Longitudinal Assessment of Transient Elastography in Cystic Fibrosis

Study Overview
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Brief Summary
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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
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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.

Official Title
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Longitudinal Assessment of Transient Elastography in Cystic Fibrosis

Conditions
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Cystic Fibrosis Liver Disease

Intervention / Treatment
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* Procedure: Transient Elastography (TE)


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 6 Years
Maximum Age: 26 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Arms and Interventions

| Intervention/Treatment |
| --- |
|Procedure: Transient 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)|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Liver Stiffness Measurement (LSM) obtained via transient elastography using FibroScanTM | 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). | Baseline | 
| Liver Stiffness Measurement (LSM) obtained via transient elastography using FibroScanTM | 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). | Year 1 | 
| Liver Stiffness Measurement (LSM) obtained via transient elastography using FibroScanTM | Liver stiffness quantifies liver fibrosis and is measured in kPa (median of 10 subsequent | Year 2 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Liver steatosis obtained via transient elastography | Controlled Attenuation Parameter (CAP) quantifies liver steatosis and is measured in dB | Baseline, Year 1, Year 2 | 
| Comparison of liver stiffness and liver steatosis measurements at each time point to grayscale ultrasound grades | Grayscale ultrasound grades will be obtained from the parent study (PUSH: Clinical Trials: NCT01144507) | Baseline, Year 1 and Year 2 | 
| Comparison of liver stiffness and liver steatosis measurements at each time point to clinical findings of portal hypertension | Grayscale ultrasound grades will be obtained from the parent study (PUSH: Clinical Trials: NCT01144507) | Baseline, Year 1 and Year 2 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Cystic Fibrosis, Cystic Fibrosis Liver Disease, Pancreatic insufficiency, Transient Elastography