Drug treatment of cystic fibrosis

Abstract

Cystic fibrosis is the most common life-limiting autosomal recessive condition in Australia. A defect in the cystic fibrosis transmembrane conductance regulator protein affects chloride transport across epithelial cells. Patients with cystic fibrosis produce thick sticky mucus. This causes problems in multiple organs, particularly the lungs. Cystic fibrosis modulator therapies can partially correct the underlying pathophysiology and improve chloride transport, thereby improving morbidity. Life expectancy is improving, so many patients are now developing chronic diseases associated with ageing. All health professionals should be aware that the cystic fibrosis modulator therapies are metabolised via cytochrome P450 pathways in the liver. There are therefore significant drug-drug interactions with medicines metabolised by the same pathways.

Keywords: cystic fibrosis transmembrane conductance regulator; elexacaftor; ivacaftor; lumacaftor; tezacaftor.