Clinical trial NCT02979340: MRI to Characterize and Predict CF Liver Disease in PUSH Cohort
Official title: 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
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Cystic Fibrosis Liver Disease
Pancreatic Insufficiency
Keywords: Cystic Fibrosis Liver Disease
Pancreatic Insufficiency
Lead sponsor: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Sponsor class: NIH
Collaborators: name: Cystic Fibrosis Foundation
First posted: 2016-12-01
Enrollment: 115
Sex: ALL
Minimum age (years): 6
Maximum age (years): 26
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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.

Brief summary: 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

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Change over Time in Liver stiffness and Lipid content over time using MRE
timeFrame: BSL, Year 1, Year 2
Secondary outcomes:
Other outcomes:
Locations:
city: Aurora
country: United States
facility: Children's Hospital Colorado
state: Colorado
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta
state: Georgia
city: Indianapolis
country: United States
facility: Riley Hospital for Children
state: Indiana
city: Baltimore
country: United States
facility: Johns Hopkins School of Medicine
state: Maryland
city: Saint Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical
state: Ohio
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
Study officials:
affiliation: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
name: Ed Doo, MD
role: STUDY_DIRECTOR
affiliation: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
name: Averell Sherker, MD
role: STUDY_DIRECTOR
References:
citation: Towbin AJ, Ye W, Huang S, Karmazyn BW, Molleston JP, Masand P, Leung DH, Chang S, Narkewicz MR, Alazraki AL, Freeman AJ, Otto RK, Green N, Kamel IR, Karnsakul WW, Magee JC, Tkach J, Palermo JJ. Prospective study of quantitative liver MRI in cystic fibrosis: feasibility and comparison to PUSH cohort ultrasound. Pediatr Radiol. 2023 Oct;53(11):2210-2220. doi: 10.1007/s00247-023-05706-6. Epub 2023 Jul 28.