Clinical trial NCT02007746: Liver Fibrosis in Sickle Cell Disease
Official title: Assessment of Liver Fibrosis in Patients With Sickle Cell Disease
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Sickle Cell Disease
Keywords: sickle cell disease
hemochromatosis
liver fibrosis
liver cirrhosis
FibroScan
Ferriscan
liver iron
Lead sponsor: University of Miami
Sponsor class: OTHER
First posted: 2013-12-11
Enrollment: 26
Sex: ALL
Minimum age (years): 10
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_CONTROL
Time perspective: CROSS_SECTIONAL
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.

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.

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.

Eligibility 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.

Primary outcomes:
measure: Liver transient elastography (FibroScan) of liver iron content and stiffness
timeFrame: at imaging visit (3 minutes)
Secondary outcomes:
measure: magnetic resonance imaging (MRI) measure of liver iron content and stiffness
timeFrame: at imaging visit (about 30-60 minutes)
measure: 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
timeFrame: at clinic visit blood draw (about 1 minute)
Other outcomes:
Locations:
city: Miami
country: United States
facility: University of Miami
state: Florida
Study officials:
affiliation: University of Miami - Director Sickle Cell Services Pediatric Hematology/Oncology
name: Ofelia Alvarez, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.
citation: 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.