Observational Study
Copyright ©The Author(s) 2019. Published by Baishideng Publishing Group Inc. All rights reserved.
World J Hepatol. Dec 27, 2019; 11(12): 761-772
Published online Dec 27, 2019. doi: 10.4254/wjh.v11.i12.761
Lumacaftor/ivacaftor therapy is associated with reduced hepatic steatosis in cystic fibrosis patients
Katherine Kutney, Shannon B Donnola, Chris A Flask, Rose Gubitosi-Klug, MaryAnn O’Riordan, Kimberly McBennett, Thomas J Sferra, Beth Kaminski
Katherine Kutney, Rose Gubitosi-Klug, MaryAnn O’Riordan, Kimberly McBennett, Thomas J Sferra, Beth Kaminski, Department of Pediatric Endocrinology, University Hospitals Cleveland Medical Center, Cleveland, OH 44106, United States
Katherine Kutney, Chris A Flask, Rose Gubitosi-Klug, MaryAnn O’Riordan, Kimberly McBennett, Thomas J Sferra, Beth Kaminski, Department of Pediatrics, Case Western Reserve University, Cleveland, OH 44106, United States
Shannon B Donnola, Chris A Flask, Department of Radiology Case Western Reserve University, Cleveland, OH 44106, United States
Chris A Flask, Department of Biomedical Engineering, Case Western Reserve University, Cleveland, OH 44106, United States
Author contributions: Kutney K designed the study, coordinated the research and wrote the manuscript; Donola SB acquired and analyzed MRI images; Flask CA analyzed MRI images and edited the manuscript; Gubitosi-Klug R designed the study and edited the manuscript; O’Riordan M performed statistical analysis; McBennett K designed the study; Sferra TJ designed the study; Kaminski B designed the study and edited the manuscript.
Supported by a grant from the University Hospitals Fellowship Research Award Program (FRAP).
Institutional review board statement: This study was approved by the University Hospitals Cleveland Medical Center Institutional Review Board.
Informed consent statement: All participants gave written informed consent prior to participating in this study.
Conflict-of-interest statement: Flask CA and Kaminski B reports grants from Cystic Fibrosis Foundation, during the conduct of the study. The other authors declare that they have no conflict of interest.
STROBE statement: The authors have read the STROBE statement-checklist of items and the manuscript was prepared and revised according to the strobe guidelines.
Open-Access: This article is an open-access article which was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/
Corresponding author: Katherine Kutney, MD, Assistant Professor, Department of Pediatric Endocrinology, University Hospitals Cleveland Medical Center, 11100 Euclid Ave., Suite 737, Cleveland, OH 44106, United States. katherine.kutney@uhhospitals.org
Telephone: +1-216-8443661 Fax: +1-216-8448900
Received: September 4, 2019
Peer-review started: September 4, 2019
First decision: October 14, 2019
Revised: October 28, 2019
Accepted: November 25, 2019
Article in press: November 25, 2019
Published online: December 27, 2019
Processing time: 112 Days and 11.3 Hours
Core Tip

Core tip: Hepatic steatosis is a common manifestation of liver disease in cystic fibrosis (CF). It remains unknown whether hepatic steatosis contributes to the development of cirrhosis in patients with CF. Lumacaftor/ivacaftor is a cystic fibrosis transmembrane conductance regulator (CFTR) modulator drug targeting the defective chloride channel that causes CF. In this cross-sectional study, CF patients receiving lumacaftor/ivacaftor had significantly lower magnetic resonance imaging proton density fat fractions than CF patients not receiving the CFTR modulator. CFTR modulator use should be included in future studies of CF liver disease.