Efficacy and safety of deferasirox in thalassemia major patients after repeated blood transfusion: A single-center study.

Background: Patients with β-thalassemia major (TM) require lifelong blood transfusions, leading to iron overload, where active intervention is required. Aims and Objectives: The aim of the study was to evaluate real-world efficacy and safety of deferasirox (DFX) in TM patients at a tertiary care teaching hospital. Materials and Methods: A prospective observational study was conducted at thalassemia ward in a tertiary care teaching hospital in Gujarat for a period of 15 months from April 2021 to July 2022. A total of 100 patients were analyzed during the study period. Patient's demographic profile, laboratory investigation, and treatment details were analyzed using descriptive statistics. Statistical analysis was done through the statistical software GraphPad Prism 9.4.1 (681). Results: A total of 100 patients were included in the study. A serial significant decrease in median serum ferritin levels was observed in >13 years age group after initiation of DFX. A significant increase in the median dose of DFX was observed at 12 months in the age group of 2-≤13 years as compared to baseline (P < 0.05). Adverse reactions observed during the study were increased bilirubin level, increased alanine aminotransferase, and increased serum creatinine, rashes with itching, and arthralgia. Conclusion: A greater reduction in median serum ferritin levels was observed in the adult age group compared to pediatric patients. [ABSTRACT FROM AUTHOR]