New blood test at the horizon for one in ten children with common liver disease



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(Glasgow, June 8, 2019) A new blood test could become a clinical practice within the next five years, thus reducing the need for a liver biopsy in the management of fatty liver disease (NAFLD) in pediatrics, as a new major international registry of children's liver collaboration gives preliminary results [1].

The researchers present today the results of the 52nd annual meeting of the European Society of Pediatric Gastroenterology, Hepatology and Nutrition, which indicate that blood tests could replace the need for a biopsy of the liver in children with non-alcoholic fatty liver disease. Currently, if a doctor is concerned about the scarring or inflammation of a child's liver, he may recommend a liver biopsy. The study involved 67 children with NAFLD and revealed that different types of fats in the blood were badociated with the characteristics of fatty liver during liver biopsy, allowing researchers to determine the presence of inflammation and scars, also called steatohepatitis and non-alcoholic fibrosis.

Non-alcoholic fatty liver affects about one in 10 children [2] and is the most common pediatric liver disorder [3]. It can progress to advanced scarring (cirrhosis), liver failure and liver cancer. Despite this, the natural history of the disease is poorly understood and there is currently no approved treatment or drug in clinical trials for children. In response to this, researchers from all over Europe have come together to pool their resources to better understand the disease and how to treat it.

Liver biopsy is currently the most accurate test for NAFLD and the only method commonly used in practice to badess the presence of scarring or inflammation. [4,5]. However, the biopsy is invasive, requires a lot of resources, is expensive, is subject to sampling error and has a low risk of significant complications. [6]. Therefore, the availability of a precise and non-invasive marker to replace the need for liver biopsy, both routinely and in clinical trials, is a major breakthrough for children, parents and children. and health professionals.

To date, all drug trials in Europe [7,8] and badessments of non-invasive markers [9] in the pediatric NAFLD have been monocentric studies, which limits the generalizability of the results. This is particularly relevant given the variation in clinical practice in the different centers.

This research is the first major discovery of the European Register of Pediatric Nonalcoholic Fats (EU-PNALFD register), an international collaboration of 11 specialized and non-specialized centers in six European countries, led by Dr. Jake Mann. . When it is fully operational, the registry will have enrolled up to 2,000 children, including 500 with a biopsy-proven pediatric NAFLD, and follow-up will continue until 30 years of age.

Commenting on the results, Dr. Jake Mann, coordinator of the registry, said:

"We are only at the beginning, but the research results are promising and could help change the way we understand and manage pediatric NAFLD: saving resources, time, and stress for patients." children and their parents The new multicentre registry offers us an opportunity The EU-PNAFLD registry will facilitate recruitment in interventional clinical trials as well as in imaging, biomarker and translational studies, and will enable a better understanding of the long-term natural history of NAFLD, enough to intervene and slow the progression of the disease to reduce the number of patients requiring liver transplantation later in life. "

Alison Taylor, Executive Director of the Children's Liver Disease Foundation (CLDF), added:

"It is good to hear that the registry, funded by CLDF research funds, already provides such fantastic information to improve the diagnosis and management of children affected by NAFLD. to see such a progression in the development of non-invasive techniques. " Tests that will have a positive impact on children, youth and their families at an extremely stressful time. We know that the European register of pediatric non-alcoholic fatty liver diseases (European register EU-PNALFD) will evolve significantly. proud to support this initiative. "

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Notes to editors

For more information, to speak to Dr. Jake Mann or an expert from ESPGHAN, please contact James M. Butcher at [email protected] or call +44 (0) 1444 811,099.

About the expert

Dr. Jake Mann, Wellcome Trust Clinical Research Scientist at the Institute of Metabolic Science, University of Cambridge.

Jake is a pediatric registrar interested in liver fat (fatty liver) and insulin resistance (type 2 diabetes). It focuses particularly on the genetic influences that lead to the development of the spectrum of steatosis and non-alcoholic fatty liver disease (NAFLD).

His current research is:

  • Understand how genetic mutations badociated with NAFLD affect the liver and insulin resistance
  • Blood tests ("serum biomarkers") can be useful in predicting who can progress to more advanced liver disease

Mr Mann is the registry coordinator for the EU-PNALFD register.

