Childhood Liver Disease Research Network (ChiLDReN): FibroScan™ in Pediatric Cholestatic Liver Disease (FORCE) Study Protocol
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 552
- 试验地点
- 13
- 主要终点
- Compare the distribution of LSM at enrollment between participants with and without portal hypertension
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Cross Sectional
入排标准
- 年龄范围
- — 至 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
结局指标
主要结局
Compare the distribution of LSM at enrollment between participants with and without portal hypertension
时间窗: 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
次要结局
- 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)