I have spoken with many patients with nontuberculous mycobacterial (NTM) lung disease over the years who are frustrated by what may add up to years of missed diagnosis or improper treatment. Although awareness of NTM lung disease is growing in the medical community, there are many unanswered questions about how to best prevent, diagnose and treat the disease. By involving patients as partners in NTM research, researchers like myself are learning ways to design studies to answer the questions most important to patients.

What is NTM Lung Disease?

NTM lung disease is a little-known respiratory infection that is caused by inhaling a common organism, called nontuberculous mycobacteria, from the environment. Most people who are exposed to NTM don’t get sick, but some people are more susceptible to developing an infection that can lead to progressive and destructive lung disease. Groups at increased risk for NTM infection include people with a chronic lung disease such as bronchiectasis or COPD, people with weakened immune systems, post-menopausal women, and older adults.1,2

The Power of Patient-Centered Research

We believe patients have a lot to contribute and are the most important voice when planning research that answers the questions that are most important to patients. In November 2015, patients, caregivers, patient advocates, clinical experts, and researchers came together for a day long NTM meeting in the hopes of identifying the top research priorities for NTM. We learned that the top patient-centered priorities include prevention of NTM infection, approving more treatments that are easier to administer and potentially have fewer side effects, understanding the best lung clearance methods, developing and using tools to measure health-related quality of life and disease severity. Following the meeting, we published these findings so that they are available for use by other NTM researchers.1

Addressing NTM Patients’ Recommendations

Educate physicians to improve diagnosis and treatment 

There are major problems with the process of diagnosing NTM lung disease, this begins with who is tested. Development of a better process for screening people at risk for NTM lung disease, is one of the top patient-centered research priorities. Patients stress the importance of educating clinicians to recognize that symptoms like cough, fatigue and weight loss could be caused by an NTM infection. Missed diagnoses can be avoided if NTM is considered by primary care providers who see patients with recurrent or antibiotic resistant pneumonia, radiologists who see certain recognizable changes on chest x-rays and CT scans, and gastroenterologists who see patients with reflux and related pneumonia. All of these may be signs of NTM infection.1

In addition, the difficulty of getting a good test sample and the time it takes to confirm an infection make it crucial that primary care providers refer patients to experts, or consult with experts, to diagnose NTM patients and evaluate treatment options.2

Educate and empower patients to improve quality of life

What do patients do when they can’t get all the information they need from their healthcare team? They often look to each other and to patient advocacy organizations. The American Lung Association, NTM Info & Research (NTMir) and the COPD Foundation all have a wealth of resources to help NTM lung disease patients learn to manage their disease, including online communities and local support groups. 

Patient advisors have emphasized the importance of helping NTM lung disease patients understand and follow a regular regimen to clear their airways of mucus, and to know how to select a technique that is effective and has the least impact on their daily lives. Our physician advisors stressed the need to reassure patients who require treatment with antibiotics that, contrary to what they might have heard, the therapies are manageable and the drugs are not “worse than the disease.”The decision to start (and stop) antibiotic treatment should be made with careful consideration of patients’ wishes and understanding of potential risks and benefits.

Patients who already have NTM disease are often concerned about the risk of re-infection or co-infection with multiple species of NTM.1,3 Patients report that they have adopted a “careful way of living”, given that NTM is ubiquitous in the water and environment.1 Unfortunately, there is little consensus among experts on how to prevent infection and reinfection with NTM, and everyone agreed that more research is needed.1 

Increase research capacity to involve patients

NTM lung disease is a rare disease, meaning fewer than 200,000 people in the U.S. are known to be living with it at any given time.2,3,4 There is one patient registry in the U.S., and it does not include patient-reported data, which is important to capture changes in how a patient feels and functions. We need to expand data sources and analysis to better understand the natural history of the disease. So, we formed an NTM clinical trials network and are currently conducting a large clinical trial at over 25 sites across the U.S. We hope to develop the infrastructure needed to increase patient access to clinical trials close to home, eliminating their need to travel to national centers of NTM expertise to participate.

Modern research is increasingly being conducted with patient partners, and we are proud to be helping to lead the way with NTM research. The U.S. Food and Drug Administration has already gathered input from NTM patients and clinical experts to inform their work.3 Funding agencies increasingly require patients to be involved in study development and clinical trial recruitment as stakeholders. The purpose is to conduct meaningful clinical research in order to answer questions most relevant to patients. Working together, we can reach one patient’s vision of “a world free of NTM lung disease”.

Despite the fact that the number of people living with NTM lung disease is on the rise, most people have never heard of it.4 It is important to get the word out to high-risk individuals and the healthcare providers that care for them so that people can be diagnosed earlier and start the right treatment when needed.

To learn more about recognizing, diagnosing and treating NTM, visit Lung.org/NTM or call the American Lung Association Lung HelpLine at 1-800-LUNG-USA.

Development of this educational content was supported by a collaborative sponsorship from Insmed Incorporated.

Henkle E, Aksamit T, Barker A, Daley CL, Griffith D, Leitman P, et al. Patient-Centered Research Priorities for Pulmonary Nontuberculous Mycobacteria (NTM) Infection. An NTM Research Consortium Workshop Report. Annals of the American Thoracic Society. 2016;13(9):S379-84.

Griffith DE, Aksamit T, Brown-Elliott BA, et al. An official ATS/IDSA statement: diagnosis, treatment, and prevention of nontuberculous mycobacterial diseases. American journal of respiratory and critical care medicine 2007;175:367-416.

Center for Drug Evaluation and Research (CDER), U.S. Food and Drug Administration (FDA).  The Voice of the Patient.  Non-Tuberculous Mycobacterial (NTM) Lung Infection.  Public Meeting: October 15, 2015.  Report Date: April, 2016.  accessed 4/30/2016 at http://www.fda.gov/downloads/ForIndustry/UserFees/PrescriptionDrugUserFee/UCM496941.pdf.

Floto RA, Olivier KN, Saiman L, et al. US Cystic Fibrosis Foundation and European Cystic Fibrosis Society consensus recommendations for the management of non-tuberculous mycobacteria in individuals with cystic fibrosis: executive summary. Thorax 2016;71:88-90.

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