Journal article

Lumacaftor-ivacaftor effects on cystic fibrosis-related liver involvement in adolescents with homozygous F508 del-CFTR.

David Drummond, Jérémy Dana, Laureline Berteloot, Elena K Schneider-Futschik, Frédérique Chedevergne, Céline Bailly-Botuha, Thao Nguyen-Khoa, Mathieu Cornet, Muriel Le Bourgeois, Dominique Debray, Muriel Girard, Isabelle Sermet-Gaudelus

J Cyst Fibros | Published : 2021

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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