European Paediatric NAFLD Registry (EU-PNAFLD)
Research type
Research Study
Full title
European Paediatric NAFLD Registry (EU-PNAFLD): a prospective, longitudinal follow-up of children with non-alcoholic fatty liver disease.
IRAS ID
174534
Contact name
David Savage
Contact email
Sponsor organisation
Cambridge University Hospitals NHS Foundation Trust and the University of Cambridge
Duration of Study in the UK
30 years, 0 months, 0 days
Research summary
Research Summary
Non-alcoholic fatty liver disease (NAFLD) is the most common liver disease affecting children. It is most often caused by obesity, which results in the build-up of fat within the liver. In some children this fat causes inflammation and scarring that can eventually lead to cirrhosis (a shrunken, very scarred liver) and liver failure. It is not known which children will develop clinically significant liver disease and in what time course it will develop. As adults the children will also be at high risk of type 2 diabetes and heart disease, which is likely to be the main cause of death. This study will look into what the long-term health complications are for children with fatty liver disease, whether we can identify those at high risk of severe disease, look for new rare genes involved in fatty liver, and will provide a registry for recruitment into further trials in the future.
The purpose of this study is:
a) to establish the natural history of paediatric NAFLD;
b) to assess the clinical effectiveness of management and quality of care;
c) to provide a register of patients for recruitment to intervention studies or genotype and biomarker studies.It is a purely observational study where we will collect data from routine clinic appointments. No additional study visits are required.
Data will be stored on a nationalised, secure online Clinical Data Management System.
Data held in the database will be pseudoanonymised and will be linked by a non-identifiable study ID number to the health professional who submitted the data. The local health professional can submit data records on his/her patients, and controls access to data records for patients under their care.
The detailed clinical characterisation will allow future enrolment into clinical trials (with separate consent) and will encourage international collaborations for patient benefit.
Summary of Results
This study aimed to help us understand the long-term history of children with fatty liver disease. Unfortunately, due to interruptions of recruitment from COVID-19 and difficulties obtaining legal agreements between participating sites, we have had to shut the study before we were able to obtain enough data to get meaningful results about the long-term history of fatty liver disease. However, we were able to do two small studies looking at the genetics of fatty liver disease and how it affects fats in the blood. We found that some common gene changes ('variants') protect against development of fatty liver. We also found that some patterns of fats in the blood are linked to more severe fatty liver. These results help us understand which children get severe fatty liver so we can know who needs targeted treatment.
REC name
East Midlands - Nottingham 2 Research Ethics Committee
REC reference
17/EM/0084
Date of REC Opinion
29 Mar 2017
REC opinion
Further Information Favourable Opinion