PCORI has identified the need for large studies that look at real-life questions facing diverse patients, caregivers, and clinicians. In 2014, PCORI launched the Pragmatic Clinical Studies initiative to support large-scale comparative effectiveness studies focusing on everyday care for a wide range of patients. The Pragmatic Clinical Studies initiative funded this research project.
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?
Mycobacterium avium complex, or MAC, is a type of bacteria that exists in water, soil, and dust. Most people don’t get sick from MAC bacteria. But older adults, people with immune systems that don’t work well, and those with lung disease have an increased risk of MAC infections. MAC infections most commonly affect the lungs, and symptoms include shortness of breath, fever, and fatigue. Treatment involves taking multiple types of antibiotics for 18 to 24 months. These antibiotics can have side effects such as nausea and diarrhea. The infection’s symptoms and treatment side effects can decrease patients’ quality of life.
In this study, the research team is comparing the effects of treating MAC infection with two versus three different antibiotics. The team wants to know whether treatment with two different antibiotics is as effective as and better tolerated than treatment with three.
Who can this research help?
Results may help patients and doctors when considering how to treat MAC lung infections.
What is the research team doing?
The research team is enrolling 500 adults with MAC lung infections at 26 U.S. and Canadian sites in the study and assigning them by chance to receive treatment with either two or three different antibiotics. All patients receive standard care from their doctors.
The research team is reviewing health records and collecting clinical data for the first 12 months of treatment to see how many patients
- No longer have an infection
- Finish 12 months of treatment
- Develop resistance to the antibiotics
Five times during the study period, the research team is surveying patients about their symptoms, treatment side effects, and quality of life. The team is comparing outcomes between patients who take two antibiotics and those who take three.
Patients, patient advocates, and clinicians are providing input on the design, outcomes, and recruitment for the study.
Research methods at a glance
|Design||Randomized controlled trial|
|Population||500 adults ages 18 and older with pulmonary MAC disease|
Primary: acid-fast bacilli culture negativity, therapy completion with satisfactory adherence (defined as taking 80% of prescribed doses or not missing more than 75 days of treatment taken three times per week)
Secondary: respiratory symptoms, quality of life, fatigue, gastrointestinal symptoms, liver function, macrolide resistance
|1-year follow-up for primary outcomes|