Management of pulmonary disease in patients with cystic fibrosis

Cystic fibrosis is the most common, life-shortening autosomal recessive disease, affecting approximately 1 in 3400 live births in the United States. Gastrointestinal and pulmonary manifestations are most common. With the introduction of pancreatic enzyme and vitamin supplementation, lung disease accounts for the vast majority of morbidity and mortality in patients with cystic fibrosis. The lungs of cystic fibrosis patients are essentially normal at birth but demonstrate evidence of airway inflammation and infection in early infancy. A vicious cycle of inflammation, infection and obstruction ultimately leads to destruction of airways, impairment of gas exchange and death. Current pharmacological management of pulmonary disease targets reducing airway obstruction, controlling infection and more recently, controlling inflammation. An increased recovery of unusual and highly resistant bacteria from patients with more advanced disease has been observed. Aggressive treatment of acute pulmonary exacerbations with combination antibiotic therapy for two to three weeks has shown pronounced beneficial effects. The routine use of prophylactic antistaphylococcal antibiotics is still controversial. Although current pharmacologic treatment is symptomatic, new agents are being developed and studied that target the underlying defect in the CFTR protein. This review focuses on current pharmacologic management of pulmonary disease in patients with cystic fibrosis and the role of new agents emerging for the treatment of this disease.