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

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  • Linnea Chika Kristensen Ejiofor, Københavns Universitet
  • ,
  • Inger Hee Mabuza Mathiesen, Københavns Universitet
  • ,
  • Søren Jensen-Fangel
  • Hanne Vebert Olesen
  • Marianne Skov, Department of Pediatrics and Adolescent Medicine
  • ,
  • Lue Katrine Drasbæk Philipsen, Københavns Universitet
  • ,
  • Camilla Lundgren Pedersen
  • ,
  • Tacjana Pressler, Københavns Universitet

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 (ppFEV1), fast deteriorating ppFEV1, low body mass index (BMI), and difficult-to-treat infections. Primary endpoints included change in ppFEV1 slope, cardiopulmonary exercise testing (CPET), and all domains of the Cystic Fibrosis Questionnaire-Revised (CFQ-R). Secondary outcomes included change in ppFEV1, BMI Z-score, and sweat chloride concentration. Results: A total of 21 patients homozygous for the F508del mutation and a median ppFEV1 of 38.7 were included. We found significant improvements in ppFEV1 (+4.2 p <.01, +5.8 p <.01, +4.8 p <.01 and +3.8 p =.03 ppFEV1 after 3, 6, 9, and 12 months of treatment compared to baseline), ppFEV1 slope (+6.84 ppFEV1/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.

TidsskriftPediatric Pulmonology
Sider (fra-til)3364-3370
StatusUdgivet - dec. 2020

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