Liver Fibrosis in Sickle Cell Disease

NCT02007746 | Completed

• 1 other identifier: 20120222
• Study Type: observational
• Target: 26
• Locations: 1 country
• Sites: 1

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.

Conditions

Sickle Cell Disease

Keywords

sickle cell disease; hemochromatosis; liver fibrosis; liver cirrhosis; FibroScan; Ferriscan; liver iron

Outcome Measures

Primary Outcomes (1)

• Liver transient elastography (FibroScan) of liver iron content and stiffness

  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 Outcomes (2)

• magnetic resonance imaging (MRI) measure of liver iron content and stiffness

  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

  at clinic visit blood draw (about 1 minute)

Study Arms (2)

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); Other: Ferriscan; Procedure: Liver biopsy in iron overloaded subjects.

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)

Interventions

Liver transient elastography (Fibroscan) OTHER

Fibroscan will be performed as a measure of liver stiffness. The study will be obtained free of charge in this study.

Also known as: FibroScan

Subjects with sickle cell disease and iron overload; control patients with sickle cell disease

Ferriscan OTHER

Ferriscan will be done to assess the quantity of liver iron in iron overloaded subjects.

Subjects with sickle cell disease and iron overload

Liver biopsy in iron overloaded subjects. PROCEDURE

Liver biopsy will be done and the results will be compared to Fibroscan results.

Subjects with sickle cell disease and iron overload

Eligibility Criteria

• Age: 10 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• pediatric patients age 10 years and older with sickle cell disease
• meeting other criteria:
• history of chronic transfusion and iron overload and/or
• known liver disease related to sickle cell or iron overload
• signed consent and assent (as applicable)

You may not qualify if:

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

Sponsors & Collaborators

• University of Miami: lead

Study Sites (1)

University of Miami

Miami, Florida, 33136, United States

Location

Related Publications (2)

• Voskaridou E, Plata E, Douskou M, Papadakis M, Delaki EE, Christoulas D, Terpos E. Treatment with deferasirox (Exjade) effectively decreases iron burden in patients with thalassaemia intermedia: results of a pilot study. Br J Haematol. 2010 Jan;148(2):332-4. doi: 10.1111/j.1365-2141.2009.07930.x. Epub 2009 Oct 26. No abstract available.

  PMID: 19863539 RESULT

• Fraquelli M, Cassinerio E, Roghi A, Rigamonti C, Casazza G, Colombo M, Massironi S, Conte D, Cappellini MD. Transient elastography in the assessment of liver fibrosis in adult thalassemia patients. Am J Hematol. 2010 Aug;85(8):564-8. doi: 10.1002/ajh.21752.

  PMID: 20658587 RESULT

MeSH Terms

Conditions

Anemia, Sickle Cell; Hemochromatosis; Liver Cirrhosis

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metal Metabolism, Inborn Errors; Metabolism, Inborn Errors; Iron Overload; Iron Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Liver Diseases; Digestive System Diseases; Fibrosis; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Ofelia Alvarez, MD

  University of Miami - Director Sickle Cell Services Pediatric Hematology/Oncology

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE CONTROL
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director, Sickle Cell Disease Center, Pediatric Hematology/Oncology

Study Record Dates

• First Submitted: December 6, 2013
• First Posted: December 11, 2013
• Study Start: July 1, 2012
• Primary Completion: June 1, 2017
• Study Completion: June 1, 2017
• Last Updated: May 26, 2021

Record last verified: 2021-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)