Learning About Medicines for Cystic Fibrosis

Cystic fibrosis, also called CF, is a disease caused by changes in a gene called the cystic fibrosis transmembrane conductance regulator (CFTR) gene. People are born with CF.

CF makes mucus, sweat, and digestive fluids thick and sticky. Thick mucus can make it hard to breathe and can cause lung infections. It can also make it hard for the body to digest and get nutrients from food.

Over time, CF can damage the lungs and other organs in the body.

Medicines help manage the symptoms and health problems caused by CF.

They can help:

• Improve breathing.
• Clear mucus from the lungs.
• Prevent and treat infections.
• Help the body digest food.
• Reduce inflammation in the body.
• Treat the CFTR protein problem that causes CF.

CF affects many parts of the body. So you may need more than one medicine every day.

CF medicines need to be taken every day to work well. They can help you breathe easier, prevent infections, and avoid hospital visits. Treatment is an important part of living with CF. It can help you live longer and feel better.

Several medicines are used to treat CF.

They help the CFTR protein work. This can help you breathe and digest food better.

These medicines are taken by mouth. They are an option for people with certain gene changes (mutations).

Examples include ivacaftor, tezacaftor, and elexacaftor.

They help relax and open the airways in the lungs. This makes it easier to breathe. They also help other inhaled medicines work better.

They are usually taken with an inhaler or nebulizer.

Examples include albuterol and levalbuterol.

They help loosen thick mucus in the lungs. This helps you cough the mucus out more easily.

Mucus thinners are inhaled, often with a nebulizer.

Examples include hypertonic saline, mannitol, and dornase alfa.

They help prevent and treat lung infections caused by bacteria in thick mucus. Inhaled antibiotics may be taken as part of regular daily treatment. Additional antibiotics may be given during exacerbations. These may be given orally or through an intravenous (I.V.) line.

Examples include tobramycin, azithromycin, and ciprofloxacin.

Many people with CF have a hard time digesting food. This happens because thick mucus blocks the release of pancreatic enzymes that the body needs for digestion. Pancreatic enzyme supplements are given to help the body digest food.

They are taken by mouth with meals and snacks.

They help reduce swelling in the lungs. This may help protect the lungs over time. Some are taken by mouth. Others are inhaled.

Examples include ibuprofen, corticosteroids, and azithromycin.

Medicines may be used together in a certain order. For example, bronchodilators and mucus thinners are used before inhaled antibiotic medicines. This helps open the airways and prepare the lungs for the antibiotics. Use treatments in the order your doctor recommends. Your doctor may also recommend that you use airway clearance techniques to clear mucus from your lungs before taking certain medicines.

Side effects for each medicine are different.

CFTR modulators may cause:

• Headaches.
• A stuffy nose.
• Belly pain.
• Liver problems.

Bronchodilators may cause:

• Shakiness.
• A fast heartbeat.
• Airway irritation.

Mucus thinners may cause:

• Coughing.
• A sore throat.
• Chest tightness.

Antibiotics may cause:

• Rashes.
• Dizziness.
• Nausea.
• Diarrhea.

Pancreatic enzyme supplements may cause:

• Bloating.
• Belly pain.
• Diarrhea.

Anti-inflammatory medicines may cause:

• Stomach problems.
• Kidney problems.
• High blood sugar.
• High blood pressure.

Using antibiotics the wrong way can also make infections harder to treat over time. This is called antibiotic resistance.

Talk with your doctor about side effects and ways to manage them.