1. European paediatric non-alcoholic fatty liver disease registry (EU-PNAFLD): Design and rationale.
- Author
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Mann JP, Vreugdenhil A, Socha P, Jańczyk 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, and Anstee QM
- Subjects
- Adolescent, Biomarkers metabolism, Carcinoma, Hepatocellular, Child, Child, Preschool, Disease Progression, Europe, Humans, Infant, Liver diagnostic imaging, Liver pathology, Liver Cirrhosis, Liver Neoplasms, Metabolomics, Non-alcoholic Fatty Liver Disease genetics, Prospective Studies, Whole Genome Sequencing, Non-alcoholic Fatty Liver Disease metabolism, Registries
- 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., (Copyright © 2018. Published by Elsevier Inc.)
- Published
- 2018
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