The purpose of this trial is to evaluate changes in cardiac iron as measured by MRI T2\* in beta-thalassemia patients with deferasirox treatment.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
30
Oral deferasirox 30mg/kg/day once per day for 77 weeks.
Childrens Hospital of Los Angeles
Los Angeles, California, United States
Children's Hospital and Research Center at Oakland
Oakland, California, United States
Children's Memorial Hospital
Chicago, Illinois, United States
Magnetic Resonance Imaging (MRI) T2* and Absolute Change From Baseline in MRI T2*
Cardiac T2\* was measured in the short axis plane at the widest point of a 4-chamber localizer using custom breath-hold R2\* gradient echo sequences modeled after techniques used by Anderson et al (2001) and Westwood et al (2003).
Time frame: From Baseline to 25, 49, 77 Week
Change From Baseline in Liver Iron Concentration (LIC) Was Measured by MRI R2 From Absolute Change From Baseline to 101 Weeks
MRI evaluation of liver iron concentration has been validated by liver biopsy (St Pierre et al 2005). Studies comparing T2\* values of liver iron concentration (LIC) with LIC as assessed by biopsy have confirmed that T2\* values reflect liver iron content (Wood et al 2003b). Direct tissue-validation of cardiac T2\* measurements in humans has not been performed because endomyocardial biopsy is a dangerous and unreliable indicator of cardiac iron overload (Olson et al 1989, Fitchett et al 1980). However, it has been shown that cardiac T2\* accurately reflects cardiac iron in a gerbil iron cardiomyopathy model (Wood et al 2004b). T2\* measurements have shown excellent inter-scanner and inter-exam reproducibility, making them suitable for longitudinal monitoring (Westwood et al 2003a, Westwood et al 2003b).
Time frame: From Baseline to 25, 49, 77 Week
Left Ventricular Ejection Fraction (LVEF) and Change in Left Ventricular Ejection Fraction From Baseline to 101 Weeks
Magnetic resonance imaging (MRI)-measured cardiac T2\* and cardiac function reflected by left and right ventricle ejection fraction. A standardized MRI protocol for T2\* acquisition technique will be used in the centers. Images will be reviewed centrally by an expert MRI reader.
Time frame: From Baseline to 25, 49, 77 Week
Serum Ferritin and Changes From Baseline in Serum Ferritin During Study
Serum ferritin will be assessed at each study visit. Analysis was performed in Completer population consists of those participants who had a Week 77 MRI.
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Time frame: From Baseline to 25, 49, 77 Week