A new blood test that will reduce the need for a liver biopsy in the management of paediatric Non-Alcoholic Fatty Liver Disease (NAFLD) could be ready within five years, finds international paediatric liver registry collaboration early results presented at the 52nd Annual Meeting of the European Society of Paediatric Gastroenterology, Hepatology and Nutrition in Glasgow.(1)
Non-Alcoholic Fatty Liver Disease affects around one in 10 children (2) and is the most common paediatric liver disorder.(3) It can progress to advanced scarring (cirrhosis), liver failure, and liver cancer. Despite this, the natural history of the condition is poorly understood and there are currently no approved treatments or drugs in clinical trials for children. In response to this, researchers from across Europe have come together to pool resources in order to better understand the condition and how to treat it.
Liver biopsy is currently the most accurate test for NAFLD and the only method routinely used in practice for assessing the presence of scarring or inflammation.(4,5) However, biopsy is invasive, resource intensive, costly, prone to sampling error and carries a risk of significant complications. (6) Therefore, the availability of an accurate and non-invasive marker to replace the need for liver biopsy, both in routine practice and in a clinical trial setting, is a major breakthrough for children, parents and healthcare professionals.
To date, all European drug trials(7,8) and assessments of non-invasive markers(9) in paediatric NAFLD have been single-centre studies, which limits the generalisability of findings. This is particularly pertinent given the variation in clinical practice at different centres.
The study looked at 67 children with NAFLD and found that different types of fats in the blood were associated with features of fatty liver on liver biopsy, allowing researchers to determine the presence of inflammation and scarring - also known as non-alcoholic steatohepatitis and fibrosis.
This research is the first major finding to be reported from the European Paediatric Non-Alcoholic Fatty Liver Disease Registry (EU-PNALFD registry), an international collaboration of 11 specialist and non-specialist centres in six European countries. When fully operational, the registry will have enrolled up to 2,000 children, including 500 with biopsy-proven paediatric NAFLD, and follow-up will continue for up to 30 years.
References:
- Mann, J., et al (2019). European Paediatric Non-Alcoholic Fatty Liver Disease (EU-PNALFD) Registry: design and rationale. Presented at the 52nd Annual Meeting of ESPGHAN.
- Anderson, Emma L., et al. "The prevalence of non-alcoholic fatty liver disease in children and adolescents: a systematic review and meta-analysis." PloS one 10.10 (2015): e0140908.
- Schwimmer, Jeffrey B., et al. "Prevalence of fatty liver in children and adolescents." Pediatrics 118.4 (2006): 1388-1393.
- P. Vajro, et al., Diagnosis of nonalcoholic fatty liver disease in children and adolescents: position paper of the ESPGHAN Hepatology Committee, J. Pediatr. Gastroenterol. Nutr. 54 (2012) 700-713.
- M. Manco, et al., Metabolic syndrome and liver histology in paediatric non-alcoholic steatohepatitis, Int. J. Obes. 32 (2008) 381-387.
- Z.M. Younossi, et al., Global epidemiology of nonalcoholic fatty liver disease--Meta-analytic assessment of prevalence, incidence, and outcomes, Hepatology 64 (2016) 73-84.
- W. Janczyk, et al., Omega-3 Fatty acids therapy in children with nonalcoholic Fatty liver disease: a randomized controlled trial, J. Pediatr. 166 (2015) 1358-1363.
- E. Zöhrer, et al., Efficacy of docosahexaenoic acid-choline-vitamin E in paediatric NASH: a randomized controlled clinical trial, Appl. Physiol. Nutr. Metab (2017) 1-7, https://doi.org/10.1139/apnm-2016-0689.
- S.M. Walenbergh, et al., Plasma cathepsin D levels: a novel tool to predict pediatric hepatic inflammation, Am. J. Gastroenterol. 110 (2015) 462-470.