Cystic Fibrosis Liver Disease
Conditions
Keywords
Cystic Fibrosis Liver Disease, Pancreatic insufficiency, Cystic Fibrosis, Transient Elastography
Brief summary
To determine if transient elastography (TE), when combined with ultrasound (US) pattern characterization can improve the prediction of progression to a nodular pattern on US. To confirm the feasibility of obtaining TE measurements in children with Cystic Fibrosis (CF) To prospectively assess whether TE data are associated with conventional laboratory markers of hepatic fibrosis To determine the variability of TE measurements taken at different sites in the same patient
Detailed description
A noninvasive assessment of hepatic fibrosis is desperately needed to advance the care of children with CF significant liver disease and to provide for measurements during clinical trials. That global assessment might serve as both a predictor/descriptor of disease course but also as a critical biomarker for clinical research. FibroScan® measurement of liver stiffness has great potential to fill this void. The underlying hypothesis of this proposal is that elastography in addition to US can improved the prediction of the development of a nodular liver on US and development of portal hypertension over time in children and young adults with CF.
Interventions
PROCEDURETransient Elastography (TE)
Participants enrolled in Cystic Fibrosis Liver Disease Network (CFLD NET)Prediction by Ultrasound of the Risk of Hepatic Cirrhosis in Cystic Fibrosis (PUSH) study in longitudinal follow up at centers with Fibroscan available (currently 8/11 centers)
Sponsors
Cystic Fibrosis Foundation
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
6 Years to 26 Years
Healthy volunteers
Yes
Inclusion criteria
* Participants enrolled in CFLD NET PUSH study in longitudinal follow up at centers with Fibroscan available (currently 8/11 centers) * Entry criteria for that study were: * CF as determined by sweat chloride \>60 meq/l * Pancreatic insufficiency * Age 3-12 years old at entry * For entry into the longitudinal follow up subjects were in one of two groups * A screening US pattern of nodular liver (CIR), heterogeneous increased echogenicity (HTG) or homogeneous increased echogenicity (HMG) * A screening US pattern of normal (NL) matched to a HTG subject (2 NL:1HTG) by age, center and pseudomonas status
Exclusion criteria
* Exited from the PUSH Study * Unable / unwilling to sign consent
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Liver Stiffness Measurement (LSM) obtained via transient elastography using FibroScanTM | Baseline | Liver stiffness quantifies liver fibrosis and is measured in kPa (median of 10 subsequent valid measurements and are deemed acceptable if the ratio of interquartile range and median is \<30% and success rate is \>60%, meaning 10 valid measurements are obtained within 16 attempts). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Liver steatosis obtained via transient elastography | Baseline, Year 1, Year 2 | Controlled Attenuation Parameter (CAP) quantifies liver steatosis and is measured in dB |
| Comparison of liver stiffness and liver steatosis measurements at each time point to grayscale ultrasound grades | Baseline, Year 1 and Year 2 | Grayscale ultrasound grades will be obtained from the parent study (PUSH: Clinical Trials: NCT01144507) |
| Comparison of liver stiffness and liver steatosis measurements at each time point to clinical findings of portal hypertension | Baseline, Year 1 and Year 2 | Grayscale ultrasound grades will be obtained from the parent study (PUSH: Clinical Trials: NCT01144507) |
Other
| Measure | Time frame | Description |
|---|---|---|
| Inter quartile range (IQR) (kPa or dB/m) of all valid measurements within the examination | Baseline, Year 1 and Year 2 | To reflect the dispersion of stiffness and steatosis |
| IQR/median ratio of all valid measurements within the examination | Baseline, Year 1 and Year 2 | This quantity should remain as low as possible to ensure reliable results (goal \< 30%) |
| Invalid measurements and success rate on liver stiffness and liver steatosis measurements | Baseline, Year 1 and Year 2 | — |
Countries
Canada, United States
Outcome results
None listed