Cystic Fibrosis Treatment

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While there is not yet a cure for cystic fibrosis, advances in treatment are helping people live longer, healthier lives. To better manage your condition, you or your child will work with cystic fibrosis specialists. In newborns with a positive screening result, treatment may begin while the diagnosis is being confirmed. Treatment for cystic fibrosis is focused on airway clearance, medicines to improve the function of the faulty CFTR protein and prevent complications, and surgery, if needed.

Your healthcare team will likely include a cystic fibrosis specialist. This is a doctor who is familiar with the complex nature of cystic fibrosis. Your doctor may work with a medical team that specializes in cystic fibrosis, often at major medical centers.

The United States has more than 100 CF Care Centers, with medical teams that include:

• Doctors specializing in the lungs, diabetes, and the digestive system
• Genetic counselors
• Nurses
• Nutritionists and dietitians
• Pharmacists
• Physical therapists
• Psychologists
• Respiratory therapists
• Social workers

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

Medicines to treat cystic fibrosis include those used to maintain and improve lung function, fight infections, clear mucus and help breathing, and work on the faulty CFTR protein.

• Antibiotics prevent or treat lung infections and improve lung function. Your doctor 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 beneficial 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 the function of the faulty CFTR protein. They improve lung function and help prevent lung problems and other complications. Examples include ivacaftor and lumacaftor and a triple combination medicine (elexacaftor–tezacaftor–ivacaftor). The combination medicine is the first approved treatment that may help up to 90% of people who have cystic fibrosis. It is currently approved to use in adults and children older than 12 years.
• Mucus thinners make it easier to clear the mucus from your airways. These treatments are taken by inhaling them.

Surgery may be an option for people with advanced conditions.

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