FibroScan in Pediatric Cholestatic Liver Disease

Non-invasive monitoring of liver fibrosis is an unmet and critical need within the clinical management of children with chronic liver disease. While liver biopsy is often used in the initial diagnostic evaluation of children with liver disease, subsequent surveillance liver biopsy is rarely performed in children because of its inherent invasiveness and risks. Therefore, our understanding of the natural history of fibrosis progression in children is limited. The patchy nature of fibrosis in many important pediatric liver diseases (e.g., biliary atresia (BA) and cystic fibrosis liver disease (CFLD)) limits the utility of sequential liver biopsy even if it were to be employed in clinical practice in pediatrics. Thus, non-invasive means of assessing liver fibrosis throughout the liver would be highly desirable and clinically useful in pediatric hepatology. The Childhood Liver Disease Research Network (ChiLDReN) is poised and uniquely qualified to conduct a comprehensive longitudinal assessment of the utility of FibroScan™-specific elastography, liver stiffness measurement (LSM) as a measure of hepatic fibrosis in children with serious chronic cholestatic liver disease.

The FibroScan in Pediatric Cholestatic Liver Disease (FORCE) study, a natural history study within ChiLDReN, was a cross-sectional and longitudinal assessment of the utility of LSM in children with chronic cholestatic liver disease. Study participants were from 13 ChiLDReN sites in the U.S. and Canada, and were also enrolled in the PROBE, BASIC, or LOGIC studies. FORCE participants were evaluated for a period of up to 24 months to assess the non-invasive ultrasound tool (FibroScan™) to detect and quantify global liver fibrosis in children with biliary atresia (BA), alpha-1 antitrypsin deficiency (A1ATD), and Alagille syndrome (ALGS). The participants were non-fasted and non-sedated during data collection. There were three visits in the study: baseline, 12-month follow-up, and 24-month follow-up. Clinical data and biosamples were collected at each visit along with repeated FibroScan measurements such as liver stiffness measurements (LSM) to quantify liver fibrosis, and controlled attenuation parameter (CAP) to quantify liver steatosis.

Baseline data are available from the Repository.