Comparison of Deferasirox and Desferrioxamine as Iron Chelators in Multi-Transfused Pediatric Patients with Beta Thalassemia Major

Abstract

Background: Beta thalassemia major is a severe transfusion-dependent hemoglobinopathy that leads to progressive iron overload, necessitating lifelong chelation therapy to prevent organ damage. Objective: To compare the efficacy, safety, and adherence rates of deferasirox and desferrioxamine in multi-transfused pediatric patients with beta thalassemia major. Methods: This comparative cross-sectional study was conducted in the Department of Pediatrics, POF Hospital Wah Cantt from 9 April 2025 to 10 July 2025. It included 85 pediatric patients with beta thalassemia major. Group A received oral deferasirox (20–40 mg/kg/day), and Group B received subcutaneous desferrioxamine (20–40 mg/kg/day, 5–7 nights/week). Results: Both chelators significantly reduced serum ferritin levels; however, mean reduction was greater with deferasirox (-842 ± 215 ng/mL) compared to desferrioxamine (-625 ± 240 ng/mL) (t = 4.07, p < 0.001). Adherence was significantly higher with deferasirox (90.7% vs. 66.7%, χ² = 7.61, p = 0.006). Gastrointestinal symptoms were more frequent in the deferasirox group (20.9% vs. 9.5%, p = 0.15), whereas infusion site pain was reported exclusively with desferrioxamine (33.3%, p < 0.001). No clinically significant hepatic or renal impairment was observed in either group. Conclusion: Both deferasirox and desferrioxamine are effective in managing transfusional iron overload in pediatric beta thalassemia major, but deferasirox offers superior ferritin reduction and better adherence due to its oral dosing and greater convenience.