Since there is no cure for cystic fibrosis, treatment is focused on easing symptoms (removing excess mucus from lungs), slowing the progression of the disease, and preventing and treating complications, such as preventing and controlling lung infections and intestinal obstruction, and providing adequate nutrition.
Medications used to treat cystic fibrosis include those that target gene mutations, antibiotics, anti-inflammatory drugs (to lessen swelling), mucus-thinning drugs (hypertonic saline), bronchodilators (to keep airways open), oral pancreatic enzymes (to improve nutrient absorption), stool softeners, acid-reduction medications, and if necessary, drugs for diabetes or liver disease.
For some patients with cystic fibrosis, surgery may become necessary, such as nasal and sinus surgery, bowel surgery, or, in severe cases, lung or liver transplantation.
Other therapies may include oxygen therapy, noninvasive ventilation, and feeding tube placement.
Airway clearance – Patients with cystic fibrosis will be taught airway clearance techniques to be used several times a day, known as chest physical therapy (CPT), which include cupping with hands on front and back of chest, breathing and coughing techniques, and the use of machines that pulse air into the lungs (vibrating vest).
Long-term pulmonary rehabilitation – Pulmonary rehabilitation for cystic fibrosis includes exercise, nutritional counseling, supportive counseling, and cystic fibrosis education.
Antibiotics – Inhaled antibiotics may be used to treat lung infections in patients with cystic fibrosis, such as tobramycin (TOBI) and aztreonam (Cayston).
Mucus-thinning medication – The medication dornase alfa (Pulmozyme) may be used to thin the thick mucus associated with cystic fibrosis.
Gene-targeted therapy – Patients with cystic fibrosis who have certain gene mutations may be prescribed cystic fibrosis transmembrane conductance regulator (CTFR) modulators to improve lung function, weight, and to reduce sodium in sweat and include elexacaftor, ivacaftor (Kalydeco), and tezacaftor (Trikafta), the combination of tezacaftor and invacaftor (Symdeko), and combination lumacaftor and ivacaftor (Orkambi).
Surgery – Surgery for patients with cystic fibrosis may be necessary to remove nasal polyps, to treat chronic sinusitis, or to repair an intestinal obstruction. In some select patients with severe lung damage due to cystic fibrosis, lung transplantation may be an option. For patients with cystic-fibrosis liver disease, such as scarring of the liver (cirrhosis), liver transplantation may be an option and can be combined with lung or pancreas transplantation.
Other therapies – Additional therapies for cystic fibrosis may include oxygen therapy to prevent pulmonary hypertension, noninvasive ventilation (CPAP) to assist breathing while sleeping, or the placement of a feeding tube to counteract or prevent malnutrition, vitamin therapy (Vitamins A, D, E, and K), pancreatic enzyme replacement, and diet therapy.