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

• Published in: Contemporary clinical trials 2018-12, Vol.75, p.67-71
• Main Authors: Mann, Jake P., Vreugdenhil, Anita, Socha, Piotr, Jańczyk, Wojciech, Baumann, Ulrich, Rajwal, Sanjay, Casswall, Thomas, Marcus, Claude, van Mourik, Indra, O'Rahilly, Stephen, Savage, David B., Noble-Jamieson, Gabriele, Lacaille, Florence, Dabbas, Myriam, Dubern, Béatrice, Kelly, Deirdre A., Nobili, Valerio, Anstee, Quentin M.
• Format: Article
• Language: English
• Subjects: Biomarkers; Cirrhosis; Exome sequencing; Fatty liver; Natural history
• ISSN: 1551-7144; 1559-2030
• DOI: 10.1016/j.cct.2018.11.003

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.