Deferoxamine for the Treatment of Hemochromatosis

Launched by NATIONAL HEART, LUNG, AND BLOOD INSTITUTE (NHLBI) · Nov 3, 1999

Keywords

ClinConnect Summary

The purposes of this protocol are two-fold: 1) to determine whether deferoxamine, given subcutaneously on a regular basis, can indefinitely prevent the cardiac, endocrine and hepatic complications of transfusional hemochromatosis; and 2) to determine whether cardiac disease can be reversed by intensive intravenous treatment in patients who already have objective evidence of cardiac dysfunction. The clinical manifestations and course of patients who require regular blood transfusions is well established. Those with congenital anemias who require transfusions from birth develop cardiac diseas...

Gender

ALL

Eligibility criteria

• • INCLUSION CRITERIA
• • Patients studied under this protocol will be at risk for or have evidence of significant excess tissue iron.
• • Most patients will be on regular blood transfusion secondary to either congenital or acquired anemia.
• • The majority of patients have homozygous beta thalassemia.
• • Patients with sickle cell anemia will be included only when there is an absolute indication for regular blood transfusions (e.g., a history of stroke).
• • Twenty to thirty adults with acquired anemia and good long-term prognosis will be accepted for study if chelation can be initiated early in their transfusion history (less than 30-50 units).
• • EXCLUSION CRITERIA
• • Such patients will be excluded from study if they have diabetes or cardiac disease due to another cause (coronary artery or valvular heart disease).