ADULT CYSTIC FIBROSIS

Abstract

Cystic fibrosis (CF) is a monogenic inherited disease affecting 54,000 people in Europe and 32,000 in the United States. CF is characterized by chronic lung infection, exocrine pancreatic insufficiency, male infertility, and co morbidities such as cystic fibrosis-related diabetes or liver disease may also occur. A management strategy including increasing mucociliary clearance, treating lung infections, and introducing CF transmembrane conductance regulator (CFTR) modulators has extended life expectancy in people with CF. Improved survival rates for people with CF have meant that more patients are living into adulthood. The review presents the current understanding of CFTR mutations, disease pathophysiology, management strategy, and the impact of CFTR modulators on both pulmonary and multisystem complications of CF.