What are the key components of cystic fibrosis (CF) management?

Key Components of Cystic Fibrosis Management

Cystic fibrosis management centers on three fundamental pillars: maintaining airway clearance, controlling infection, and optimizing nutrition, with CFTR modulator therapy now transforming outcomes for up to 90% of genetically eligible patients. 1, 2

Airway Clearance Therapy

Daily airway clearance therapy is essential and should be initiated shortly after CF diagnosis. 2, 3

• Multiple techniques are available including positive expiratory pressure (PEP), active cycle of breathing technique (ACBT), autogenic drainage, oscillating PEP devices, and conventional chest physiotherapy 3
• No single airway clearance technique has proven superior to others; selection should be based on patient preference and ability to perform independently 3
• Self-administered techniques (PEP, ACBT, autogenic drainage) facilitate independence and flexibility compared to conventional chest physiotherapy which requires assistance 3
• Airway clearance must be continued and intensified during respiratory infections—never discontinued 4

Chronic Maintenance Medications

Mucoactive Agents

• Dornase alfa (PULMOZYME) 2.5 mg inhaled once daily is indicated for pediatric and adult CF patients to improve pulmonary function 5
• In patients with FVC ≥40% predicted, daily dornase alfa reduces the risk of respiratory tract infections requiring parenteral antibiotics 5
• Hypertonic saline improves lung function, quality of life, and reduces exacerbations in children aged 6 years and older 4

Anti-inflammatory Therapy

• Chronic azithromycin is recommended for children aged 6 years and older without Pseudomonas aeruginosa to reduce exacerbations 4
• Discontinue azithromycin for at least 2 weeks before collecting sputum for non-tuberculous mycobacterial cultures if NTM disease is suspected 4

CFTR Modulator Therapy

• Highly effective triple combination CFTR modulator therapy (elexacaftor/tezacaftor/ivacaftor) has unprecedented clinical benefits in up to 90% of genetically eligible patients 2
• This breakthrough therapy fundamentally changes prognosis and the therapeutic landscape 2
• Patients on effective CFTR modulators may be candidates for de-escalation of supportive therapies in a sequential approach 6

Infection Management

Pseudomonas aeruginosa

• Regular microbiologic monitoring with respiratory cultures every 6-12 months helps identify new pathogens and reduce chronic P. aeruginosa infections 1, 4
• Aggressive antibiotic treatment of P. aeruginosa infections (oral, inhaled, or intravenous) reduces chronic infection rates 1
• Eradication therapy for incident P. aeruginosa infection is standard practice 1

Non-Tuberculous Mycobacteria

• NTM isolates must undergo molecular identification to species level 1
• Treatment of M. abscessus complex requires an intensive phase (daily oral macrolide plus 3-12 weeks IV amikacin and additional IV antibiotics) followed by continuation phase (oral macrolide, inhaled amikacin, and 2-3 oral antibiotics) 1
• Monotherapy with a macrolide or other antimicrobial should never be used for NTM treatment 1
• Management requires collaboration with NTM and CF experts due to frequent drug intolerance and toxicity 1

Infection Prevention

• Children with CF must avoid direct contact with other CF patients to prevent person-to-person transmission of P. aeruginosa and other pathogens 4
• All routine childhood vaccinations including annual influenza vaccine should be administered 4

Nutritional Management

• Pancreatic enzyme replacement and fat-soluble vitamin supplements are essential for patients with pancreatic insufficiency 1, 7
• High-fat diets (not low-fat) are recommended to offset malabsorption 1
• Optimal nutritional status is a fundamental treatment goal 7

Monitoring and Follow-up

• Schedule outpatient clinic visits every 3-6 months to monitor respiratory status and detect complications early 4
• Pulmonary function testing tracks disease progression 1
• Sweat chloride testing confirms diagnosis (≥60 mmol/L diagnostic for CF) 1

Pulmonary Rehabilitation

• Exercise training improves exercise capacity, strength, and quality of life in CF patients 1
• Walking exercise decreases sputum mechanical impedance, supporting a role in maintaining bronchial hygiene 1
• Physical activity is an essential component of all CF treatment regimens 8

Specialized CF Center Care

Patients should receive care at specialized CF centers offering comprehensive, multidisciplinary approaches including physicians, nurses, physiotherapists, dietitians, and psychosocial support. 1, 8

• CF Foundation accredits a national network of such centers and publishes clinical practice guidelines 1
• The multidisciplinary team is essential for optimal outcomes 8

Important Caveats

• Never dilute or mix dornase alfa with other drugs in the nebulizer, as this could lead to adverse physicochemical changes 5
• Local infection control policies may preclude participation in group exercise programs due to cross-infection risks 1
• People with CF ineligible for CFTR modulators based on genotype or lacking access remain with high unmet medical need 2