Management

• All patients with CF require periodic follow-up with a multidisciplinary team for specialized management.

• Management should include the following goal-directed interventions:

  • Preservation of lung function

    • Pharmacological and nonpharmacological interventions

    • Prevention of infection and reduction of exacerbations

  • Optimization of nutrition

  • Screening and monitoring for comorbidities and complications

• Patients with certain mutations may benefit from treatment with CFTR modulators.

• Acute pulmonary exacerbations require rapid and effective treatment.

Lung function is preserved by combining pharmacological and nonpharmacological interventions to improve mucociliary clearance, reduce mucus viscosity, and mobilize secretions.

Pharmacological interventions

• High-dose ibuprofen: can slow the progression of lung disease

• Bronchodilators: SABA (e.g., albuterol), LABA (e.g., salmeterol)

• Mucolytics

  • Hypertonic saline nebulization (6–7% NaCl): mucociliary and osmotic effect that can improve mucociliary clearance and thin the mucus

  • Dornase alfa, aerosolized : a recombinant DNase that thins the mucus by breaking down extracellular DNA in sputum [30]

• Other therapies

  • N-acetylcysteine: efficacy is unproven in CF [20][29]

  • Corticosteroids (inhaled and systemic): insufficient evidence; not routinely used

• Airway clearance techniques: a mainstay of CF treatment that loosens and mobilizes mucus secretions

  • Conventional chest physiotherapy (CPT): postural drainage with percussion and/or clapping

  • Alternative airway clearance methods

    • High-frequency chest compression

    • Airway oscillating devices

    • Positive expiratory pressure devices

  • Huff coughing

• Exercise (e.g., swimming, jogging, cycling)

Patients with CF benefit from a regular airway clearance routine that combines pharmacological measures with airway clearance techniques to preserve lung function and reduce symptoms.

• Pulmonary exacerbations in patients with CF are often triggered by chronic lung infections of pathogenic organisms.

• Eradication and/or suppression regimens can prevent exacerbations and improve lung function.

• Consider treatment after early detection of relevant pathogens during routine surveillance sputum cultures.

• Eradication regimens include:

  • P. aeruginosa: inhaled tobramycin

  • MRSA: inhaled vancomycin PLUS oral antibiotics , PLUS extrapulmonary eradication

Antibiotic eradication and suppression regimens for patients with CF should always be selected in consultation with a specialist.

There is a direct correlation between BMI and pulmonary lung function in patients with CF. The following are common dietary recommendations for patients with CF:

• High-energy diet to compensate for increased demand (target BMI ≥ 50thpercentile) [20][33]

• Pancreatic enzyme supplements [10]

• Additional NaCl intake [33]

• Oral supplementation of fat-soluble vitamins (ADEK)

A relatively new therapy for the long-term management of CF that targets specific defects in the CFTR protein to improve its function.

• Indications

  • Approved for patients with specific CFTR mutations (e.g., ΔF508, G511D mutations)

  • Their use can potentially reduce CF complications and comorbidities

• Mechanism of action: improve CFTR protein function by targeting underlying protein defects [29]

  • Potentiators (e.g., ivacaftor): increase CFTR Cl- channel gate opening and conductance and improve Cl- transport

  • Correctors (e.g., lumacaftor, tezacaftor, elexacaftor): improve protein folding, protein stability, and the transport of functional CFTR protein to the cell surface [35]

• Combination therapy

  • Two or more CFTR modulators with different mechanisms of action can be combined to synergistically improve CFTR protein quantity and function.

  • Combination therapy increases the yield of CFTR modulators and can therefore benefit larger numbers of patients. [10]

Perform CFTR genetic testing in all patients diagnosed with CF to assess for specific mutations that can be targeted by CFTR modulators.

CFTR modulators can improve FEV1, help patients gain weight, reduce exacerbations, and decrease sweat Cl- concentration.

• Oxygen therapy: frequent oxygen monitoring and support for chronic respiratory insufficiency as needed

• Double-lung transplant

  • A treatment option for patients with end-stage lung disease [20][36]

CF is a chronic, progressive, and ultimately life-shortening condition. Preferably, CF should be managed by a multidisciplinary team that can provide comprehensive care, which emphasizes shared decision-making.