research team is comparing two medicine regimens for treating MG. In one regimen, patients receive azathioprine. In the other, patients receive mycophenolate mofetil. The team wants to know how each regimen affects patient outcomes such as MG symptoms, quality of life, and physical function. Also, the team wants to know how these outcomes vary for patients who take the recommended dose and duration of their treatment, based on treatment guidelines, compared with patients who don’t.
Who can this research help?
Patients and doctors can use the results when considering how to treat MG.
What is the research team doing?
The research team is recruiting 220 adult patients with MG treated at 20 clinics in the United States and Canada. Patients in the study receive standard clinical care. At the start of the study, and again one and two years later, the team is reviewing data about patients’ quality of life, symptoms, treatment side effects, physical function, symptom severity, and ability to perform daily activities. The team is also looking at treatment effects, physical exams, muscle function, and whether patients went to the hospital for MG during the two-year study. The team is comparing patients who receive azathioprine to those who get mycophenolate mofetil. Also, the team is comparing patients who get the recommended dose and duration to those who don’t.
Patients with MG, doctors who treat MG, health insurers, and patient organizations help design and conduct this study. They help make sure that the study looks at outcomes that are important to patients.