Deferiprone (DFO) at the standard daily dose of 75 mg/kg was given to 13 transfusion-dependent patients with thalassemia in whom conventional desferrioxamine (DFX) therapy had proven ineffective and caused adverse side effects. In six patients, serum ferritin and alanine aminotransferase levels decreased significantly and urinary iron excretion increased. In seven patients in whom DFO administration alone was ineffectual, DFX was added at a daily dose of 40 to 50 mg/kg given subcutaneously for 7 to 10 days following transfusion. All patients exhibited a significant decrease in serum ferritin levels and an increase in urinary iron excretion, with alanine aminotransferase levels decreased in four patients. The combined DFX and DFO therapy could represent an effective alternative to conventional DFX therapy not only in nonresponding patients with thalassemia but also, by lowering to less than 25% the DFX dosage, in patients who exhibit important DFX-related side effects.
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