Abstract
In cystic fibrosis (CF), absent or dysfunctional CF transmembrane conductance regulator
(CFTR) on the surface of airway epithelial cells causes abnormal mucociliary clearance,
leading to chronic endobronchial infection and inflammation, in turn resulting in
life-shortening progressive obstructive lung disease and structural airway damage.
Fortunately, CF-specific therapies have been developed that improve lung function
and reduce pulmonary exacerbations, contributing significantly to improved survival
over the past 4 decades. Therapies not originally developed for CF, such as bronchodilators
and corticosteroids, are also widely used by people living with CF. Therapies to be
reviewed in this article include mucolytics, airway surface liquid hydrators, anti-inflammatory
medications, bronchodilators, inhaled and oral antibiotics, and airway clearance techniques.
Determining which therapies to utilize can be challenging, as there is variable evidence
for each treatment, differing national guidelines, few head-to-head studies, potential
for drug–drug interactions, and synergistic toxicities, as well as issues with burden
of care. In this review, we summarize the mechanism of action and available evidence,
and compare national guidelines for each major medication used to treat the airway
consequences of CFTR dysfunction.
Keywords
cystic fibrosis - therapies - pulmonary
