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European paediatric non-alcoholic fatty liver disease registry (EU-PNAFLD): Design and rationale.

Lookup NU author(s): Professor Quentin AnsteeORCiD

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This work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License (CC BY-NC-ND).

Abstract

Non-alcoholic fatty liver disease (NAFLD) is the most common liver disorder in children and has the potential to progress to advanced fibrosis/cirrhosis, end-stage liver disease and hepatocellular carcinoma. However, the natural history of the condition is poorly understood and there are no approved treatments. The European Paediatric Non-Alcoholic Fatty Liver Disease Registry (EU-PNAFLD) is a multi-centre registry of paediatric NAFLD that will serve as a prospective, observational, natural history study and provide a tractable back-bone to support recruitment into subsequent interventional trials. Collection of samples into a bio-repository will facilitate translational studies, including genome sequencing and metabolomics. EU-PNAFLD will work closely alongside the existing adult European NAFLD Registry to obtain data on clinical outcomes after 20-30 years. Through an international, well-characterised large-scale cohort, EU-PNAFLD will address the key questions in paediatric NAFLD and benefit patients with the condition.

Publication metadata

Author(s): Mann JP, Vreugdenhil A, Socha P, Janczyk W, Baumann U, Rajwal S, Casswall T, Marcus C, van Mourik I, O'Rahilly S, Savage DB, Noble-Jamieson G, Lacaille F, Dabbas M, Dubern B, Kelly DA, Nobili V, Anstee QM

Publication type: Article

Publication status: Published

Journal: Contemporary Clinical Trials

Year: 2018

Volume: 75

Pages: 67-71

Print publication date: 01/12/2018

Online publication date: 05/11/2018

Acceptance date: 04/11/2018

Date deposited: 19/11/2018

ISSN (print): 1551-7144

ISSN (electronic): 1559-2030

Publisher: Elsevier Inc.

URL: https://doi.org/10.1016/j.cct.2018.11.003

DOI: 10.1016/j.cct.2018.11.003

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