Cystic fibrosis (CF) is the most common, life-shortening inherited disease of Caucasians. Much of its morbidity and mortality is related to progressive airway involvement, the consequence of defective function of the cystic fibrosis transmembrane conductance regulator (CFTR) and associated apical ion channels that lead to impaired mucociliary clearance and innate defenses. The lungs of a person with CF are susceptible to chronic infection that induces an intense inflammatory response, causing airway obstruction, bronchiectasis, and ultimately respiratory failure. Current management of CF incorporates antibiotics, inhaled mucolytics, airway clearance techniques, antiinflammatory agents, and, increasingly, small molecule potentiators and correctors that target specific CFTR mutations. Indeed, treatment for CF lung disease has evolved over the past 5 years, and newer therapies have begun to radically change clinical outcomes. In this chapter, we will review the pathophysiology of CF lung disease and describe current and emerging therapies for this progressive lung disease.
|Title of host publication||Kendig's Disorders of the Respiratory Tract in Children|
|State||Published - Jan 1 2019|
- Cystic fibrosis