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


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Article type :

Original Article

Author :

Shaik Mohammad Abubakar, Krishnakanth K, Jhansi Vani Chopra, Elizabeth B

Volume :

11

Issue :

3

Abstract :

Aim and Objective: 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. Materials and Methods: 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). Results: 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 Conclusion: 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.

Keyword :

Beta-thalassemia major, Serum ferritin, Iron overload, Cardiac MRI T2*, Liver iron concentration (LIC)