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?
all patients scheduled for a clinically liver biopsy at the Queen Silvia Children's Hospital
any form of suspected or confirmed liver disease
age between 0-18 years
consent by the child/parents to participate.
technical limitations regarding the measurements
inability to fully understand the informed consent documents.
All participants are children at Queen Silvias Children's hospital, meeting the eligible criteria Study 1-(main study): All children referred for a liver biopsy Study 2a: All children with cystic fibrosis Study 2b: All children liver transplanted Study 2c: All children monitored for intenstinal failiure Study 2d: All children treated with either bariatric surgery or novel appetite-regulating drugs (GLP1 analogues) Study 3: Children who are participating in study 1 and 2, in whom MRI is clinically indicated as a supplement to ultrasound examination - for example, children with obesity for whom ultrasound may be insufficient
Other
Prospective
Not listed
Diagnostic accuracy of ultrasound-based liver biomarkers (SWE, SWD, ATI) (STUDY 1)
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)
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)
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)
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
Association between imaging biomarkers and blood liver markers and demographic variabels (Study 1)
Secondary outcome STUDY 1 Assess the relationship between SWE/SWD/ATI values and standard blood-based liver function tests and evaluate how age, sex, and BMI influence SWE/SWD/ATI measurements.
Correlation between SWE/SWD values and clinical outcomes in livertransplats . (Study 2b)
Imaging results will be compared with clinical assessments to determine association to transplant-related complications
Correlation between SWE/SWD/ATI values and serological liver markers (Study 2c)
Blood tests will be analyzed alongside imaging biomarkers to assess liver function and disease progression.
Correlation between MRE and histological findings from liver biopsy. (study3)
MRE measurements will be compared to biopsy results to validate global imaging biomarkers.
Inter-operator variability in ultrasound measurements (STUDY 1)
Assess reproducibility of SWE/SWD/ATI across trained radiologists.
Longitudinal changes in imaging biomarkers in patients with repeated biopsies (Study 1)
Evaluate SWE/SWD/ATI trends over time in patients undergoing serial liver biopsies.
Comparison of imaging biomarkers between controls and patients (STUDY 1)
Establish reference values and diagnostic thresholds for SWE/SWD/ATI in a pediatric cohort without liver disease
Correlation of imaging biomarkers with BMI, weight, and serological liver markers (Study2d)
Evaluate the relationship between SWE/SWD/ATI values and anthropometric measures and liver function tests.
Click on the lead sponsor to view all of their trials.
Sahlgrenska University Hospital
Lead sponsor
Queen Silvia Children's Hospital, Gothenburg, Sweden
Collaborator
