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

Contemp Clin Trials. 2018 Dec:75:67-71. doi: 10.1016/j.cct.2018.11.003. Epub 2018 Nov 5.

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.

Keywords: Biomarkers; Cirrhosis; Exome sequencing; Fatty liver; Natural history.

Publication types

  • Observational Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • 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
  • Non-alcoholic Fatty Liver Disease / metabolism*
  • Prospective Studies
  • Registries*
  • Whole Genome Sequencing

Substances

  • Biomarkers