Medical Treatments for Cystic Fibrosis in Children

Our doctors usually prescribe several medications for cystic fibrosis that may improve lung function, clear mucus from the airways, and prevent mucus from trapping bacteria that can cause infections. In rare cases, for children with severe lung damage and decreased lung function, transplantation may be recommended.

Medications

Doctors at Hassenfeld Children’s Hospital at NYU Langone usually prescribe a combination of medications for children with cystic fibrosis that work to open airways and reduce mucus and inflammation.

Medications such as albuterol relax the muscles surrounding the airways, or bronchial tubes. Young children inhale albuterol through a breathing device called a nebulizer, while older children use an inhaler. Inhalers containing nasal steroids decrease swelling and irritation in the airways. Mucolytic medications, which can also be inhaled, help liquefy mucus so that it can be cleared from the lungs. Inhaling a concentrated salt and water mist, known as 7 percent hypertonic saline, draws water into the airways, helping to thin mucus.

Recently, the U.S. Food and Drug Administration (FDA) approved a third medication to treat an underlying cause of cystic fibrosis. Symdeko^™ (tezacaftor/ivacaftor) is a combination oral medication for children age 12 and older. It helps the CFTR proteins work better by allowing more sodium chloride to pass through the body’s cells, improving the balance of salt and water in the lungs and other organs. Two other cystic fibrosis treatments, Kalydeco^® (ivacaftor) and Orkambi^® (lumacaftor/ivacaftor), are also FDA-approved for the treatment of more than 60 percent of children and adults with cystic fibrosis.

Kalydeco^® has been shown to improve lung function, lower sweat chloride levels, and improve weight gain in people with those mutations. Orkambi^® has been approved for children age six and older. Doctors at NYU Langone can help assess whether a child has the specific mutation that would benefit from these drugs.

As a Cystic Fibrosis Foundation–accredited care center, NYU Langone also participates in clinical trials of promising new therapies that may be appropriate for some children. Your doctor can talk to you about whether your child may be a candidate for one of these trials.

Lung Transplantation

For some children with cystic fibrosis, repeated infections and other complications cause severe lung damage. At NYU Langone’s Transplant Institute, our doctors perform lung transplants for adults with severe lung damage and can evaluate your adolescent child and make appropriate referrals, if needed.

Thanks to improved and earlier treatments that increase quality of life, children with cystic fibrosis rarely need lung transplants.