The purpose of this study is to determine the effects of the oral iron chelator Deferasirox on liver iron content after one year of treatment in patients with iron overload from repeated blood transfusions. Beta-thalassemia patients unable to be treated with deferoxamine or patients with rare chronic anemias such as Myelodysplastic Syndrome, Fanconi's Syndrome, Blackfan-Diamond Syndrome, and Pure Red Blood Cell Anemia are eligible for this study. Liver iron content will be measured by liver biopsy at the beginning of the study and after one year of treatment. However, those patients living in the San Francisco/Oakland area may have a SQUID in place of the liver biopsy if the biopsy is not medically possible for them. The SQUID is a non-invasive magnetic means to measure liver iron content.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
175
Children's Hospital Oakland
Oakland, California, United States
Stanford Hospital
Stanford, California, United States
Northwest Medical Specialists
Arlington Heights, Illinois, United States
Children's Hospital Boston
Boston, Massachusetts, United States
Weill Medical College of Cornell University
New York, New York, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
To evaluate the effects of treatment on the liver iron content(LIC)
Evaluate tolerability profile
Estimate the absolute and relative change of LIC and total body iron excretion (TBIE) rate
Evaluate the relationship between LIC and potential surrogate markers
Evaluate the relationship between PD and safety variables
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