Hematologic Diseases Clinical Trial
To determine whether deferoxamine prevented the complications of transfusional iron overload.
BACKGROUND:
The prognosis of congenital or long-term anemia was formerly limited by the complications of
blood transfusion, splenectomy, or infection, problems now largely overcome by sophisticated
clinical care. Lifespan is now determined by the rate of myocardial iron deposition, with
death occurring from cardiac failure or arrhythmia, usually between the ages of 15 and 25.
Endocrine complications and hepatic enlargement are also evident by this age. Deferoxamine
increases urinary iron excretion and is the only chelator currently available for chronic
administration. Daily administration of deferoxamine results in negative iron balance in
most patients by the age of 10; this study was designed to determine whether the onset of
cardiac complications was delayed and life prolonged by iron removal.
This trial began in 1978. Its forerunner was a study involving both deferoxamine and
ascorbic acid. Although ascorbic acid promotes iron removal, its administration was followed
by cardiac deterioration in several patients. In this study, patients receiving subcutaneous
deferoxamine were randomized to receive either ascorbic acid or placebo, thereby providing a
controlled test of this agent in treatment of iron overload. Sixty-five patients with
homozygous beta-thalassemia participated in the long-term chelation trial. Of these, 49 were
randomized to the ascorbic acid trial.
Several noninvasive techniques have been developed to evaluate organ function in
iron-overloaded patients, thereby facilitating the assessment of chelation therapy. These
techniques included chest x-rays, electrocardiograms, echocardiograms, and 24-hour Holter
monitoring to assess cardiac function. Liver function was evaluated by standard liver
function tests, CAT scan, and live biopsy. During the last six years of the study, hepatic
iron stores were measured magnetically with a dual channel superconducting
quantum-interference susceptomer. Endocrine function was also assessed by standard tests.
DESIGN NARRATIVE:
All patients received subcutaneous deferoxamine and iron removal was determined by
measurement of serum ferritin and periodic non-invasive measurements of liver iron
concentration. Clinical status was evaluated by non-invasive testing of cardiac and
endocrine function.
The study completion date listed in this record was inferred from the last publication
listed in the Citations section of this study record.
;
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