Treatment

There is no cure for cystic fibrosis, but treatment can ease symptoms, reduce complications and improve quality of life. Close monitoring and early, aggressive intervention is recommended to slow the progression of CF, which can lead to a longer life.

Managing cystic fibrosis is complex, so consider getting treatment at a center with a multispecialty team of doctors and medical professionals trained in CF to evaluate and treat your condition.

The goals of treatment include:

• Preventing and controlling infections that occur in the lungs
• Removing and loosening mucus from the lungs
• Treating and preventing intestinal blockage
• Providing adequate nutrition

Medications

Options include:

• Medications that target gene mutations, including a new medication that combines three drugs to treat the most common genetic mutation causing CF and is considered a major achievement in treatment
• Antibiotics to treat and prevent lung infections
• Anti-inflammatory medications to lessen swelling in the airways in your lungs
• Mucus-thinning drugs, such as hypertonic saline, to help you cough up the mucus, which can improve lung function
• Inhaled medications called bronchodilators that can help keep your airways open by relaxing the muscles around your bronchial tubes
• Oral pancreatic enzymes to help your digestive tract absorb nutrients
• Stool softeners to prevent constipation or bowel obstruction
• Acid-reducing medications to help pancreatic enzymes work better
• Specific drugs for diabetes or liver disease, when appropriate

Medications that target genes

For those with cystic fibrosis who have certain gene mutations, doctors may recommend cystic fibrosis transmembrane conductance regulator (CTFR) modulators. These newer medications help improve the function of the faulty CFTR protein. They may improve lung function and weight, and reduce the amount of salt in sweat.

The FDA has approved these medications for treating CF in people with one or more mutations in the CFTR gene:

• The newest combination medication containing elexacaftor, ivacaftor and tezacaftor (Trikafta) is approved for people age 12 years and older and considered a breakthrough by many experts.
• The combination medication containing tezacaftor and ivacaftor (Symdeko) is approved for people age 6 years and older.
• The combination medication containing lumacaftor and ivacaftor (Orkambi) is approved for people who are age 2 years and older.
• Ivacaftor (Kalydeco) has been approved for people who are 6 months and older.

Doctors may conduct liver function tests and eye exams before prescribing these medications. While taking these drugs, testing on a regular basis is needed to check for side effects such as liver function abnormalities and cataracts. Ask your doctor and pharmacist for information on possible side effects and what to watch for.

Keep regular follow-up appointments so your doctor can monitor you while taking these medications. Talk to your doctor about any side effects that you experience.

Airway clearance techniques

Airway clearance techniques — also called chest physical therapy (CPT) — can relieve mucus obstruction and help to reduce infection and inflammation in the airways. These techniques loosen the thick mucus in the lungs, making it easier to cough up.

Airway clearing techniques are usually done several times a day. Different types of CPT can be used to loosen and remove mucus, and a combination of techniques may be recommended.

• A common technique is clapping with cupped hands on the front and back of the chest.
• Certain breathing and coughing techniques also may be used to help loosen the mucus.
• Mechanical devices can help loosen lung mucus. Devices include a tube that you blow into and a machine that pulses air into the lungs (vibrating vest). Vigorous exercise also may be used to clear mucus.

Your doctor will instruct you on the type and frequency of chest physical therapy that's best for you.

Pulmonary rehabilitation

Your doctor may recommend a long-term program that may improve your lung function and overall well-being. Pulmonary rehabilitation is usually done on an outpatient basis and may include:

• Physical exercise that may improve your condition
• Breathing techniques that may help loosen mucus and improve breathing
• Nutritional counseling
• Counseling and support
• Education about your condition

Surgical and other procedures

Options for certain conditions caused by cystic fibrosis include:

• Nasal and sinus surgery. Your doctor may recommend surgery to remove nasal polyps that obstruct breathing. Sinus surgery may be done to treat recurrent or chronic sinusitis.
• Oxygen therapy. If your blood oxygen level declines, your doctor may recommend that you breathe pure oxygen to prevent high blood pressure in the lungs (pulmonary hypertension).
• Noninvasive ventilation. Typically used while sleeping, noninvasive ventilation uses a nose or mouth mask to provide positive pressure in the airway and lungs when you breathe in. It's often used in combination with oxygen therapy. Noninvasive ventilation can increase air exchange in the lungs and decrease the work of breathing. The treatment may also help with airway clearance.
• Feeding tube. Cystic fibrosis interferes with digestion, so you can't absorb nutrients from food very well. Your doctor may suggest using a feeding tube to deliver extra nutrition. This tube may be a temporary tube inserted into your nose and guided to your stomach, or the tube may be surgically implanted in the abdomen. The tube can be used to give extra calories during the day or night and does not prevent eating by mouth.
• Bowel surgery. If a blockage develops in your bowel, you may need surgery to remove it. Intussusception, where a segment of intestine has telescoped inside an adjacent section of intestine, also may require surgical repair.

• Lung transplant. If you have severe breathing problems, life-threatening lung complications or increasing resistance to antibiotics for lung infections, lung transplantation may be an option. Because bacteria line the airways in diseases that cause permanent widening of the large airways (bronchiectasis), such as cystic fibrosis, both lungs need to be replaced.

  Cystic fibrosis does not recur in transplanted lungs. However, other complications associated with CF — such as sinus infections, diabetes, pancreas conditions and osteoporosis — can still occur after a lung transplant.

• Liver transplant. For severe cystic fibrosis-related liver disease, such as cirrhosis, liver transplant may be an option. In some people, a liver transplant may be combined with lung or pancreas transplants.