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Liver Fibrosis in Sickle Cell Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02007746
Recruitment Status : Completed
First Posted : December 11, 2013
Last Update Posted : May 26, 2021
Sponsor:
Information provided by (Responsible Party):
Ofelia Alvarez, University of Miami

Study Description
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Brief Summary:
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.

Condition or disease Intervention/treatment
Sickle Cell Disease Other: Liver transient elastography (Fibroscan) Other: Ferriscan Procedure: Liver biopsy in iron overloaded subjects.

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 Design
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Study Type : Observational
Actual Enrollment : 26 participants
Observational Model: Case-Control
Time Perspective: Cross-Sectional
Official Title: Assessment of Liver Fibrosis in Patients With Sickle Cell Disease
Actual Study Start Date : July 2012
Actual Primary Completion Date : June 1, 2017
Actual Study Completion Date : June 1, 2017

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Iron

Groups and Cohorts
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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 Name: 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 Name: FibroScan


Outcome Measures
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Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. magnetic resonance imaging (MRI) measure of liver iron content and stiffness [ Time Frame: at imaging visit (about 30-60 minutes) ]
  2. 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) ]

Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   10 Years to 65 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
Criteria

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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02007746


Locations
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United States, Florida
University of Miami
Miami, Florida, United States, 33136
Sponsors and Collaborators
University of Miami
Investigators
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Principal Investigator: Ofelia Alvarez, MD University of Miami - Director Sickle Cell Services Pediatric Hematology/Oncology
More Information
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Publications of Results:

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Responsible Party: Ofelia Alvarez, Director, Sickle Cell Disease Center, Pediatric Hematology/Oncology, University of Miami
ClinicalTrials.gov Identifier: NCT02007746    
Other Study ID Numbers: 20120222
First Posted: December 11, 2013    Key Record Dates
Last Update Posted: May 26, 2021
Last Verified: May 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Ofelia Alvarez, University of Miami:
sickle cell disease
hemochromatosis
liver fibrosis
liver cirrhosis
 FibroScan
Ferriscan
liver iron
Additional relevant MeSH terms:
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Liver Cirrhosis
Anemia, Sickle Cell
Fibrosis
Pathologic Processes
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
 Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Liver Diseases
Digestive System Diseases
Liver Extracts
Hematinics