Sickle Cell Disease Clinical Trial
Official title:
Modulation of Iron Deposition in Sickle Cell Disease and Other Hemoglobinopathies SURVEY STUDY
The purpose of this study is to demonstrate that a sufficient number of iron-overloaded thalassemia (THAL), Sickle Cell Disease (SCD)and Diamond Blackfan Anemia (DBA) populations with similar duration of chronic transfusion, and age at start of transfusions would be available for a confirmatory study. The study will examine the hypothesis that a chronic inflammatory state in SCD leads to hepcidin- and cytokine-mediated iron withholding within the RES (reticuloendothelial system), lower plasma NTBI (non-transferrin bound iron) levels, less distribution of iron to the heart in SCD.
A detailed iron burden, transfusion and chelation history will be obtained from chart review
or from participant recall.
Iron burden data will include: 1) documentation of liver iron, and 2) average annual ferritin
values.
Transfusion data will include: (1) age at onset of regular transfusions, (2) years of chronic
transfusion therapy, and (3) pre-transfusion Hb calculated as average of all assessments for
each year.
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