What treatments are available for cystic fibrosis?

[Anonymous]

What treatments are available for cystic fibrosis?

[Genetic Counselor]

There is no cure yet, but treatment has changed dramatically over the past decade. Care now has two parts: treating the effects of thick mucus, and, for many people, treating the faulty CFTR protein itself.

• Airway clearance every day Chest physiotherapy, breathing techniques, and devices that help loosen mucus are the backbone of lung care. Families are taught to do this at home, usually once or twice daily. Regular physical activity also helps clear the airways.
• Inhaled medications Salt-water (hypertonic saline) inhalations and a medication called dornase alfa help thin the mucus so it is easier to cough up. Inhaled antibiotics are used for people with long-term Pseudomonas infection.
• Antibiotics for infections Chest infections are treated early and firmly, by mouth, by inhalation, or through a vein in hospital, to limit lasting lung damage.
• Pancreatic enzyme capsules and nutrition People whose pancreas does not work take enzyme capsules with every meal and snack to digest food. A high-calorie diet, extra salt (especially in hot weather), and supplements of vitamins A, D, E, and K are also part of routine care.
• CFTR modulator medications These tablets work directly on the faulty CFTR protein, helping it fold correctly, reach the cell surface, or open properly. The most widely used combination, elexacaftor with tezacaftor and ivacaftor, can markedly improve lung function, weight, and quality of life, and in the United States it is now approved from 2 years of age. A newer once-daily combination, vanzacaftor with tezacaftor and deutivacaftor, is approved from 6 years of age. Whether these medicines will work depends on the person’s specific CFTR changes, which is one reason genetic testing is so important. In India, access to these medications remains limited and expensive, and families should discuss current options with their treating team.
• Treating complications Diabetes related to cystic fibrosis, liver disease, and bone thinning are treated as they arise. For people with advanced lung disease, lung transplantation may be considered.

Care works best through a specialized cystic fibrosis team, including a lung specialist, dietitian, physiotherapist, and genetic counselor, working together over the long term.

[Author]

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