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

Jake P. Mann*, Anita Vreugdenhil, Piotr Soch, Wojciech Jariczyk, Ulrich Baumann, Sanjay Rajwal, Thomas Casswall, Claude Marcus, Indra van Mourik, Stephen O'Rahilly, David B. Savage, Gabriele Noble-Jamieson, Florence Lacaille, Myriam Dabbas, Beatrice Dubern, Deirdre A. Kelly, Valerio Nobili, Quentin M. Anstee

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review

12 Citations (Web of Science)

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.
Original languageEnglish
Pages (from-to)67-71
Number of pages5
JournalContemporary Clinical Trials
Volume75
DOIs
Publication statusPublished - 1 Dec 2018

Keywords

  • Fatty liver
  • Cirrhosis
  • Natural history
  • Exome sequencing
  • Biomarkers
  • CONFERS SUSCEPTIBILITY
  • ADVANCED FIBROSIS
  • YOUNG-ADULTS
  • CHILDREN
  • NAFLD
  • RISK
  • ADOLESCENTS
  • VARIANT
  • STEATOHEPATITIS
  • PREVALENCE

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