Latest Treatment Guidelines For Cystic Fibrosis

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This Article covers everything you need to know about the latest cystic fibrosis treatment, such as medication, treatment guidelines, and the latest updates of U.S drugs for cystic fibrosis.

What’s In The Article?

• What Is Cystic Fibrosis?

• What Are The Medications For Cystic Fibrosis?

• What Are The Cystic Fibrosis Latest Treatment Guidelines?

• What Are the Updates on U.S Drugs For Cystic Fibrosis?

• Final Thoughts

What Is Cystic Fibrosis?

Cystic fibrosis is an inherited chronic disease that affects the lungs and digestive system of both children and adults. This illness causes thick, sticky mucus to build up in a person’s lungs and digestive tract. The mucus clogs the lungs’ airways and traps bacteria in the digestive tract.

What Are The Medications For Cystic Fibrosis?

Currently, these are the drugs used to manage cystic fibrosis and its symptoms:

• Acid-reducing medications

• Antibiotics

• Anti-inflammatory medications

• Bronchodilators

• CFTR correctors

• CFTR potentiators

• Fat-soluble multivitamins

• Mucolytics

• Pancreatic enzyme supplements

• Stool softeners

These are the clinical manifestations that these drugs address:

• Gene mutations to address the most typical type of genetic mutation that leads to cystic fibrosis

• Lung infections

• Airway swelling that leads to narrowing

• Mucus thickening

• Inability of the digestive tract to absorb nutrients effectively

• Bowel obstruction or constipation

• Diabetes

• Liver disease

Depending on the gene mutation that the patient has concerning cystic fibrosis, the physician may prescribe CFTR (cystic fibrosis transmembrane conductance regulator) modulators. These drugs improve symptoms, such as increased salt in sweat and limited lung capacity.

Some of the drug and drug combinations that the U.S. FDA has already approved to target CFTRs include the following:

• Ivacaftor:

  For patients who are at least six months old

• Ivacaftor

  and lumacaftor:

  For patients who are at least 1 year old

• Ivacaftor and tezacaftor:

  For patients who are at least six years old

• Tezacaftor,

  ivacaftor, and elexacaftor:

  For patients aged 6 through 11

What Are The Cystic Fibrosis Latest Treatment Guidelines?

The

• Antibiotics:

  If the patient manifests with acute exacerbation of symptoms, they should be admitted for IV administration of medications. Once the acute exacerbations subside, they must closely adhere to their usual medications while undergoing intensified airway clearance protocols. Currently, there is insufficient evidence to recommend using multiple antibiotics to address the infections that may arise from acute attacks.

• Anti-inflammatory drugs:

  The Cystic Fibrosis Foundation does not recommend using leukotriene modifiers, oral corticosteroids, and inhaled corticosteroids for chronic treatment for reduced worsening of symptoms since there is no evidence to prove that it can improve the patient’s lung function for patients who are below six years old.

Moreover, there is insufficient evidence to support that ibuprofen can improve the patient’s lung function or quality of life in patients 18 years or older.

Patients 6-17 years old may benefit from chronic administration of ibuprofen in high dosages, provided that they have an FEV1 (forced expiratory volume) of more than 60%.

• CFTR modulator treatment:

  The Cystic Fibrosis Foundation released an updated set of guidelines that recommend using ivacaftor for chronic administration in patients at least two years old, provided that they only have one CFTR mutation.

A combination drug, 125 mg ivacaftor and 200 mg lumacaftor, is recommended for patients with a CFTR of Phe508del (two copies) once they turn six years. Patients with this prescription medication should take it with food containing fat.

• Dornase alfa:

  Generally, it is administered through nebulization once or twice a day for patients six years or older.

• Inhaled hypertonic saline:

  This medication is administered via nebulization to ensure hydration of the airways and more effective sputum expectoration. It is one of the treatment choices for patients who are at least six years old.

Hypertonic saline is typically administered with the standard 7% strength. However, it may also be given in a 3% formulation if the patient experiences side effects, such as chest tightness, sore throat, and cough.

What Are the Updates on U.S Drugs For Cystic Fibrosis?

Most recently made available drugs have already shown great results during the Phase III of their respective clinical trials. Most of these medications are designed to address the underlying cause of ineffective CFTR protein production.

Ivacaftor targets the underlying gene defects but has not yet proven effective for patients with F508del mutation. This medication may be used alone or in combination with other similar drugs, such as tezacaftor and elexacaftor.

While the drug combinations are generally effective in managing the patient’s symptoms, they may also bring some side effects, which can include the following:

• Abdominal pain

• Cataracts (in some children and adolescents)

• Diarrhea

• Dizziness

• Headache

• Hepatic impairment

• Nasal congestion

• Nasopharyngitis

• Nausea

• Rashes

Because of these side effects, the patient must undergo a liver function assessment every three months before each round of drug therapy for the first year of treatment. Moreover, they should also undergo eye examinations before and during the proper treatment.

The following are some other drugs that are currently undergoing development and trials:

• ABBV-3067:

  Phase I clinical trials completed; currently in phase II

• ABBV-2222 (GLPG-2222):

  Currently undergoing phase II clinical trials

• PTI-428, PTI-801, and PTI-808:

  Tested as a combination drug; phase II clinical trials completed; ready to proceed to phase III

Final Thoughts

The treatment of cystic fibrosis has mainly focused on slowing the progression of respiratory symptoms. Aside from this, research focuses more on understanding gene mutations, coupled with the ability to identify newborns who are carriers of recessive mutations that cause cystic fibrosis. These have led to a new standard of care for individuals with the disease. The new treatment approaches have enormously impacted survival and quality of life for patients with cystic fibrosis.

Cystic fibrosis tends to be a progressive disease, and treatments for the condition have limitations. However, with strategic treatment, regular follow-up with a lung specialist, and a strong support network, many individuals with this disease can lead healthy, productive lives into adulthood.