Evaluating the efficacy and safety of deferasirox in management of iron overload in transfusion-dependent beta-thalassemia pediatric patients

The objective of this study was to evaluate the efficacy and safety of Deferasirox (DFX) in managing iron overload in pediatric patients with transfusion-dependent beta-thalassemia over a period of 12-months. It was a descriptive observational study on Children between the age of 2 years to 12 years who present with transfusion dependent thalassemia and areon blood transfusion and develop iron overload, which is evaluated by serum ferritin levels more than 2000 mcg/l are administered iron chelator Deferasirox (14 mg/kg/d) and patients are evaluated for myocardial, hepatic, pancreatic iron burden and conditions of iron toxicity with the help of Cardiac MRI T2, LIC (Liver Iron Concentration), MRI T2 Pancreas, LVEF (Left Ventricular Ejection Fraction). A total of 22 patients enrolled in study a significant reduction in mean serum ferritin levels was seen at the end of 6 months (p<0.0005) as compared to baseline, A significant reduction in mean transferrin saturation at the end of 6 months from 70.45% saturation to 64.32% saturation (p<0.00005), A significant reduction in mean serum transaminases from 44.55 to 40.27 at the end of 6 months (p=0.003), A significant reduction in serum total bilirubin from mean 1.05 mg/dl to 0.54mg/dl was seen at the end of 12 months (p=0.000019). A significant increase in mean cardiac T2 from 19.55 to 22.95 was seen at the end of 6 months (p=0.0016) and at the end of 12 months from 19.55 to 28.23 (p=0.045). A significant reduction in mean direct bilirubin was seen from 0.46 to 0.45 (p=0.29), A significant reduction in mean LIC (Liver Iron Concentration) from 20.73 mg Fe/g dw to 11.59 mg Fe/g dw was seen at the end of 6 months (p=0.00005). A significant increase in mean pancreatic T2 from 15.96 to 20.23 at the end of 12 months (p=0.007). The patients had not experienced any therapy-related adverse events. Deferasirox has demonstrated significant efficacy in reducing iron overload in paediatric patients with transfusion-dependent beta-thalassemia over a 12-month period. The substantial improvements in serum ferritin, cardiac MRI T2*, LIC, transferrin saturation, and pancreatic T2, coupled with its excellent safety profile, support the use of DFX as a cornerstone in the management of iron overload in this vulnerable population.