Efficacy of Deferasirox as an Oral Iron Chelator in Paediatric Thalassaemia Patients

Abstract

Introduction: Thalassaemia Major patients require frequent blood transfusion leading to iron overload. Excessive iron gets deposited in vital organs and leads to dysfunction of the heart, liver, anterior pituitary, pancreas, and joints. Our body has limited mechanism to excrete iron, so patients with iron overload and its complications need safe and effective iron chelation therapy. Aim: To assess the efficacy of Deferasirox (DFX) as an iron chelator, with specific reference to reduction in serum ferritin level. Materials and Methods: This is a prospective; observational study done in 45 multitransfused Thalassaemia Major Children receiving DFX therapy at registered Thalassaemia society Raipur Chhattisgarh. DFX was given in an initial dose of 20 mg/kg/day and according to response increased to a maximum of 40 mg/kg/ day. Serum ferritin level was estimated at time of registration and at every three monthly intervals (four times during study period). The primary end point of the study was change in serum ferritin level after 12 months of DFX therapy. Results: The mean serum ferritin before DFX therapy of all cases was 3727.02 ng/mL. After 12 months of mean dose of 38 mg/kg/ day of DFX, the mean decline in serum ferritin was 1207.11 ng/ mL (drop by 32.38%, p-value <0.001). Conclusion: DFX monotherapy has a good safety profile and effectively chelates total body iron in Thalassaemia major patients.

Keywords

• ferritin
• iron chelation
• management