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

Abstract

Abstract Background Several studies have assessed safety and efficacy outcomes for lumacaftor/ivacaftor therapy. We report on lumacaftor/ivacaftor's impact 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 these studies. Methods This follow‐up study examined lumacaftor/ivacaftor's effect in a highly selected CF population. Inclusion criteria included low percent predicted forced expiratory volume in one second (ppFEV 1 ), fast deteriorating ppFEV 1 , low body mass index (BMI), and difficult‐to‐treat infections. Primary endpoints included change in ppFEV 1 slope, cardiopulmonary exercise testing (CPET), and all domains of the Cystic Fibrosis Questionnaire‐Revised (CFQ‐R). Secondary outcomes included change in ppFEV 1 , BMI Z‐score, and sweat chloride concentration. Results A total of 21 patients homozygous for the F508del mutation and a median ppFEV 1 of 38.7 were included. We found significant improvements in ppFEV 1 (+4.2 p < .01, +5.8 p < .01, +4.8 p < .01 and +3.8 p = .03 ppFEV 1 after 3, 6, 9, and 12 months of treatment compared to baseline), ppFEV 1 slope (+6.84 ppFEV 1 /year between the year before and the year after treatment initiation; p = .02), and saturation at CPET initiation (+1.4%, p < .02) and termination (+2.6%, p < .01) after 6 months of treatment. Finally, HRQOL improved significantly in all CFQ‐R domains except Emotion and Treat. Conclusions Our findings suggest that lumacaftor/ivacaftor reduces lung function decline, improves lung function, physical performance, and HRQOL to a greater extent in PWCF with severe lung disease than previously recognized.

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