Cystic Fibrosis medications & treatments

Medically reviewed by  Jamie Winn, PharmD

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Cystic fibrosis (CF) is a genetic disorder that can cause severe damage to the lungs, digestive system, and other organs in the body. It is estimated that over 30,000 children and adults in the United States have been diagnosed with this condition, with 1,000 new cases each year. While it is more common in the white population (1 in about 3,000) of the US, it affects every racial and ethnic group. It affects about 1 in 17,000 African Americans and 1 in 31,000 Asian Americans.

What is cystic fibrosis?

Cystic fibrosis is a progressive, genetic disease that causes long-lasting lung infections and limits the ability to breathe over time. It is due to a mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene which causes the CFTR protein to not work properly. With this protein not working correctly, it is unable to move chloride and water to the cell surface. This results in the production of thick, sticky mucus by the cells that line the passageways of the lungs, liver, pancreas, and intestines, as well as sweat glands. This thick mucus can build up and cause clogs and blockages in the lungs and digestive system, which can lead to infections, breathing problems, and other complications.

Cystic fibrosis causes

Cystic fibrosis is an inherited disease that must come from both parents. If only one parent passes the CFTR gene mutation to their child, they will generally be healthy but can pass it along to any children they have.

Cystic fibrosis is a chronic disease with life-threatening complications. Improved screening and treatments have increased the life expectancy for people with CF. Fifty years ago, CF patients would barely make it to their teenage years while now can live into their 40s and 50s.

How is cystic fibrosis diagnosed?

Newborns in the US are now routinely screened for cystic fibrosis, among other diseases and conditions. Some of the tests used for the diagnosis of cystic fibrosis include:

• Newborn screening test. This blood test measures the amount of a pancreatic protein called trypsinogen using IRT (immunoreactive trypsinogen test). There are several reasons that a newborn’s IRT levels may be elevated so further testing will need to be done to confirm a CF diagnosis.
• Sweat test. This test is usually done once an infant reaches 2 weeks old and it measures the amount of chloride in their sweat. This is the standard test to confirm a CF diagnosis. This test should be done by a Cystic Fibrosis Foundation accredited care center.
• Genetic testing. This will look for mutations in the CFTR gene, which is responsible for CF.

Cystic fibrosis symptoms

Some of the common symptoms of cystic fibrosis you might see after a positive diagnosis include:

• Persistent cough
• Shortness of breath
• Wheezing
• Sinus infections
• Nasal polyps
• Constipation
• Diarrhea
• Salty skin and sweat
• Greasy, foul-smelling bowel movements

What are some cystic fibrosis treatment options?

If you have cystic fibrosis, you will have to go through a daily routine of treatments to stay healthy. The goal of CF treatment is to prevent lung infections, loosen and remove sputum (mucus), and nutritional support, among others. Some of these treatments can include:

Medications

Medicines used to treat cystic fibrosis help maintain and improve lung function, treat infections, and manage other symptoms and complications. These include:

• Mucus thinners. These medications help loosen and thin secretions so you can cough them up and get rid of them. They are usually inhaled and include Pulmozyme (dornase alfa) and hypertonic saline solution.
• Bronchodilators. These medicines are taken via an inhaler or nebulizer. Bronchodilators relax and widen your airways to make breathing easier. They include Atrovent HFA (ipratropium bromide) and Ventolin HFA (albuterol sulfate).
• Anti-inflammatory medications. Meds such as Advil (ibuprofen) and corticosteroids are used to reduce swelling in the lungs to improve shortness of breath. Inhaled corticosteroids can include QVAR (beclomethasone) and Pulmicort (budesonide).
• Pancreatic enzymes. These oral medications are taken with every meal to help digest and absorb nutrients from your meals and include Creon (pancrelipase).
• CFTR modulators. This treatment works on improving the function of the faulty CFTR gene. They can help improve lung function and reduce complications. Some examples include Kalydeco (ivacaftor) and Orkambi (lumacaftor/ivacaftor).

Some other medications that may be used to help treat complications from CF include antibiotics, laxatives, acid reducers, and insulin.

Airway clearance techniques

These techniques, also called chest physical therapy (CPT), can help loosen and clear mucus from the lungs which reduces infections and improves breathing. They can involve special ways of breathing and coughing, devices used by the mouth, and therapy vests that use vibrations to loosen mucus.

Nutritional therapy for digestive symptoms

Cystic fibrosis can affect how well you digest food and absorb nutrients, so receiving professional dietary guidance can be helpful. A dietitian may recommend:

• Fat-soluble vitamin supplements.
• Eating a high-fat diet to increase absorption of fat-soluble vitamins.
• Eating a high-calorie diet to help with weight gain.
• Extra salt during hot weather or before exercising
• Water to help thin mucus.