About the registry

The European Register of Pediatric Nonalcoholic Fats (EU-PNAFLD) is a multi-center pediatric NAFLD registry that serves as a prospective study on natural history and provides a manageable spine to facilitate recruitment during pregnancy. Later interventional tests. Collection of samples in a biological repository will facilitate translational studies, including genome sequencing and metabolomics. EU-PNAFLD will work closely with the existing European Adult NAFLD Registry to obtain data on clinical outcomes after 20 to 30 years. In the context of a well-characterized large-scale international cohort, the EU-PNAFLD will address key issues relating to pediatric NAFLD and benefit patients with this disease.

The EU-PNAFLD was created with funding from the European Association for the Study of the Liver (EASL).

Jake Mann and ESPGHAN wish to acknowledge the important contribution of the late Professor Valerio Nobili, who unfortunately pbaded away this year and without which the register would not have been possible.

About ESPGHAN

The European Society for Pediatric Gastroenterology in Hepatology and Nutrition (ESPGHAN) is a multiprofessional organization whose aim is to promote children's health by paying particular attention to the gastrointestinal tract, liver and nutrition through the creation of knowledge , dissemination of scientific information. , promoting best practices in the provision of care and providing high quality education to pediatric gastroenterology, hepatology and nutrition professionals in Europe and beyond. For more information, visit http: // www.ESPGHAN.org

About the 52nd Annual Meeting of ESPGHAN

The 52nd Annual Meeting of ESPGHAN will be held from 5 to 8 June 2019 at the SEC in Glasgow, UK.

Each year, the annual ESPGHAN congress attracts more than 4,600 experts and opinion leaders in the field of pediatric gastroenterology, hepatology and nutrition from 100 countries in Europe and five continents , making it the largest conference of its kind in the world.

The 52nd Annual Meeting of the European Society of Gastroenterology, Pediatric Hepatology and Nutrition in Glasgow is supported by the VisitScotland National Conference Application Fund and a grant from the Glasgow Convention Bureau. ESPGHAN would like to thank VisitScotland and the Glasgow Convention Bureau for their support.

For more information on the ESPGHAN congress, including to view the program, go to: https: //www.espghancongress.org /

References

1. Mann, J. et al (2019). European register of pediatric non-alcoholic fatty liver diseases (EU-PNALFD): design and justification. Presented at the 52nd Annual Meeting of ESPGHAN.

2. Anderson, Emma L., et al. "The prevalence of non-alcoholic fatty liver disease in children and adolescents: a systematic review and a meta-badysis." PloS a 10.10 (2015): e0140908.

3. Schwimmer, Jeffrey B., et al. "Prevalence of fatty liver in children and adolescents." pediatrics 118.4 (2006): 1388-1393.

4. P. Vajro, et al., Diagnosis of non-alcoholic fatty liver disease in children and adolescents:
position paper of the ESPGHAN hepatology commission, J. Pediatr. Gastroenterol. Nutr. 54 (2012) 700-713.

5. Mr. Manco, et al., Metabolic syndrome and liver histology in pediatric nonalcoholic steatohepatitis, Int. J. Obes. 32 (2008) 381-387.

6. Z.M. Younossi, et al., World Epidemiology of Non-alcoholic Fatty Liver Disease – Meta-Analytical Assessment of Prevalence, Incidence and Outcomes, hepatology 64 (2016) 73-84.

7. W. Janczyk, et al., Treatment of Omega-3 Fatty Acids in Children with Non-Alcoholic Fatty Liver Disease: Randomized Controlled Trial, J. Pediatr. 166 (2015) 1358-1363.

8. E. Zöhrer, et alEfficacy of docosahexaenoic acid-choline-vitamin E in pediatric NASH: randomized controlled clinical trial, Appl. Physiol. Nutr. Metab (2017) 1-7, https: //do I.org /ten.1139 /APNM-2016-0689.

9. Mr. M. Walenbergh, et al., Plasma levels of cathepsin D: a new tool to predict pediatric liver inflammation, A m. J. Gastroenterol. 110 (2015) 462-470.

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