Multi-Center Study of Iron Overload: Pilot and Survey Study

Status:	Archived
Conditions:	Anemia, Hematology
Therapuetic Areas:	Hematology
Healthy:	No
Age Range:	Any
Updated:	7/1/2011
Start Date:	November 2009
End Date:	February 2011

Pilot Study Examining Mechanisms of Iron Trafficking and Extra-hepatic Iron Distribution in Sickle Cell Disease, Thalassemia, and Other Iron Loading Anemias

The purpose of this study is to initiate pilot studies to demonstrate that a sufficient
number of iron-overloaded thalassemia, SCD and DBA populations with similar duration of
chronic transfusion, and age at start of transfusions would be available for a confirmatory
study and to validate that proposed multicenter MRI and biochemical studies can be
completed. The study will examine the hypothesis that a chronic inflammatory state in
Sickle Cell Disease (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.

MRI will be performed measuring pituitary, cardiac, and liver iron.

Laboratory samples should be obtained pre-transfusion and mid-cycle.

All interviews, exams, laboratory tests, study procedures and MRI assessments should be
completed within a 0 to 12 weeks time span.

In addition, a healthy control group will also be recruited with similar age, gender, and
ethnicity as the disease groups.

We found this trial at

sites

Children's Hospital Los Angeles ( Gastroenterology & Nutrition)

Los Angeles, California 20027

from

Los Angeles, CA