Surgery

This option is usually used if you have complications from advanced cystic fibrosis. Your doctor may recommend:

• A lung transplant for those with severe lung disease or respiratory failure.
• A liver transplant for advanced liver disease such as cirrhosis.
• Bowel surgery to remove any blockages in your digestive tract.

What is the best medication for cystic fibrosis?

The best medication for the treatment of cystic fibrosis will depend on the individual’s specific medical cystic fibrosis, medical history, medications that the individual is already taking that may potentially interact with cystic fibrosis medications and the individual’s potential response to the treatment. It is advisable to always speak with your healthcare provider about the best treatment plan for you. The table below includes a list of the most prescribed or over-the-counter cystic fibrosis medications approved by the Food and Drug Administration (FDA).

Best medications for cystic fibrosis

Your healthcare professional will determine the right dosage based on your response to the treatment, medical cystic fibrosis, weight, and age. Other possible side effects may exist; this is not a complete list.

What are the most common side effects of cystic fibrosis medications?

As with all medicines, those used for cystic fibrosis will have some side effects, depending on the class you are taking:

• CFTR modulators’ most common side effect is a headache. They also can cause diarrhea, shortness of breath, and nausea. Caution should be taken if you have liver disease or cataracts as it can worsen your condition.
• Bronchodilators can cause nervousness, insomnia, tremor, nausea, and headache.
• Mucus thinners typically only cause minor side effects such as throat irritation, cough, runny nose, and a bad taste in your mouth.
• Anticholinergics are headache, common cold symptoms, cough, throat irritation, and dry mouth.
• Pancreatic enzymes’ side effects are typically stomach related and include, stomach pain, cramping, diarrhea, and bloating.

What are some home remedies for cystic fibrosis?

Cystic fibrosis is a serious lifelong condition that needs daily care. Along with medications and airway clearance techniques, some daily lifestyle changes will improve your long-term health and help minimize complications.

Cystic Fibrosis prevention

Try some of the following self-care tips to help manage your CF:

• Eat a high-fat diet. A recent study suggests that you should eat a diet high in fat with fat-soluble vitamins to compensate for malabsorption.
• Eat a high-calorie diet. You should also eat a diet high in calories to maintain a healthy weight. According to the Cystic Fibrosis Foundation, women should consume 2500 to 3000 calories a day, while men should consume 3000 to 3700 calories a day. It may also help if you eat more foods containing digestive enzymes such as papaya and pineapple.
• Drink plenty of fluids. It is important to stay hydrated, so make sure you are drinking enough water, especially when the weather is hot.
• Increase your sodium intake. It is also important to replace the sodium lost during sweating due to exercise or living in a hot climate.
• Exercise regularly. Exercise can help keep the mucus in your airways loose and strengthens your heart and lungs.
• Stop smoking. Exposing your lungs to smoke can add to the lung damage caused by the disease.
• Wash your hands regularly. Regular hand washing is effective in protecting yourself from infections.

Frequently asked questions about cystic fibrosis

What are some complications of cystic fibrosis?

With CF affecting most of your major organs, complications will vary based on the affected organs and the severity of the disease. Some complications may include:

• Cystic fibrosis-related diabetes
• Malnutrition
• Intestinal obstruction
• Pancreatitis
• Osteoporosis
• Respiratory failure
• Liver disease
• Infertility, especially in men
• Mental health issues

What is your life expectancy if you have cystic fibrosis?

Life expectancy will vary from person to person and will depend on the severity of the disease. Research estimates that people with CF may reach their 40s before requiring a lung transplant. They will live an average of 8.5 years after receiving a new lung.

Can you exercise if you have cystic fibrosis?

Exercise is beneficial if you have CF. Regular exercise, as tolerated will help loosen mucus and strengthen the heart and lungs.

How early is cystic fibrosis diagnosed?

Most people are diagnosed with CF at birth with newborn screening, or before 2 years of age.

Related resources for cystic fibrosis

• What is cystic fibrosis? Cystic Fibrosis Foundation
• Cystic fibrosis overview. Mayo Clinic
• What is cystic fibrosis? National Heart, Lung, and Blood Institute
• What is cystic fibrosis? WebMD
• Everything you need to know about cystic fibrosis. MedicalNewsToday
• What is cystic fibrosis? Cystic Fibrosis Trust
• Cystic fibrosis. Medlineplus