Journal article
Lumacaftor-ivacaftor effects on cystic fibrosis-related liver involvement in adolescents with homozygous F508 del-CFTR
D Drummond, J Dana, L Berteloot, EK Schneider-Futschik, F Chedevergne, C Bailly-Botuha, T Nguyen-Khoa, M Cornet, M Le Bourgeois, D Debray, M Girard, I Sermet-Gaudelus
Journal of Cystic Fibrosis | Published : 2022
Abstract
Background: The effects of lumacaftor-ivacaftor on cystic fibrosis transmembrane conductance regulator (CFTR)-associated liver disease remain unclear. The objective of the study was to describe the effect of this treatment on features of liver involvement in a cystic fibrosis (CF) adolescent population homozygous for F508del. Methods: Clinical characteristics, liver blood tests, abdominal ultrasonography (US), and pancreas and liver proton density fat fraction (PDFF) by magnetic resonance imaging, were obtained at treatment initiation and at 12 months for all patients. Biomarkers of CFTR activity (sweat chloride test, nasal potential difference, and intestinal current measurement) were asses..
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Funding Acknowledgements
M C is supporterd by Fondation de la Recherche Medicale Grant. E.K.S.-F. is supported by a research grant from The University of Melbourne and the Australian National Health and Medical Research Council (NHMRC) as Biomedical Research Fellow. The content is solely the responsibility of the authors and does not necessarily represent the official views of the Australian National Health and Medical Research Council ISG is supported by research grants from Vaincre La Mucoviscidose, Agence Nationale de la Recherche, Programme Hospitalier de Recherche Clinique, Vertex Research Awards and Filiere de Soins pour la Mucoviscidose. Dominique Debray is supported by research grants from Vaincre La Mucoviscidose. All authors have approved the final manuscript as submitted and agree to be accountable for all aspects of the work.