Childhood Liver Disease Research Network (ChiLDReN): FibroScan™ in Pediatric Cholestatic Liver Disease (FORCE) Study Protocol
Overview
- Phase
- Not Applicable
- Intervention
- Not specified
- Conditions
- Biliary Atresia
- Sponsor
- Arbor Research Collaborative for Health
- Enrollment
- 552
- Locations
- 13
- Primary Endpoint
- Compare the distribution of LSM at enrollment between participants with and without portal hypertension
- Status
- Completed
- Last Updated
- last year
Overview
Brief Summary
Noninvasive monitoring of liver fibrosis is an unmet need within the clinical management of pediatric chronic liver disease. While liver biopsy is often used in the initial diagnostic evaluation, subsequent biopsies are rarely performed because of inherent invasiveness and risks. This study will evaluate the role of non-invasive FibroScan™ technology to detect and quantify liver fibrosis.
Detailed Description
Noninvasive 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. 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.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Age less than 21 years at the time of enrollment
- •Participants enrolled in a ChiLDReN based prospective observational cohort study (PROBE, BASIC, or LOGIC)
- •Willingness and ability to participate in the study for up to 24 months
- •One of the following three diagnoses
- •Biliary atresia per ChiLDReN criteria or,
- •Alpha-1 antitrypsin deficiency (PiZZ or SZ) or,
- •Alagille Syndrome per ChiLDReN criteria
Exclusion Criteria
- •BA with known situs inversus or polysplenia/asplenia
- •Presence of clinically significant ascites detected on physical examination
- •Open wound near expected FibroScan probe application site
- •Use of implantable active medical device such as a pacemaker or defibrillator
- •Known pregnancy
- •Prior liver transplant
- •Unable or unwilling to give informed consent or assent
Outcomes
Primary Outcomes
Compare the distribution of LSM at enrollment between participants with and without portal hypertension
Time Frame: Enrollment
A linear model will be fit to FibroScan™ values at enrollment to assess the impact of portal hypertension on LSM, controlling for important covariates such as age, gender, and race
Secondary Outcomes
- FibroScan™ LSM values at enrollment and conventional laboratory determinants of liver disease ((Pediatric End Stage Liver Disease (PELD) and APRI (Aspartate Aminotransferase (AST) to Platelet Ratio Index)).(Baseline)
- Change in Liver Stiffness Measurement (LSM) obtained via transient elastography from baseline to LSM at the Year 1 and Year 2 visits in participants with biliary atresia (BA).(Baseline, Year 1 Visit, Year 2 Visit)
- Number of participants in whom a valid FibroScan™ LSM can be obtained(Baseline, Year 1 Visit, Year 2 Visit)