'Liquid Biopsy' for Pediatric Fatty Liver Disease on the Horizon

By Megan Brooks

June 12, 2019

NEW YORK (Reuters Health) - A blood test could replace the need for liver biopsy in the management of non-alcoholic fatty liver disease (NAFLD) in children within five years, European researchers predict.

They studied 67 children (mean age 10.6 years) with NAFLD diagnosed by liver biopsy, including two-thirds with non-alcoholic steatohepatitis (NASH). Children with NASH were older (8.6 vs 11.4 years), but no other baseline anthropometric or biochemistry measurement differentiated steatosis from NASH.

They found that several different types of blood fats were associated with features of fatty liver on liver biopsy and correlated with the presence of NASH and fibrosis.

"The blood test is high-throughput lipidomics, also known as lipid profiling," Dr. Jake Mann, Wellcome Trust Clinical Research Fellow at the Institute of Metabolic Science, University of Cambridge, explained in email to Reuters Health. "All the patients included so far have fatty liver (and many) of the lipids were associated with more severe/advanced fatty liver disease."

All associations between lipid species and histology were independent of genotype, total lipids, BMI, and insulin resistance, Dr. Mann noted.

He reported the results June 8 at the annual meeting of the European Society of Paediatric Gastroenterology, Hepatology and Nutrition (ESPGHAN) in Glasgow, Scotland.

"It is early days but the results of the research are promising and could help shift the way we understand and manage paediatric NAFLD: saving resources, time, and stress for children and their parents," Dr. Mann said in a news release.

"Our next step is to compare the results to children without fatty liver, but we expect there will be enormous differences between healthy children and those with fatty liver," he told Reuters Health.

NAFLD is the most common pediatric liver disorder affecting about 1 in 10 children. It can progress to cirrhosis, liver failure, and liver cancer. Yet, the natural history NAFLD is poorly understood and there are currently no approved treatments or drugs in clinical trials for children.

Dr. Mann's study is part of the European Pediatric Non-Alcoholic Fatty Liver Disease Registry (EU-PNALFD registry), an international collaboration of 11 specialist and non-specialist centers in six European countries, which he leads. When fully operational, the registry will have enrolled up to 2,000 children, including 500 with biopsy-proven pediatric NAFLD, and follow-up will continue for up to 30 years.

"The EU-PNAFLD registry will facilitate recruitment into interventional clinical trials as well as imaging, biomarker, and translational studies, plus allow greater understanding of the long-term natural history of NAFLD. The ultimate aim is to understand the condition sufficiently to intervene and slow disease progression so we can reduce the number of patients requiring liver transplantation later in life," Dr. Mann said in the release.

"It is great to hear that the registry, supported through CLDF research funds, is already yielding such fantastic insights to improve the diagnosis and care of children affected by NAFLD," Alison Taylor, Chief Executive of the Children's Liver Disease Foundation (CLDF), commented in the release. "In particular, it is wonderful to see such progression in the development of non-invasive tests which will positively impact children, young people and their families at a time that is incredibly stressful. We know that further significant developments in care will come via the EU-PNALFD registry and we are proud to be supporting the initiative."

Funding for creation of the EU-PNAFLD was provided by the European Association for the Study of the Liver (EASL). Support for the current study was provided by a grant from the UK Children's Liver Disease Foundation.

SOURCE: https://www.espghancongress.org/

ESPGHAN 2019.

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