Multi-Center Study of Iron Overload: Survey Study (MCSIO)
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.
Sickle Cell Disease
|Study Design:||Observational Model: Case Control
Time Perspective: Prospective
|Official Title:||Modulation of Iron Deposition in Sickle Cell Disease and Other Hemoglobinopathies SURVEY STUDY|
- Identification of iron overloaded patients with Sickle Cell Disease and Thalassemia eligible for future study of iron deposition and biochemical mechanisms [ Time Frame: March 2010 - July 2013 ] [ Designated as safety issue: No ]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.
|Study Start Date:||March 2010|
|Estimated Study Completion Date:||October 2013|
|Primary Completion Date:||July 2013 (Final data collection date for primary outcome measure)|
Sickle Cell Disease (SCD)
Patients with sickle cell diseases, 16 years or older with 10-20 years of transfusion (defined as 0.2-0.6mg Fe/kg/day exposure with annual ferritin levels greater than 2500 in at least 60% of years of chronic transfusion); 0 to 9 years old at the initiation of chronic transfusions; no exchange transfusions in the previous 6 months; and iron overload documented by either liver biopsy, MRI or SQUID with estimated LIC (liver iron content) of greater than 7 mg/g dry wt in the previous 6 months or ferritin level greater than 1500mg/dl.
Thalassemia Major (TM)
Patients with β-thalassemia major and transfusion-dependent E-beta THAL. 16 years or older with 10-20 years of chronic transfusion (defined above), 0 to 9 years old at the initiation of chronic transfusions, iron overload documented by either liver biopsy, MRI or SQUID with estimated LIC of greater than 7 mg/g dry wt in the previous 6 months.
Diamond Blackfan Anemia (DBA)
Patients with DBA, 16 years or older with 10-20 years of transfusion, 0 to 9 years old at the initiation of chronic transfusions, iron overload documented by either liver biopsy, MRI or SQUID with estimated LIC of greater than 7 mg/g dry wt in the previous 6 months.
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01913548
|United States, California|
|Children's Hospital & Research Center Oakland|
|Oakland, California, United States, 94609|
|United States, Georgia|
|Georgia Regents University|
|Augusta, Georgia, United States, 30912|
|United States, Illinois|
|Children's Memorial Hospital|
|Chicago, Illinois, United States, 60614|
|United States, North Carolina|
|Adult Comprehensive Sickle Cell Center, Duke University Medical Center|
|Durham, North Carolina, United States, 27710|
|United States, Pennsylvania|
|Thomas Jefferson SCD Program|
|Philadelphia, Pennsylvania, United States, 19107|
|United States, Tennessee|
|St. Jude Children's Research Hospital|
|Memphis, Tennessee, United States, 38105-3678|
|UCL Cancer Institute|
|London, United Kingdom, WC1E 6BT|
|Principal Investigator:||Elliott Vichinsky, MD||Children's Hospital & Research Center Oakland|
|Principal Investigator:||John Porter, MD||University College London Cancer Institute|