Real-world use of ivacaftor in Canada: A retrospective analysis using the Canadian Cystic Fibrosis Registry

Cystic fibrosis (CF) is an autosomal recessive disease that is associated with a significant reduction in life expectancy along with multiple comorbidities including recurrent pulmonary exacerbations, cystic fibrosis related diabetes (CFRD), and pancreatic insufficiency, amongst others. Recently, cystic fibrosis transmembrane conductance regulator (CFTR) modulators have shown remarkable improvements in outcomes. [1 –3] CFTR potentiators are considered a subtype of CFTR modulators. The CFTR potentiator ivacaftor, when used in individuals with CF who carry the G551D mutation, has been shown to increase lung function on average by 10.6% compared to placebo.

Source: Journal of Cystic Fibrosis - March 30, 2021 Category: Respiratory Medicine Authors: Christopher R. Kawala, Xiayi Ma, Jenna Sykes, Sanja Stanojevic, Adele Coriati, Anne L. Stephenson Tags: Original Article Source Type: research