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

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cam.issuedOnline2018-11-05
dc.contributor.authorMann, Jake P
dc.contributor.authorVreugdenhil, Anita
dc.contributor.authorSocha, Piotr
dc.contributor.authorJańczyk, Wojciech
dc.contributor.authorBaumann, Ulrich
dc.contributor.authorRajwal, Sanjay
dc.contributor.authorCasswall, Thomas
dc.contributor.authorMarcus, Claude
dc.contributor.authorvan Mourik, Indra
dc.contributor.authorO'Rahilly, Stephen
dc.contributor.authorSavage, David B
dc.contributor.authorNoble-Jamieson, Gabriele
dc.contributor.authorLacaille, Florence
dc.contributor.authorDabbas, Myriam
dc.contributor.authorDubern, Béatrice
dc.contributor.authorKelly, Deirdre A
dc.contributor.authorNobili, Valerio
dc.contributor.authorAnstee, Quentin M
dc.contributor.orcidMann, Jake [0000-0002-4711-9215]
dc.contributor.orcidO'Rahilly, Stephen [0000-0003-2199-4449]
dc.contributor.orcidSavage, David [0000-0002-7857-7032]
dc.date.accessioned2018-11-12T15:38:24Z
dc.date.available2018-11-12T15:38:24Z
dc.date.issued2018-12
dc.description.abstractNon-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.
dc.description.sponsorshipFunding 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.
dc.format.mediumPrint-Electronic
dc.identifier.doi10.17863/CAM.32303
dc.identifier.eissn1559-2030
dc.identifier.issn1551-7144
dc.identifier.urihttps://www.repository.cam.ac.uk/handle/1810/284932
dc.languageeng
dc.publisherElsevier BV
dc.publisher.urlhttp://dx.doi.org/10.1016/j.cct.2018.11.003
dc.subjectBiomarkers
dc.subjectCirrhosis
dc.subjectExome sequencing
dc.subjectFatty liver
dc.subjectNatural history
dc.subjectAdolescent
dc.subjectBiomarkers
dc.subjectCarcinoma, Hepatocellular
dc.subjectChild
dc.subjectChild, Preschool
dc.subjectDisease Progression
dc.subjectEurope
dc.subjectHumans
dc.subjectInfant
dc.subjectLiver
dc.subjectLiver Cirrhosis
dc.subjectLiver Neoplasms
dc.subjectMetabolomics
dc.subjectNon-alcoholic Fatty Liver Disease
dc.subjectProspective Studies
dc.subjectRegistries
dc.subjectWhole Genome Sequencing
dc.titleEuropean paediatric non-alcoholic fatty liver disease registry (EU-PNAFLD): Design and rationale.
dc.typeArticle
dcterms.dateAccepted2018-11-04
prism.endingPage71
prism.publicationDate2018
prism.publicationNameContemp Clin Trials
prism.startingPage67
prism.volume75
pubs.funder-project-idWellcome Trust (095515/Z/11/Z)
pubs.funder-project-idEuropean Commission Horizon 2020 (H2020) Societal Challenges (634413)
rioxxterms.licenseref.startdate2018-12
rioxxterms.licenseref.urihttp://www.rioxx.net/licenses/all-rights-reserved
rioxxterms.typeJournal Article/Review
rioxxterms.versionAM
rioxxterms.versionofrecord10.1016/j.cct.2018.11.003
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