Liver and the Spleen Stifness in Adult Patients With Gaucher's Disease Using Ultrasound Shear Wave Elastography

Prospective Study: Shear-Wave Elastography of the Liver and the Spleen in Adult Patients With Gaucher's Disease

The investigated cohort will examine liver and spleen fibrosis in patients with Gaucher Disease(GD) by using Shear Wave Elastography- SWE to evaluation fibrosis of the tissue and to check the correlation of fibrosis with the biomarkers of disease severity.

Study Overview

• Status: Terminated
• Conditions: Liver Fibrosis; Gaucher Disease; Spleen; Fibrosis

Detailed Description

To assess liver and spleen stiffness measurement using Shear Wave Elastography-SWE and evaluate liver and spleen fibrosis in patients with Gaucher Disease. To compare the elastography with the biomarkers that taken at the GD haematology clinic and to check the correlation with severity of liver and spleen fibrosis.

SWE 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 relatively new and safe method could avoid complications and invasive procedure in GD patients .

• Study Type: Observational
• Enrollment (Actual): 18

Contacts and Locations

Study Locations

• Israel
  • Tsafon
    • Afula, Tsafon, Israel, 1834111
      • HaEmek Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Probability Sample

Study Population

adult patient with Gaucher disease.

Description

Inclusion Criteria:

1. Patients with Gaucher disease over the age of 18 years.
2. Patients able to sign informed consent form.
3. Women are not pregnant.
4. Patients who read and understood the contents of the consent form and gave their written consent to participate in the study.

Exclusion Criteria:

1. Participants under the age of 18.
2. Pregnant women.
3. Participants are unable to sign an informed consent form.
4. Participants with known liver disease not associated with Gaucher disease such as liver cirrhosis, patients with HBV, HBC or liver cirrhosis.

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Shear Wave Elastography of liver to asses liver fibrosis in Gaucher disease patients.	Shear wave elastography(SWE),an ultrasound imaging technique that can produce 2D and 3D images and quantified measurements of tissue stiffness noninvasively. SWE obtained from the right liver lobe for staging liver fibrosis according to Metavir Fibrosis Score with sensitivity86%-98% and specificity 90%-93%. SWE well corelated with Metavir Fibrosis Score which is system used to assess the extent of inflammation and fibrosis by histopathological evaluation in a liver biopsy. The biomarkers of disease severity will be correlated to METAVIR score. METAVIR score(F0-F4) ,Fibrosis Level measured by KPa(kiloPascal) using SWE. F0 = Below 5 KPa, No fibrosis. F1=5.0-7.1 KPa, Mild Fibrosis. F2=7.1-8.7 KPa, Significant Fibrosis. F3=8.7-10.4 KPa, Sever Fibrosis. F4=10.4-19 KPa ,Cirrhosis. SWE can differentiate patients with no or minimal (F0 and F1) fibrosis from those with severe fibrosis or cirrhosis (F3 and F4) with no need for biopsy unless there are other factors that need to be considered.	0-2 hours

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Shear Wave Elastography of spleen to asses spleen fibrosis in Gaucher disease patients.	Shear wave elastography (SWE), an ultrasound imaging technique that can produce 2D and 3D images and quantified measurements of tissue stiffness noninvasively. Participants with Gaucher disease will under go shear wave elastography for spleen. Ultrasound Elastography is a non-invasive test which uses the technology of Shear Wave Elastography (SWE) obtained from the spleen for staging tissue fibrosis according to a study, Spleen Elastography was performed in 59 healthy individuals and the mean value measured in KPa (kiloPascal) was 16.6 ± 2.5 kPa, independent of patient age, sex or spleen size. above the normal value it is considerd fibrosis of the tisuue of the spleen.	0-2 hours

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Biomarkers of the Gaucher Disease	Gaucher Disease (GD) patients will be referred from the (GD) hematology outpatient clinic, in different patients different biomarker are already taken (as blood test). Biochemical abnormalities which might be already taken from patients, for example; blood level of ferritin (unit: ng/mL), angiotensin converting enzyme (unit: kU/L), chitotriosidase (unit: U/L) and polyclonal immunoglobulins (unit: ng/dL) . Elevated biomarkers can predict the severity of the disease.	Up to 1 month

Collaborators and Investigators

• Sponsor: HaEmek Medical Center, Israel
• Investigators: Principal Investigator: ziv neeman, MD, HaEmek Medical Center

Study record dates

Study Major Dates

• Study Start (Actual): March 30, 2019
• Primary Completion (Actual): October 31, 2022
• Study Completion (Actual): October 31, 2022

Study Registration Dates

• First Submitted: March 17, 2019
• First Submitted That Met QC Criteria: March 27, 2019
• First Posted (Actual): April 1, 2019

Study Record Updates

• Last Update Posted (Estimate): March 6, 2023
• Last Update Submitted That Met QC Criteria: March 2, 2023
• Last Verified: March 1, 2023

More Information

Terms related to this study

• Keywords: shear wave elastography
• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Metabolic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Liver Diseases; Genetic Diseases, Inborn; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Lipid Metabolism Disorders; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Lipidoses; Lipid Metabolism, Inborn Errors; Fibrosis; Liver Cirrhosis; Gaucher Disease
• Other Study ID Numbers: 0135-18-EMC

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No