Deferasirox in Chelation Naive Children with Transfusional Iron Overload in Basra, Iraq: A Two-Year Single Center Study

Background: Effective management of iron overload in patients receiving longterm blood transfusion requires assessment and monitoring of both severity of iron overload and excessive iron chelation. We aimed to evaluate the efficacy and safety of Deferasirox (DFX) in chelation naive patients with transfusion dependent thalassemia and sickle cell disease. Methods: Chelation naive patients with transfusion dependent thalassemia (TDT) and sickle cell disease (SCD), aged 2-5 years, who had received DFX for at least 2 years were enrolled. Safety of DFX was assessed based on alanine aminotransferase (ALT) and serum creatinine levels, while efficacy was assessed based on serum ferritin levels. Results: The study included 93 chelation naive patients; 64 (68.82%) with TDT and 29 (31.18%) with SCD. Mean SF levels declined significantly from 2297.40±1037.46 ng/ml at baseline to 1700.65±1038.7 ng/ml at the end of the study. The efficacy of DFX was increased with increasing DFX dose to ≥30 mg/ kg/day. The most commonly observed adverse effects were abdominal pain in 20 (21.50%), nausea in 8 (8.60%), and vomiting in 4 (4.30%) patients, which were transient and mild to moderate in severity. A minor, although significant change in the mean serum creatinine was reported after 24 months of treatment with DFX compared to the baseline (75.72±7.87 vs. 71.59±11.14 mmol/L) (P<0.05). The mean ALT (17.69±1.44 vs. 21.75±3.37 U/L) and median height-SDS at the end of the study did not show significant changes compared to the baseline levels. Conclusion: Although Deferasirox was found to be a safe, tolerable, and effective drug for reducing iron overload, monitoring safety markers and serum ferritin to ensure appropriate drug dosing can improve its efficacy.