Treatment for Cystic Fibrosis

Treatment for cystic fibrosis (CF) will depend on your child’s symptoms, age, and general health. It will also depend on how severe the condition is.

There is no cure for CF. But research in gene therapy is being done to better understand the disease. Over time, gene therapy research may cure, prevent, or slow the disease's progress. The goals of treatment are to ease the severity of symptoms and slow the progress of the disease. Treatment may include:

• Using CFTR modulator therapies. CFTR (cystic fibrosis transmembrane conductance regulator) is a protein. In people with CF, this protein is missing, deficient, or not formed correctly. There are new CFTR modulator therapies that are designed to correct the function of the defective protein made by the CF gene. These proteins are only appropriate for certain people with CF with certain genetic abnormalities. 

• Managing problems that cause lung dysfunction. This may be done with:

  • Chest physical therapy. This helps loosen and clear lung secretions. Treatment may include postural drainage. It may also include devices that vibrate the chest wall and loosen secretions. Chest physical therapy can be done using the hands. Or it can done mechanically with a vest.

  • Exercise. This helps loosen mucus, stimulate coughing, and improve overall physical condition.

  • Medicines such as bronchodilators, mucus thinners, and anti-inflammatories. These are used to decrease inflammation and swelling, thin the mucus, open airways, and help breathing.

  • Antibiotics. These treat infections.

• Managing digestive problems. This may be done with:

  • Changes to your child's diet

  • Pancreatic enzymes to help digestion

  • Vitamin supplements

  • Treatments for intestinal obstruction

• Getting psychosocial support.  This helps with coping, independence, sterility, sex problems, money issues, and relationships.

As lung disease gets near the end stage, a lung transplant may be an option. The type of transplant done is often a double lung transplant. That’s because leaving the other, sicker lung in place puts it at risk for infection and damage. Not everyone is a candidate for a lung transplant. A liver transplant may be considered for people with advanced liver disease due to cystic fibrosis. Ask your child's healthcare provider for more information.

With medical and psychosocial support, many children and teens with CF can cope well and lead a productive life.