European paediatric non-alcoholic fatty liver disease registry (EU-PNAFLD): Design and rationale.
van Mourik, Indra
Kelly, Deirdre A
Anstee, Quentin M
Contemp Clin Trials
MetadataShow full item record
Mann, J., Vreugdenhil, A., Socha, P., Jańczyk, W., Baumann, U., Rajwal, S., Casswall, T., et al. (2018). European paediatric non-alcoholic fatty liver disease registry (EU-PNAFLD): Design and rationale.. Contemp Clin Trials, 75 67-71. https://doi.org/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.
Liver, Humans, Carcinoma, Hepatocellular, Liver Neoplasms, Liver Cirrhosis, Disease Progression, Registries, Prospective Studies, Adolescent, Child, Child, Preschool, Infant, Europe, Metabolomics, Non-alcoholic Fatty Liver Disease, Biomarkers, Whole Genome Sequencing
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.
Wellcome Trust (095515/Z/11/Z)
European Commission Horizon 2020 (H2020) Societal Challenges (634413)
External DOI: https://doi.org/10.1016/j.cct.2018.11.003
This record's URL: https://www.repository.cam.ac.uk/handle/1810/284932