What is cystic fibrosis (CF)?

From the Guidelines

Cystic fibrosis patients should receive a comprehensive treatment approach, including airway clearance techniques, medication regimens, and nutritional support, to manage symptoms and prevent complications, as recommended by the most recent guidelines 1. The treatment of cystic fibrosis (CF) is a complex and multifaceted process that requires a comprehensive approach to manage symptoms and prevent complications. CF is a genetic disorder that primarily affects the lungs and digestive system by causing the production of thick, sticky mucus, leading to chronic pulmonary infections and inflammation.

Key Components of CF Treatment

• Airway clearance techniques: performed 2-4 times daily, including chest physiotherapy, oscillating positive expiratory pressure devices, or high-frequency chest wall oscillation vests.
• Medication regimens: inhaled bronchodilators like albuterol, mucolytics such as hypertonic saline (3-7%) and DNase (Pulmozyme), and antibiotics for infection management.
• Nutritional support: pancreatic enzyme replacement therapy with meals (typically 500-4,000 lipase units per kg per meal), fat-soluble vitamin supplements (A, D, E, K), and a high-calorie, high-protein diet.

Monitoring and Adjustments

Regular monitoring through pulmonary function tests, sputum cultures, and nutritional assessments is essential to adjust treatments as needed. Early diagnosis through newborn screening and comprehensive care at specialized CF centers has significantly improved life expectancy, which now extends into the 40s and beyond for many patients 1.

Recent Advances in CF Treatment

Recent advances in CF treatment, including the use of CFTR modulators like ivacaftor (Kalydeco), lumacaftor/ivacaftor (Orkambi), tezacaftor/ivacaftor (Symdeko), or elexacaftor/tezacaftor/ivacaftor (Trikafta), have dramatically improved lung function in patients with specific mutations 1.

Importance of Comprehensive Care

Comprehensive care at specialized CF centers is crucial to improve life expectancy and quality of life for CF patients. The MAESTRO committee recommends a standardized approach to lung imaging in CF, including the use of chest radiographs, computed tomography (CT), and chest magnetic resonance imaging (MRI) to monitor disease progression and adjust treatment plans accordingly 1. By prioritizing comprehensive care and staying up-to-date with the latest advances in CF treatment, healthcare providers can significantly improve the morbidity, mortality, and quality of life outcomes for patients with this complex and debilitating disease.

From the Research

Definition and Clinical Manifestations of Cystic Fibrosis

• Cystic fibrosis (CF) is a multiorgan, genetic disease resulting from dysfunction of the CF transmembrane conductance regulator 2.
• The primary clinical manifestations are bronchiectasis, chronic pulmonary infections with recurrent exacerbations, and pancreatic insufficiency 2.

Care and Management of Cystic Fibrosis

• Advances in CF care have led to improved survival, with more than one-half of those affected being adults 2.
• Patients with CF are at risk for numerous conditions that require ICU admission, including respiratory failure, massive hemoptysis, pneumothorax, hepatic failure, and bowel obstruction 2.
• Optimal care of patients with CF requires a multidisciplinary care team that includes respiratory therapists, dieticians, social workers, psychologists, pharmacists, and physicians who have expertise in the treatment of this complex, multisystem disorder 2.

Airway Clearance Techniques for Cystic Fibrosis

• Conventional chest physiotherapy (CCPT) is compared to other airway clearance techniques (ACTs) for CF, including positive expiratory pressure (PEP), extrapulmonary mechanical percussion, active cycle of breathing technique (ACBT), oscillating PEP devices (O-PEP), autogenic drainage (AD), and exercise 3.
• The evidence suggests that CCPT is comparable to alternative ACTs in terms of lung function and respiratory exacerbations, but the certainty of the evidence is very low 3.
• Narrative reports indicate that participants prefer self-administered ACTs, such as PEP, over CCPT 3.

Effects of Albuterol on Lung Function in Cystic Fibrosis

• Albuterol inhalation aerosol has been shown to improve lung function in hospitalized patients with CF, with significant increases in FVC, FEV1, and FEF25-75% compared to placebo 4.
• The response to albuterol was more positive and homogeneous than to placebo, with a greater than 8% change in FEV1 separating changes with albuterol versus placebo with 96% specificity 4.

Meta-Analyses of Physical Therapy Modalities for Cystic Fibrosis

• A meta-analysis of physical therapy modalities for CF found that standard physical therapy resulted in a significantly greater sputum expectoration than no treatment, and that the combination of standard therapy with exercise was associated with a statistically significant increase in FEV1 over standard therapy alone 5.
• Another overview of Cochrane systematic reviews found some evidence to support the inclusion of physical therapies in the care-management plan of CF, including airway clearance and physical training 6.