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Patients with cystic fibrosis and advanced lung disease benefit from lumacaftor/ivacaftor treatment

Pediatric Pulmonology Sep 23, 2020

Ejiofor LCK, Mathiesen IHM, Jensen‐Fangel S, et al. - Via this follow‐up study, researchers examined lumacaftor/ivacaftor's effect on lung function, physical performance, and health‐related quality of life (HRQOL) in a subpopulation of Danish people with cystic fibrosis (CF; PWCF) with advanced pulmonary disease who would not fulfill inclusion criteria for studies assessing safety and efficacy outcomes for lumacaftor/ivacaftor therapy. Inclusion was performed of a total of 21 patients homozygous for the F508del mutation and a median ppFEV1 of 38.7. Per outcomes, lumacaftor/ivacaftor was effective in reducing lung function decline, improving lung function, physical performance, and HRQOL to a greater extent in PWCF with severe lung disease than previously identified.

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