Emerging New Diagnostic Modalities and Therapies of Nonalcoholic Fatty Liver Disease
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PEDIATRIC GASTROENTEROLOGY (S ORENSTEIN AND S KHAN, SECTION EDITORS)
Emerging New Diagnostic Modalities and Therapies of Nonalcoholic Fatty Liver Disease Chuan-Hao Lin 1
&
Rohit Kohli 1
# Springer Science+Business Media, LLC, part of Springer Nature 2020
Abstract Purpose of Review Nonalcoholic fatty liver disease (NAFLD) has emerged as the leading cause of chronic liver disease in both adults and children. In this article, we review recent developments in the screening, diagnosis, and treatment of pediatric NAFLD. Recent Findings Although alanine aminotransferase (ALT) remains the best screening test for NAFLD in children, and liver biopsy is still required for the diagnosis of nonalcoholic steatohepatitis (NASH), other noninvasive biomarker/imaging studies (MRI-PDFF and VCTE) have emerged as diagnostic methods for pediatric NAFLD. Two large clinical therapeutic trials testing vitamin E, metformin, and cysteamine in pediatric NAFLD yielded mostly inconclusive results. Bariatric surgery has begun to be used in adolescents with severe obesity. An adult phase 2 study using obeticholic acid (OCA) to treat NASH patients with fibrosis showed some positive results. As we continue to await the first FDA-approved therapeutic agent for NASH, lifestyle change remains the main modality of treatment. Summary Newer diagnostic and treatment modalities for pediatric NAFLD continue to be in development under FDA guidance. Keywords Nonalcoholic fatty liver disease . Alanine aminotransferase (ALT) . Vibration-controlled transient elastography (VCTE) . MRI-estimated liver proton density fat fraction (PDFF) . Obeticholic acid (OCA)
Introduction Nonalcoholic fatty liver disease (NAFLD) is a chronic liver disease with excessive fat accumulation in the liver. NAFLD is defined as (1) evidence of abnormally increased fat (≥ 5% hepatic steatosis, either by imaging or histology), and (2) lack of secondary causes of fat accumulation such as significant alcohol consumption, chronic viral hepatitis, long-term use of a steatogenic medication, or monogenic hereditary disorders [1]. NAFLD includes a spectrum of diseases, ranging from nonalcoholic fatty liver (NAFL) to the aggressive nonalcoholic steatohepatitis (NASH), fibrosis, and eventually cirrhosis [2]. NAFL is defined as the presence of ≥ 5% hepatic steatosis without evidence of significant inflammation or Topical Collection on Pediatric Gastroenterology * Chuan-Hao Lin [email protected] 1
Division of Gastroenterology, Hepatology and Nutrition, Children’s Hospital Los Angeles, and the Department of Pediatrics, Keck School of Medicine, University of Southern California, 4650 Sunset Boulevard, Mail Stop 78, Los Angeles, CA 90027, USA
hepatocellular injury. NASH is defined as the presence of steatosis with inflammation and/or hepatocyte injury, such as ballooning, with or without liver fibrosis [1]. Pediatric NAFLD is defined as chronic hepatic steatosis in children (18 years or younger). Due to the epidemic of obesity and type 2 diabetes, NAFLD has emerged as the leading cause of chron
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