NCT01913548

Multi-Center Study of Iron Overload: Survey Study (MCSIO)

Modulation of Iron Deposition in Sickle Cell Disease and Other Hemoglobinopathies SURVEY STUDY

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 423 (actual)
• Sponsor: UCSF Benioff Children's Hospital Oakland · Academic / Other
• Sex: All
• Age: 16 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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.

Conditions

• Sickle Cell Disease
• Thalassemia
• Diamond-Blackfan Anemia

Timeline

• Start date: 2010-03-31
• Primary completion: 2013-08-31
• Completion: 2013-10-31
• First posted: 2013-08-01
• Last updated: 2020-09-23

Locations

8 sites across 3 countries: United States, Germany, United Kingdom