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The investigated cohort will examine liver and spleen fibrosis in patients with Gaucher disease divided into two groups, naive GD patients and GD patients treated with ERT. As liver biopsy in these patients not recommended because the risk of bleeding using Fibroscan is a safe with diagnostic accuracy regarding the liver (& Spleen) fibrosis. Estimating spleen fibrosis is an innovative approach in liver disease and Gaucher.
The evaluation of fibrosis with this new and safe method could avoid complications antiinvasive procedure in GD patients. The addition of fibrosis biomarkers will help for patients score evaluation. The finding of liver and spleen stiffness will be evaluated in native and ERT treated Gaucher patients in order to assess ERT effect on fibrosis.
The Aims are: 1) To assess liver and spleen stiffness measurement using fibroscan and evaluate liver and spleen fibrosis in patients with GD.
To compare the elastography in two cohorts of GD patients: ERT treated and naïve GD patients and two control groups of patients: healthy and Non Alcoholic Steatohepatitis (NASH) patients.
To correlate the elastography findings with clinical and laboratory data in the four patient groups focusing on Gaucher disease manifestations and GD severity. To compare the elastography in GD naïve and ERT treated patients.
Full description
The investigated cohort will include 4 groups of patients 20 probands in each group including: 1) Naïve GD patients 2) GD treated with ERT 3) Healthy control 4) Patients with NonAlcoholic Steatohepatitis( NASH) which are followed at hepatology unit of the Ziv Hospital and healthy controls.
Serum fibrosis markers will be tested including: Bilirubin ,GGT, Haptoglobin,AST,ALT .The findings of spleen and liver fibrosis will be correlated with disease severity usingZimran's Severity Score Index (SSI) liver function tests , serology for viral hepatitis, GDbiomarkers, hemoglobin, platelet levels, and a GD severity score.
Protein C will be measured in all groups of patients Protein C activity as it may be used as a sensitive marker of hepatocellular damage even in those patients with mild liver affection .Also Patients with cirrhosis possess an imbalance in pro-coagulant versus anticoagulantactivity due to increased factor VIII and decreased protein C.
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• Active liver disease ,cirrhosis patients.
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Interventional model
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40 participants in 4 patient groups
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Central trial contact
Maher Hijazi; Hanna Rosenbaum, MD
Data sourced from clinicaltrials.gov
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