Study on the role of iron chelators in the management of iron overload among transfusion-dependent thalassemia (TDT) pediatric patients

Abstract

This study aims to investigate the efficacy and safety of Deferasirox, an oral iron chelator, in reducing iron burden in pediatric patients with transfusion-dependent beta-thalassemia. Thalassemia syndromes, particularly beta-thalassemia, are inherited hemoglobin disorders requiring regular blood transfusions, leading to iron overload and subsequent complications. Effective management of iron overload is crucial to prevent morbidity and mortality. It was a descriptive observational study on Children between the ages of 2 years and 12 years who present with transfusion-dependent thalassemia and areon blood transfusion and develop iron overload, which is evaluated by serum ferritin levels of more than 2000mcg/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 the study; significant reductions were observed in mean serum ferritin levels (2,388 mcg/dl to 2,054 mcg/dl, p=0.0009), transferrin saturation (70.45% to 64.32%, p=0.00005), and serum transaminases (44.55 U/L to 40.27 U/L, p=0.003) at 6 months. Cardiac MRI T2* increased from 19.55 ms to 22.95 ms (p=0.045) at the end of 6 months and at the end of 12 months from 19.55 to 28.23 (p=0.0016), and LIC reduced from 20.73 mg Fe/g dw to 11.59 mg Fe/g dw (p=0.00005). Pancreatic T2 improved from 15.96 ms to 20.23 ms at 12 months (p=0.007). A transient increase in serum creatinine was observed at 6 months from 0.64+/-0.14 mg/dL to 0.7+/-0.13mg/dL(p=0.009), which returned to normal at the end of 12 months to 0.63 mg/dL, no additional therapy-related adverse events were reported. Deferasirox has demonstrated significant efficacy in reducing iron overload in pediatric 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.