Protocol Number: 85-H-0087

Title:

Clinical Course of Patients with Transfusional Hemochromatosis on Deferoxamine

Number:

85-H-0087

Summary:

When patients receive repeated blood transfusions the level of iron in the patient's blood can rise. When iron is processed in the body a protein known as hemosiderin can begin collecting in the organs. If too much hemosiderin collects in the organs they can begin to malfunction. This condition is called transfusional hemochromatosis.

An organ of particular importance in transfusional hemochromatosis is the heart. Patients born with diseases requiring blood transfusions at birth begin to develop heart problems in their teens. These patients typically only live for 17 years. Adults that require transfusions can begin experiencing heart problems after 100-200 units of backed red blood cells.

Deferoxamine (Desferal) is a drug that binds to iron and allows it to be excreted from the body. It is the only effective way to remove iron from patients who have been overloaded with iron because of multiple transfusions. Previous studies have lead researchers to believe that deferoxamine, when given as an injection under the skin (subcutaneous), can be delay or prevent heart complications.

Researchers plan to continue studying patients receiving deferoxamine as treatment for the prevention of heart complications associated with repeated blood transfusions. In this study researchers will attempt;

1)To determine if deferoxamine, given regularly, can indefinitely prevent the heart, liver, and endocrine complications associated with transfusional hemochromatosis

2)To determine whether heart disease caused by transfusional hemochromatosis can be reversed by intensive treatment with deferoxamine.

Sponsoring Institute:

National Heart, Lung and Blood Institute (NHLBI)

Recruitment Detail

Type: Follow-up Of Previously Enrolled Subjects Only

Gender: Male & Female

Referral Letter Required: No

Population Exclusion(s): None

Eligibility Criteria: This study is not currently recruiting new subjects. If you have questions about participating in a study, please contact the Patient Recruitment and Public Liaison Office, CC.

Special Instructions: Currently Not Provided

Disease Category:

PROTICD

Keywords:

Desferal

Thalassemia

Liver Iron Concentration

Endocrine Evaluation

Diabetes Mellitus

Cardiac Disease

Recruitment Keywords:

Acquired Anemia

Conditions:

Diabetes Mellitus

Heart Disease

Hemochromatosis

Thalassemia

Investigational Drug(s):

None

Investigational Device(s):

None

Contacts:

This study is not currently recruiting new subjects. If you have questions about participating in a study, please contact the Patient Recruitment and Public Liaison Office, CC.

Citations:

Bone marrow transplantation in patients with thalassemia

5-Azacytidine selectivity increases gamma-globin aynthesis in a patient with beta+ thalassemia

Magnetic susceptibility measurement of human iron stores

• Questions about participating in a study, please contact the Patient Recruitment and Public Liaison Office, CC.
• Technical questions regarding the Clinical Center web site, please contact the Department of Networks and Applications, CC.

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Warren Grant Magnuson Clinical Center (CC)
National Institutes of Health (NIH)
Bethesda, Maryland 20892. Last update: 10/23/2004