Introduction: Cardiac disease due to iron overload remains the main cause of death in beta-thalassemia major (TM). Successful iron chelation is therefore essential for the optimal management of thalassemia. Deferoxamine (DFO) is still the first-line chelating agent in these patients, but compliance is a major hindrance to achieving optimal therapeutic results. Some studies have suggested that deferiprone (L1), an orally active chelating drug, provides superior cardioprotection compared to deferoxamine. The minimum time for this effect is over 6 months. We compared L1 with DFO, in beta-thalassemia major patients for cardioprotection during a one-yare period.
Methods: A prospective clinical study was performed on 48 patients assigned to two equal groups. Group 1 patients only received deferiprone and group 2 patients received deferoxamine. We compared serum ferritin and left ventricular ejection fraction (LVEE) of the two groups after one year.
Results: Left ventricular ejection fraction and serum ferritin were not significantly different between the two groups at the end of study (mean LVEE±SD: 62.5±5.5 in group 1, 61.2±5.8 in group 2, P=0.43; mean serum ferritin±SD: 2354±1200 ng/cc in group 1, 2284±1244 ng/cc in group2, P=0.92). Transient gastrointestinal symptoms were the most frequent adverse effects in deferipronetreated patients and there were no episodes of agranulocytosis.
Conclusions: Monotherapy with L1 during a one-year period offered no superior cardioproteetion compared to DFO. Future longitudinal studies with longer follow-ups are warranted.