Cystic Fibrosis: Recent Successes Present New Challenges

Remarkable progress has been made in the care and sur-vival of patients with cystic fibrosis (CF). First de-scribed in 1938 as a life-shortening disease characterized by cystic changes of the pancreas, CF is now known to be caused by any of many known mutations in a single gene, resulting in abnormal function of a chloride channel and multisystem disease (1). Before the development of pancre-atic enzymes and nutritional supplements, most patients with CF died early from malnutrition related to pancreatic insufficiency. After this early threat became more con-trollable, progressive pulmonary disease characterized by chronic airway infection, inflammation, bronchiectasis, and respiratory failure emerged as the predominant cause of morbidity and mortality. Today, with supportive thera-pies targeting mucus obstruction and infections, most pa-tients survive to adulthood and CF is no longer strictly