Project Summary

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.

Research methods at a glance

Design Elements Description
Design Observational: prospective cohort study
Population Adults ages 18 and older diagnosed with MG
  • Azathioprine
  • Mycophenolate mofetil 

Primary: MG quality of life, composite of clinical improvement and adverse effects of treatments

Secondary: physical function, clinical examination findings, activities of daily living, muscle function, patient perception of disease severity, patient perception of treatment side effects, hospitalizations for MG

Timeframe 2-year follow-up for primary outcomes

Project Information

Donald B. Sanders, BS, MD
Pushpa Narayanaswami, MB, BS, DM
Duke University
Prospective Multicenter Observational Cohort Study Of Comparative Effectiveness of Disease-Modifying Treatments for Myasthenia Gravis (MG)

Key Dates

August 2017
February 2023

Study Registration Information


Award Type
Health Conditions Health Conditions These are the broad terms we use to categorize our funded research studies; specific diseases or conditions are included within the appropriate larger category. Note: not all of our funded projects focus on a single disease or condition; some touch on multiple diseases or conditions, research methods, or broader health system interventions. Such projects won’t be listed by a primary disease/condition and so won’t appear if you use this filter tool to find them. View Glossary
Populations Populations PCORI is interested in research that seeks to better understand how different clinical and health system options work for different people. These populations are frequently studied in our portfolio or identified as being of interest by our stakeholders. View Glossary
Intervention Strategy Intervention Strategies PCORI funds comparative clinical effectiveness research (CER) studies that compare two or more options or approaches to health care, or that compare different ways of delivering or receiving care. View Glossary
State State The state where the project originates, or where the primary institution or organization is located. View Glossary
Last updated: August 23, 2022