MRI to Characterize and Predict CF Liver Disease in PUSH Cohort

Analysis of Prediction by Ultrasound of the Risk of Hepatic Cirrhosis in Cystic Fibrosis (PUSH) Cohort Using Advanced MRI to Characterize and Predict CF Liver Disease

Specific Aim 1. Determine if valid results of non-sedated MRI based assessments of liver stiffness and lipid content can be obtained in more than 90% of children and young adults with cystic fibrosis.

Specific Aim 2. Determine hepatic lipid content using the HepaFat sequence and liver stiffness using MRE. Investigators will compare the results obtained by MRI with PUSH study grayscale ultrasounds in CF patients with normal, heterogeneous, homogeneous or nodular (cirrhotic) pattern on ultrasound.

Specific Aim 3. Creation of an imaging core lab to centralize evaluation of MR imaging data, allow for remote image upload, electronic data storage, and remote image viewing/interpretation. This infrastructure will be utilized to standardize image post processing.

Specific Aim 4. Using the longitudinal PUSH study, determine if MRI based imaging improves discrimination of subjects at risk for progression to advanced CF liver disease (development of cirrhosis) compared to using US imaging alone

Study Overview

• Status: Completed
• Conditions: Pancreatic Insufficiency; Cystic Fibrosis Liver Disease
• Intervention / Treatment: Procedure: MRI

Detailed Description

This proposal will be a cross sectional study of the current well-characterized PUSH cohort. Patients will receive an MRI at the time of their scheduled PUSH ultrasound. Ideally, the MRI will be performed the same day or a similar time point near liver ultrasound (+/-90 days, but Investigators will allow a larger window due to the slow progression of fibrosis).

Blinded radiology review of imaging sequences will be performed by a local study radiologist with central review performed by the Imaging Core. Analysis by the Imaging Core will include calculation of the liver stiffness, liver and spleen volume, and hepatic and lipid content for all MRI studies.

• Study Type: Observational
• Enrollment (Actual): 115

Contacts and Locations

Study Locations

• United States
  • Colorado
    • Aurora, Colorado, United States, 80045
      • Children's Hospital Colorado
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Children's Healthcare of Atlanta
  • Indiana
    • Indianapolis, Indiana, United States, 46202
      • Riley Hospital for Children
  • Maryland
    • Baltimore, Maryland, United States, 21217
      • Johns Hopkins School of Medicine
  • Missouri
    • Saint Louis, Missouri, United States, 63110
      • Washington University School of Medicine
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Cincinnati Children's Hospital Medical
  • Texas
    • Houston, Texas, United States, 77030
      • Texas Children's Hospital
  • Washington
    • Seattle, Washington, United States, 98105
      • Seattle Children's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years to 26 years (Child, Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Children aged 3 through 12 years of age at time of enrollment diagnosed with Cystic Fibrosis and pancreatic insufficiency and enrolled in the CFLD PUSH longitudinal study.

Description

Inclusion Criteria:

1. Currently enrolled in longitudinal follow up in PUSH study at a site with MR elastography.

Exclusion Criteria:

1. Age under 6 years (all current patients at time of opening of this study will be greater than 6 years of age).
2. Internal appliance or hardware that is not compatible with MR.
3. Inability to obtain MRI within 6 months of US.
4. Inability to cooperate with MRI.

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change over Time in Liver stiffness and Lipid content over time using MRE	Analysis of variance (ANOVA) will be used to compare the distribution of liver stiffness (in kPa) and lipid content measures between all groups defined by PUSH study grayscale ultrasounds results over time.	BSL, Year 1, Year 2

Collaborators and Investigators

• Sponsor: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
• Collaborators: Cystic Fibrosis Foundation
• Investigators: Study Director: Ed Doo, MD, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); Study Director: Averell Sherker, MD, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Publications and helpful links

Helpful Links

• MRE is a study in the ChiLDReN Network

Study record dates

Study Major Dates

• Study Start (Actual): January 27, 2017
• Primary Completion (Actual): December 27, 2019
• Study Completion (Actual): December 27, 2019

Study Registration Dates

• First Submitted: November 18, 2016
• First Submitted That Met QC Criteria: November 28, 2016
• First Posted (Estimated): December 1, 2016

Study Record Updates

• Last Update Posted (Actual): November 3, 2023
• Last Update Submitted That Met QC Criteria: November 1, 2023
• Last Verified: October 1, 2023

More Information

Terms related to this study

• Keywords: Pancreatic Insufficiency; Cystic Fibrosis Liver Disease
• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Respiratory Tract Diseases; Lung Diseases; Infant, Newborn, Diseases; Genetic Diseases, Inborn; Pancreatic Diseases; Liver Diseases; Fibrosis; Cystic Fibrosis; Exocrine Pancreatic Insufficiency
• Other Study ID Numbers: CFLD-MRE; U01DK062456 (U.S. NIH Grant/Contract)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO