Results of Deferasirox for Regular Transfusion in Thalassemia and Non-thalassemia Pediatric Patients

Abstract

          Thalassemia is the most common genetic blood disease in Thailand. Currently based on their clinical severity and transfusion requirement lead to complication of iron overload. Iron chelation need to effectively manage, closed monitoring serum ferritin levels and adverse effects of drug. Objective study design for Deferasirox (DFX) used in transfusion dependent thalassemia (TDT) and non thalassemia anemia with iron overloadin children. Retrospective review data in thalassemia clinic of Khon Kaen hospital since Sep. 2009 – Dec. 2019, ten years in 25 patients. Analytical serum ferritin levels by Wilcoxon signed rank test and serum aspartate transaminase (AST), serum alanine transaminase (ALT), serum creatinine by paired t-test.

          Monotherapy iron chelation by Deferaxirox in TDT these on regular transfusion every 3-4 week, ferritin at start was 1,359 ng/ml and 1,593 ng/ml in the end. Another group, non thalassemia anemia ferritin at start was 1,092 ng/ml and end at 1,309 ng/ml in one year. DFX can treat iron overload in both groups, serum  ferritin were not increased significantly. In addition to appropriate dose adjustment for chelation 20-40 mg/kg/day more effective dose than lower 20 mg/kg/day in both groups. No severe adverse effects over 10 years of study. Finally, DFX can used for monotherapy iron chelation in both groups were effectively and safely in pediatrics.