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

Area of Science:

• Hepatology
• Pediatric Gastroenterology
• Clinical Research

Background:

• Non-alcoholic fatty liver disease (NAFLD) is the most prevalent liver disorder in children.
• NAFLD can progress to severe liver conditions like cirrhosis and liver cancer.
• The natural progression and long-term outcomes of pediatric NAFLD are not well understood, and approved treatments are lacking.

Purpose of the Study:

• To establish a multi-center registry for pediatric NAFLD (EU-PNAFLD).
• To conduct a prospective, observational study on the natural history of pediatric NAFLD.
• To create a foundation for future interventional trials and translational research, including genomics and metabolomics.

Main Methods:

• Establishment of the European Paediatric Non-Alcoholic Fatty Liver Disease Registry (EU-PNAFLD).
• Prospective, observational data collection on pediatric NAFLD patients.
• Creation of a bio-repository for samples to enable translational studies.
• Collaboration with the adult European NAFLD Registry for long-term outcome data.

Main Results:

• The EU-PNAFLD registry is established as a prospective, observational study.
• A bio-repository for sample collection is in place to facilitate translational research.
• The registry will collect data over 20-30 years, aligning with adult NAFLD registry data.

Conclusions:

• The EU-PNAFLD registry will provide crucial insights into the natural history of pediatric NAFLD.
• This large-scale, international cohort will address key knowledge gaps in childhood NAFLD.
• The initiative aims to ultimately benefit children affected by NAFLD through improved understanding and future therapies.