VALidation of Imaging-based Liver Biomarkers in PEDiatric Patients

Condition(s) studied

Study summary

The goal of this observational (diagnostic validation) study is to validate imaging-based biomarkers for assessing liver involvement in pediatric patients with established or suspected liver disease. The projekt involves 3 larger studies, each with specific aims specified in detailed description below.

The main study (study 1) compares imaging-based biomarkers-with liver biopsy to evaluate their correlation with histologically confirmed liver disease, including fibrosis, inflammation, and steatosis. The main question the overall project aims to answer is:

• Can multiple novel imaging-based biomarkers, such as shear wave elastography (SWE), shear wave dispersion (SWD), and attenuation imaging (ATI), be used for detailed, non-invasive liver characterization in children, and if so, how should they be used?

Eligibility

Primary outcome measure(s)

• Diagnostic accuracy of ultrasound-based liver biomarkers (SWE, SWD, ATI) (STUDY 1) — At time of liver biopsy
  Primary outcome study 1 To evaluate the sensitivity, specificity, and correlation of SWE (for fibrosis), SWD (for inflammation), and ATI (for steatosis) against histologically confirmed liver disease in pediatric patients. Using Area under the ROC curve (AUC), sensitivity, specificity, correlation coefficients
• Longitudinal change in SWE/SWD/ATI biomarkers before and after treatment with CFTR modulators, (Study 2A - Children with Cystic Fibrosis) — baseline (pretreatment) up to 2 years
  Evaluate the effect of CFTR modulator therapy on liver biomarkers. Metric: Change in SWE/SWD/ATI values over time
• Change in liver biomarkers measured by SWE and SWD during routine ultrasound surveillance post-liver transplantation. (Study 2b) — Up to 1 year
  SWE and SWD will be added to conventional ultrasound to assess liver stiffness and viscocity. These imaging biomarkers will be tracked longitudinally to evaluate their utility in improving post-transplant surveillance.
• Detection of early liver damage using SWE, SWD, and ATI (Attenuation Imaging) in children with intestinal failure. (Study 2c) — up to 2 years
  Non-invasive ultrasound biomarkers will be measured during yearly check-ups to identify early signs of liver fibrosis and steatosis in children at risk of IFALD
• Change in SWE/SWD/ATI biomarkers following obesity treatment (GLP-1 analogues or bariatric surgery) (Study2d) — through study completion, an average of 1 years
  Assess whether imaging biomarkers reflect early liver response to obesity interventions. Metric: Change in SWE/SWD/ATI values over time
• Correlation between ultrasound biomarkers and MR biomarkers . (Study 3) — periprocedurally
  SWE/SWD/ATI and MRE will be performed within a 2 week period and compared to assess the relationship between regional and global liver stiffness and steatosis measurements.

Trial sites (1)

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