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 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 include:

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

Airway clearance techniques help loosen lung  mucus  so it can be cleared, reducing infections and improving breathing. The techniques may include special ways to breathe and cough, devices used by mouth, therapy vests that use vibrations to loosen mucus, and chest physical therapy. These techniques are often used along with medicines such as bronchodilators (medicines that open up the airways) and mucus thinners.

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 increased blood sugar and blood pressure.
• Bronchodilators relax and open airways. These treatments are taken by inhaling them.

• CFTR modulators improve how a faulty CFTR protein works. They help with lung function and can help prevent lung problems or other complications. Not all CFTR modulators work for all people with cystic fibrosis. The choice of CFTR modulator depends on which CFTR gene mutation you have. Before you start taking a CFTR modulator, your healthcare provider or a genetic counselor will order genetic testing to figure out whether a 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. There are several CFTR modulator therapies approved for use in cystic fibrosis:

  • A triple combination medicine (elexacaftor-tezacaftor-ivacaftor) is the first approved treatment and 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 won’t 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 trials . Scientists continue to work on new treatments for cystic fibrosis. 

  It can be very hard for people to find out that CFTR modulators won’t work for them because of the type of CFTR gene mutation they have. It is common to feel depressed or anxious if you learn that you are not eligible for CFTR modulators. Talk with your healthcare team about mental health support and other treatment options for you.

• Mucus thinners make it easier to clear the mucus from your airways. These treatments are taken by inhaling them.

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 who have 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  before returning the blood 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.

Surgery may be needed for people with advanced conditions.

• 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.