Cystic Fibrosis Treatment

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Although there is not yet a cure for cystic fibrosis, advances in treatment are helping many people live longer, healthier lives. To manage the disease, you will work with a team of healthcare providers, including cystic fibrosis specialists. For newborns with a positive screening result, treatment may begin while the diagnosis is being confirmed.

Treatment for cystic fibrosis focuses on medicines and other ways to clear the airway. Other medicines can improve the function of the faulty CFTR protein and help prevent complications. Sometimes breathing support, nutritional support, or surgery is needed.

Your team of healthcare providers will likely include a cystic fibrosis specialist. This is a doctor who is familiar with the complex nature of cystic fibrosis.

Your cystic fibrosis care team may also include:

• Doctors specializing in the lungs, diabetes, and the digestive system
• Respiratory therapists
• Genetic counselors
• Fertility and reproductive health specialists
• Physical therapists
• Nurses
• Nutritionists and dietitians
• Pharmacists
• Psychologists
• Social workers
• Transplant doctors and nurses

Young people and the transition to adult care

As they grow more independent, teens need to take charge of their health, manage their own appointments, and take their medicines and other treatments. Not following treatment recommendations can lead to serious illness and hospital stays.

Airway clearance techniques help loosen lung mucus so it can be coughed out. Removing mucus improves breathing and lowers the chance of infections. The techniques may include:

• Special ways to breathe and cough, such as active cycle of breathing (ACBT) and autogenic drainage. You perform a set of breathing exercises to relax the airways and push the mucus out of your lungs.
• Devices used by mouth, called positive expiratory pressure (PEP) devices. You use the PEP device for a certain number of breaths and then forcefully exhale or cough. The device makes it harder to breathe out, which helps to push the mucus out of your lungs. Oscillating PEP is a type of PEP that also vibrates while you breathe out.
• Manual chest physical therapy. Another person, such as a respiratory therapist or trained family member, claps or shakes the chest wall. It can also be done using a percussion device. Lying in certain positions during therapy helps the mucus drain out of the lungs. 
• Therapy vests, also called high-frequency chest wall oscillation. You wear a vest attached to a machine that quickly inflates and deflates the vest. The vest vibrates the chest wall and airways to loosen mucus.

Medicines to treat cystic fibrosis may maintain and improve lung function, fight infections, clear mucus and help breathing, or try to improve how well someone’s CFTR protein works.

• Antibiotics prevent or treat lung infections and improve lung function. Your healthcare provider may prescribe oral, inhaled, or intravenous (IV) antibiotics.
• Anti-inflammatory medicines, such as ibuprofen or corticosteroids, reduce inflammation. Inflammation causes many of the changes in cystic fibrosis, such as lung disease. Ibuprofen is especially helpful for children, but side effects can include kidney and stomach problems. Corticosteroids can cause bone thinning and increase blood sugar and blood pressure.
• Bronchodilators relax and open airways. These treatments are taken by inhaling them.
• Mucus thinners make it easier to clear the mucus from your airways. These treatments are taken by inhaling them.
• CFTR modulators make faulty CFTR proteins work better. They help with lung function and can help prevent lung problems or other complications. CFTR modulators do not work for all people with cystic fibrosis because they only work for certain CFTR gene mutations. Your healthcare provider will first order genetic testing to figure out whether CFTR modulator therapy is likely to work for you. These medicines are taken by mouth. Some CFTR modulators may interact with other medicines, including birth control pills. They may cause side effects, including temporary worsening of breathing symptoms, nasal or sinus congestion, headache, or abdominal pain. While it is not common, some people experience anxiety or depression when taking CFTR modulators. There are several CFTR modulator therapies:
  • A triple combination medicine (elexacaftor-tezacaftor-ivacaftor) may help up to 90% of people who have cystic fibrosis. It is currently approved for adults and children age 2 and older with certain CFTR mutations.
  • Ivacaftor is approved for adults and children as young as 4 months old.
  • A combination of lumacaftor and ivacaftor is approved for people who are at least 1 year old.
  • A combination of tezacaftor and ivacaftor is approved for people as young as 4 months old.

People who are Asian, Black, or Hispanic are more likely to have mutations that will not respond to available CFTR modulators. 

If you are not eligible for treatment with a CFTR modulator, your healthcare provider may recommend participating in a clinical study. Scientists continue to work on new treatments for cystic fibrosis. 

It can be very hard to learn that CFTR modulators will not work for you because of the type of CFTR gene mutation you have. If that is the case, talk with your healthcare team about mental health support and other treatment options for you.

People living with cystic fibrosis who have severe lung problems sometimes need other treatments, too.

• Oxygen therapy gives you more oxygen to breathe. You can receive oxygen therapy from tubes placed in your nose, a face mask, or a tube placed in your trachea (windpipe).
• Pulmonary rehabilitation is a supervised medical program that helps people with lung diseases live and breathe better. During the program, you will learn exercises and breathing techniques.
• Ventilator support uses a machine to blow moist air into your lungs through a breathing tube or tightly fitting mask. Your healthcare provider sets the ventilator to control how often it pushes air into your lungs and how much air you get.
• Extracorporeal membrane oxygenation (ECMO) pumps your blood through an artificial lung to add oxygen and remove carbon dioxide . The blood is then returned to your body. Healthcare providers use ECMO to treat people with severe breathing problems. It may be used for several days or weeks to give the lungs a chance to recover. Some complications of ECMO are blood clots, bleeding, and infections, all of which can be life-threatening.

People with advanced conditions may need surgery.

• A lung transplant may help people with severe lung disease and respiratory failure.
• A liver transplant may be an option for advanced liver disease such as cirrhosis.

Regular checkups with your healthcare provider are an important part of your follow-up and treatment. How often these checkups take place depends on your age. Younger people and those who have just been diagnosed will have more frequent visits. As an adult, you may see your provider less often, maybe every 3 months and then once a year.

Regular checkups may include:

• Education about the importance of following treatment recommendations, such as airway clearance therapies, infection control, taking medicines as prescribed, and other steps to lower your risk of complications
• Height and weight measurement and calculation of body mass index (BMI)
• Nutrition guidance, including regular assessments and a personalized food plan to meet your energy and nutrition goals
• Physical activity guidance
• Physical exam, including examination of the heart, lungs, and abdomen to check the liver and look for abdominal pain
• Physical therapy evaluation 
• Psychological assessment and support
• Respiratory therapy evaluation 

Even if your symptoms get better, be sure to see your providers regularly for ongoing care.