Chelation therapy in patients with thalassemia using the orally active iron chelator deferiprone (L1).

Abstract

BACKGROUND AND OBJECTIVE: Excessive hemosiderosis is the main reason for the multi-organ failure observed in multitransfused patients. Deferiprone (1,2-dimethyl-3-hydroxy-pyridine-4-one, L1) is an orally active iron chelator mainly excreted via urine. We conducted a study in order to determine the efficacy and safety of L1 in Greek thalassemic patients. DESIGN AND METHODS: A group of 11 thalassaemic patients entered the study; L1, the Cipla formulation for deferiprone, at a daily dose of 75-100 mg/kg bw t.i.d. was used. After giving informed consent all patients were subjected to clinical examination and biological tests. RESULTS: All patients tolerated the L1 well; there were no significant side effects (except for slight gastrointestinal disturbances for the first days). The net urinary iron excretion ranged from 6.96 to 26.1 mg/24h. Serum ferritin declined within 4-6 months in most of the patients. INTERPRETATION AND CONCLUSIONS: The results suggest that L1 is a rather safe drug which decreases iron overload without causing any considerable side-effects in Greek thalassemics.