Comparison between deferoxamine and deferiprone (L1) in iron-loaded thalassemia patients

Introduction: Iron-chelating therapy with deferoxamine in patients with tha lassemia major has dramatically improved the prognosis of this disease. How ever, the limitations of this treatment have stimulated the design of alter native orally active iron chelators. Objective: To compare the effectivenes s and safety of, and compliance with, oral deferiprone (LI), and deferoxami ne, in thalassemia major patients. Methods: All patients were followed up i n one center in Lebanon. Sixteen patients were on Ll (75 mg/kg/d), and 40 p atients on subcutaneous deferoxamine (20-50 mg/kg/d). Serum ferritin level, urinary iron excretion (UIE) and side effects were monitored over a two ye ar period. Results: Patients on Ll had an initial serum ferritin concentrat ion of 3663 +/- 566 mug/l (mean +/- SEM), that dropped to 2599 +/- 314 at 6 months (p <0.02; paired t-test), and stabilised at that level over the 24 months follow up. Patients on deferoxamine had an initial mean serum ferrit in concentration of 3480 +/- 417 (NS compared to the L1 group), which dropp ed gradually to 3143 +/- 417 (p <0.05) and 2819 +/- 291 (p <0.02) at 6 and 24 months, respectively. The most common adverse reactions associated with L1 were arthralgia and nausea, but they did not necessitate stopping the dr ug. Conclusion: L1 had comparable efficacy as deferoxamine with minimal sid e effects and better compliance. Provided long term side effects are not en countered, L1 seems to be a valuable lb alternative iron chelator for patie nts unable or unwilling to use deferoxamine effectively.