Deferoxamine for the Treatment of Hemochromatosis
| Status: | Completed |
|---|---|
| Conditions: | Peripheral Vascular Disease, Cardiology, Hematology, Hematology, Diabetes |
| Therapuetic Areas: | Cardiology / Vascular Diseases, Endocrinology, Hematology |
| Healthy: | No |
| Age Range: | 4 - Any |
| Updated: | 4/5/2019 |
| Start Date: | April 22, 1985 |
| End Date: | November 9, 2015 |
Clinical Course of Patients With Transfusional Hemochromatosis on Deferoxamine
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.
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.
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 disease in their teens and their mean of
survival is only 17 years. Adults with acquired anemias begin to exhibit cardiac
manifestations of iron deposition after 100-200 units of packed red cells. Deferoxamine, when
given by the subcutaneous route, has been shown to reduce substantially the total iron burden
in thalassemic patients. Our results indicate that cardiac complications are delayed or
prevented. We plan to continue to follow our cohort of patients on optimal medical management
to determine if chelation alters disease outcome. Patients with heavy iron burdens who
already manifest cardiac disease will be chelated intensely to determine whether reducing the
iron burden is associated with reversal of cardiac complications.
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 disease in their teens and their mean of
survival is only 17 years. Adults with acquired anemias begin to exhibit cardiac
manifestations of iron deposition after 100-200 units of packed red cells. Deferoxamine, when
given by the subcutaneous route, has been shown to reduce substantially the total iron burden
in thalassemic patients. Our results indicate that cardiac complications are delayed or
prevented. We plan to continue to follow our cohort of patients on optimal medical management
to determine if chelation alters disease outcome. Patients with heavy iron burdens who
already manifest cardiac disease will be chelated intensely to determine whether reducing the
iron burden is associated with reversal of cardiac complications.
- 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).
We found this trial at
1
site
9000 Rockville Pike
Bethesda, Maryland 20892
Bethesda, Maryland 20892
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