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

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

Author information

1
Metabolic Research Laboratories, Institute of Metabolic Science, University of Cambridge, Cambridge, UK; Department of Paediatrics, University of Cambridge, Cambridge, UK. Electronic address: jm2032@cam.ac.uk.
2
Department of Pediatrics, Maastricht University Medical Centre, Maastricht, the Netherlands.
3
Department of Gastroenterology, Hepatology, Nutritional Disorders and Paediatrics, Children's Memorial Health Institute, Warsaw, Poland.
4
Division of Pediatric Gastroenterology and Hepatology, Department of Pediatric Kidney, Liver, and Metabolic Diseases Hannover, Hannover Medical School, Germany; The Liver Unit, Birmingham Children's Hospital, Birmingham, United Kingdom.
5
Children's Liver Unit, Leeds Teaching Hospitals NHS Trust, Leeds Children's Hospital, Leeds, United Kingdom.
6
Department of Paediatrics, Karolinska University Hospital, CLINTEC, Karolinska Institute, Stockholm, Sweden.
7
Division of Pediatrics, Department of Clinical Science, Intervention and Technology, Karolinska Institutet, Stockholm, Sweden.
8
The Liver Unit, Birmingham Children's Hospital, Birmingham, United Kingdom.
9
Metabolic Research Laboratories, Institute of Metabolic Science, University of Cambridge, Cambridge, UK.
10
Department of Paediatric Gastroenterology, Addenbrookes Hospital, Cambridge, UK.
11
Gastroenterology-Hepatology-Nutrition Unit, Hôpital Universitaire Necker-Enfants maladies, Paris, France.
12
Nutrition et Gastroentérologie Pédiatriques, Hopital Armand-Trousseau, Paris, France.
13
Department of Pediatrics, University "La Sapienza", Rome, Italy; Hepatology, Gastroenterology, and Nutrition, Bambino Gesu' Hospital, Rome, Italy.
14
Institute of Cellular Medicine, Faculty of Medical Sciences, Newcastle University, Newcastle Upon Tyne, United Kingdom; Liver Unit, Newcastle Upon Tyne Hospitals NHS Foundation Trust, Freeman Hospital, Newcastle upon Tyne, United Kingdom.

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

PMID:
30408605
DOI:
10.1016/j.cct.2018.11.003

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