Liver Fibrosis in Sickle Cell Disease

May 21, 2021 updated by: Ofelia Alvarez, University of Miami

Assessment of Liver Fibrosis in Patients With Sickle Cell Disease

Patients with sickle cell disease many have a number of systemic complications, including liver problems. Some of these liver problems lead to liver fibrosis/cirrhosis, secondary to chronic blood transfusions. The purpose of this study is to investigate FibroScan readings in patients with sickle cell disease and iron overload secondary to blood transfusions, and to correlate the FibroScan results with Ferriscan. A comparison with the results of FibroScan to patients with Sickle cell without known liver disease, who have never been on chronic transfusions and with normal liver function profiles will also be made.The primary hypothesis is that the results of FibroScan will correlate with the results of Ferriscan and liver biopsy.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

Liver biopsy is the gold standard to examine the liver for iron deposits and histology. However, liver biopsy is invasive and involves a risk of bleeding and pain. Biopsy may also miss significant pathology if the small biopsy specimen is taken from an uninvolved part of the liver. Non-invasive techniques such as MRI are now used to evaluate the liver iron content. MRI can visualize the whole liver and measure liver iron content. MRI, however, will not detect liver scarring.

Liver transient elastography (FibroScan) is a non-invasive ultrasound tool for assessing liver fibrosis or scarring by measuring liver stiffness (LSM). Compared with liver biopsy, FibroScan provides immediate results and is a painless, short (3 mins), simple procedure to perform. In some studies FibroScan reports have correlated well with liver biopsy results of fibrosis and cirrhosis, and with Ferriscan, ferritin and liver function tests.

This purpose of this study is to investigate the role of FibroScan in individuals with sickle cell anemia and iron overload or who have a diagnosis of liver disease, and to compare FibroScan readings with magnetic resonance imaging.

We will also compare the results of the Fibroscan with liver biopsy.

This study will also compare the results of FibroScan to patients with Sickle cell without any known liver disease, who have never been on chronic transfusions (defined as no more than 4 transfusions in lifetime) and who have normal liver function profiles.

Study Type

Observational

Enrollment (Actual)

26

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Florida
      • Miami, Florida, United States, 33136
        • University of Miami

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 years to 61 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

30 subjects with sickle cell disease and iron overload. 30 control subjects without history of chronic transfusions (less than 4 transfusions in lifetime) and without evidence of liver pathology.

Description

Inclusion Criteria:

  • pediatric patients age 10 years and older with sickle cell disease

  • meeting other criteria:

    1. history of chronic transfusion and iron overload and/or
    2. known liver disease related to sickle cell or iron overload
  • signed consent and assent (as applicable)

Exclusion Criteria:

  • children younger than 10 years
  • Pregnant females
  • Prisoners
  • Other causes of liver disease, unrelated to sickle cell or iron overload

Inclusion criteria for controls:

  • Patients 10 years and older with sickle cell disease without history of chronic transfusions (less than 4 transfusions in a lifetime)
  • and without obvious liver disease.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Case-Control
  • Time Perspectives: Cross-Sectional

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Subjects with sickle cell disease and iron overload
Patients will have blood tests done to evaluate liver function and general health, then have FibroScan and Ferriscan. A blinded (no access to laboratory parameters or available data) radiologist will interpret the Ferriscan. Liver biopsy will be also obtained (if not done within the previous year). Otherwise, the results of the recent liver biopsy will be collected.
Other: Liver transient elastography (Fibroscan)
Fibroscan will be performed as a measure of liver stiffness. The study will be obtained free of charge in this study.
Other Names:
  • FibroScan
Other: Ferriscan
Ferriscan will be done to assess the quantity of liver iron in iron overloaded subjects.
Procedure: Liver biopsy in iron overloaded subjects.
Liver biopsy will be done and the results will be compared to Fibroscan results.
control patients with sickle cell disease
Patients 10 years and older with sickle cell disease without history of chronic transfusions (less than 4 transfusions in a lifetime) and without obvious liver disease. Will have liver function blood tests and general health check ups. Then will have FibroScan performed. No liver biopsy will be performed in control patients.
Other: Liver transient elastography (Fibroscan)
Fibroscan will be performed as a measure of liver stiffness. The study will be obtained free of charge in this study.
Other Names:
  • FibroScan

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Liver transient elastography (FibroScan) of liver iron content and stiffness
Time Frame: at imaging visit (3 minutes)
Liver transient elastography (FibroScan) uses a probe consisting of an ultrasound transducer located at the end of a vibrating piston. The piston produces a vibration of low amplitude and frequency, which generate a shear wave that passes through the skin and liver tissue. The ultrasound then detects the propagation of the shear wave through the liver (at a depth of 25 - 65 mm below the skin surface) by measuring its velocity. The shear wave velocity is directly related to the tissue stiffness, with a higher velocity equating to higher tissue stiffness, corresponding to increasing severity of fibrosis.
at imaging visit (3 minutes)

Secondary Outcome Measures

Outcome Measure
Time Frame
magnetic resonance imaging (MRI) measure of liver iron content and stiffness
Time Frame: at imaging visit (about 30-60 minutes)
at imaging visit (about 30-60 minutes)
liver function tests (ALT, AST, serum alkaline phosphate, GGTP, total bilirubin, direct bilirubin), complete blood count, platelets, reticulocyte count, serum ferritin to assess liver function and evaluate overall health
Time Frame: at clinic visit blood draw (about 1 minute)
at clinic visit blood draw (about 1 minute)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University of Miami

Investigators

  • Principal Investigator: Ofelia Alvarez, MD, University of Miami - Director Sickle Cell Services Pediatric Hematology/Oncology

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 1, 2012

Primary Completion (Actual)

June 1, 2017

Study Completion (Actual)

June 1, 2017

Study Registration Dates

First Submitted

December 6, 2013

First Submitted That Met QC Criteria

December 6, 2013

First Posted (Estimate)

December 11, 2013

Study Record Updates

Last Update Posted (Actual)

May 26, 2021

Last Update Submitted That Met QC Criteria

May 21, 2021

Last Verified

May 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 20120222

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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