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.
Study Type
OBSERVATIONAL
Enrollment
423
Children's Hospital & Research Center Oakland
Oakland, California, United States
Georgia Regents University
Augusta, Georgia, United States
Children's Memorial Hospital
Chicago, Illinois, United States
Adult Comprehensive Sickle Cell Center, Duke University Medical Center
Durham, North Carolina, United States
Thomas Jefferson SCD Program
Philadelphia, Pennsylvania, United States
St. Jude Children's Research Hospital
Memphis, Tennessee, United States
Universitätsklinikum Hamburg-Eppendorf
Hamburg-Eppendorf, Germany
UCL Cancer Institute
London, United Kingdom
Identification of iron overloaded patients with Sickle Cell Disease and Thalassemia eligible for future study of iron deposition and biochemical mechanisms
Patients with similar duration of chronic transfusion and age at onset of chronic transfusion therapy will be identified from 10 participating centers. Detailed information on iron burden and transfusion, medical, and chelation histories will be obtained in order to establish a cohort of patients that could be available for a future powered study of extra-hepatic iron deposition and underlying biochemical mechanisms.
Time frame: March 2010 - July 2013
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