Safety and efficacy of combination of oral iron chelators in thalassemia major patients

Abstract

Background: To assess the safety and efficacy of combination of oral iron chelation therapy in transfusion dependent thalassemia patients and to compare it with other regimes. Method: 150 transfusion dependent thalassemia patients with iron overload were recruited for a prospective, case control study. They were divided into 4 groups based on their chelation regime. The drugs used were deferasirox (DFX) alone, deferiprone (DFP) alone, DFP+DFX in combination, parenteral desferrioxamine (DFO) with either DFX or DFP with 50, 15, 70 and 15 in each group respectively. Doses used were DFX-30 mg/kg, DFP- 75 mg/kg/day, DFP+DFX-50 +30 mg/kg/day, DFO+DFP-25 mg/kg/d+50 mg/kg/day and DFO+DFX-25 mg/kg/d +30 mg/kg/d daily. Base line haematological, hepatic and renal profile and S. ferritin samples were collected and analyzed. Patients were monitored monthly for compliance and side effects. 30 patients were excluded during the course of study. Tests were repeated after 18 months period and the results of 120 patients were compared between the groups and statistically analyzed. Results: There was statistically significant reduction of S. ferritin in both groups taking oral combination therapy and parenteral therapy (p<0.0001). There were no serious hematological, renal/hepatic side effects in any groups. Conclusions: The oral combination therapy of iron chelators is safer and was well tolerated by all the patients and is similarly efficacious to the parenteral therapy in lowering S. ferritin.