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

Accepted version
Peer-reviewed

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Type

Article

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Authors

Mann, Jake P 
Vreugdenhil, Anita 
Socha, Piotr 
Jańczyk, Wojciech 
Baumann, Ulrich 

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.

Description

Keywords

Biomarkers, Cirrhosis, Exome sequencing, Fatty liver, Natural history, Adolescent, Biomarkers, Carcinoma, Hepatocellular, Child, Child, Preschool, Disease Progression, Europe, Humans, Infant, Liver, Liver Cirrhosis, Liver Neoplasms, Metabolomics, Non-alcoholic Fatty Liver Disease, Prospective Studies, Registries, Whole Genome Sequencing

Journal Title

Contemp Clin Trials

Conference Name

Journal ISSN

1551-7144
1559-2030

Volume Title

75

Publisher

Elsevier BV
Sponsorship
Wellcome Trust (095515/Z/11/Z)
European Commission Horizon 2020 (H2020) Societal Challenges (634413)
Funding from institutional grants to SOR & DBS from Wellcome Trust UK, to VN from the European Association for the Study of the Liver (EASL), and to JPM from the Children’'s Liver Disease Foundation (CLDF). QMA is supported by the EPoS (Elucidating Pathways of Steatohepatitis) consortium funded by the Horizon 2020 Framework Program of the European Union under Grant Agreement 634413, an EASL Registry Grant and the Newcastle NIHR Biomedical Research Centre.