FibroScan™ in Pediatric Cholestatic Liver Disease (FORCE)

Arbor Research Collaborative for Health

Status: Completed

Conditions: Alpha1 Anti-Trypsin Deficiency; Portal Hypertension; Alagille Syndrome; Cholestasis; Liver Fibrosis; Biliary Atresia

Treatments: Other: Liver Stiffness Measurement (LSM)

Study type: Observational

Funder types: Other; NIH

Identifiers

NCT02922751

U01DK062456 (U.S. NIH Grant/Contract)

U01DK062500 (U.S. NIH Grant/Contract)

U01DK103135 (U.S. NIH Grant/Contract)

U01DK103149 (U.S. NIH Grant/Contract)

U01DK062453 (U.S. NIH Grant/Contract)

U01DK084538 (U.S. NIH Grant/Contract)

U01DK062470 (U.S. NIH Grant/Contract)

U01DK084575 (U.S. NIH Grant/Contract)

U01DK103140 (U.S. NIH Grant/Contract)

FORCE Study - ChiLDReN Network

U01DK062445 (U.S. NIH Grant/Contract)

U01DK062452 (U.S. NIH Grant/Contract)

U01DK062466 (U.S. NIH Grant/Contract)

U01DK062436 (U.S. NIH Grant/Contract)

U01DK062497 (U.S. NIH Grant/Contract)

U01DK062503 (U.S. NIH Grant/Contract)

U01DK084536 (U.S. NIH Grant/Contract)

U01DK062481 (U.S. NIH Grant/Contract)

Details and patient eligibility

About

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.

Full 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.

Enrollment: 552 patients

Sex: All

Ages: Under 21 years old

Volunteers: No Healthy Volunteers

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

Trial design

552 participants in 1 patient group

All Subjects

Description:

All subjects will be recruited from the Children parent studies: LOGIC (NCT00571272), BASIC (NCT00345553) and PROBE (NCT00061828) and will undergo Liver Stiffness Measurement (LSM). Subjects in these studies have one or more of the following conditions: biliary atresia (BA), Alpha1 Anti-trypsin Deficiency (A1AT) or Alagille Syndrome (ALGS).

Treatment:

Other: Liver Stiffness Measurement (LSM)