Primary objective: assessment of liver fibrose in children with cystic fibrosis, using transient elastography (Fibroscan). Results will be compared with other surrogate markers of CFRLD: liver ultrasonography and biochemical markers: AST, ALT, PT,…
ID
Source
Brief title
Condition
- Gastrointestinal signs and symptoms
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Primary study outcome: liver-elasticity (as measured by Fibroscan): in kPA,
related to: ultrasonography of the liver (liver parenchyma, intra- and
extrahepatic biliary tree, portal vein, presence of collateral circulation,
splenomegaly, flow direction in portal vein) , and related to biochemical
markers: cholestasis (elevated blirubin, gamma-GT, alkaline phosphatase) or
hepatitis (elevated serum transaminase levels). and liver function tests:
prothrombin time, albumin.
Secondary outcome
CFTR mutation, pulmonary function (FEV1), nutritional status, pancreas
insufficientie
Background summary
Patients with cystic fibrosis can develop liver disease. CF related liver
disease (CFRLD). CFRLD consists of progressive fibrosis and focal cirrhosis.
Fibrosis develops already in the first 10 years after diagnosis. CFRLD is seen
in 18 - 37 % of CF patients.
The detection of (early) fibrosis is important, because early treatment with
ursodeoxycholic acid (UDCA) can be considered. Liver biopsy is another
technique to evaluate fibrosis of the liver. A liver biopsy is an invasive
technique, and because of the focal nature of the fibrosis, biopsies can be
false-negative. Conventional ultrasonography is not accurate enough to detect
early fibrosis.
Transient elastography (Fibroscan) is a promising technique to detect fibrosis
in an non-invasive manner.
No studies are yet published on transient elastography in a large cohort of
children with CF.
Study objective
Primary objective: assessment of liver fibrose in children with cystic
fibrosis, using transient elastography (Fibroscan). Results will be compared
with other surrogate markers of CFRLD: liver ultrasonography and biochemical
markers: AST, ALT, PT, platelet count, gamma-GT, alkaline phosphatase,
bilirubin, albumin),
Secundary objective: to determine the incidence of CFRLD in a large cohort of
patients, 2. to evealuate risk factors to develop CFRLD: like FEV1,
CFTR-mutation, nutritional status and pancreatic insufficiency.
Study design
Crossectional, monocentre cohort studie, in which all children eligible for
inclusion will be included at the moment they visit the CF center for their
yearly check-up.
Study burden and risks
Patient will lie down for 30 minutes on the examination table (on the back or
on the side).
Dr Molewaterplein 60
3015 GJ Rotterdam
Nederland
Dr Molewaterplein 60
3015 GJ Rotterdam
Nederland
Listed location countries
Age
Inclusion criteria
Children with cystic fibrosis treated in the Erasmus Medical Centre Rotterdam / Sophia Children's Hospital, age 2 - 18 years, written informed consent.
Exclusion criteria
Age 0 - 2 year, measurement with Fibroscan is not feasible, as a result of small intercostal margins and/or ascits
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| CCMO | NL20512.078.07 |