Universitas Indonesia Conferences, Asian Federation for Pharmaceutical Sciences (AFPS) 2019

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Effectiveness and safety analysis of combination iron chelation with mono-therapy in pediatric thalassemia major in Cipto Mangunkusumo hospital 2014 – 2018
Dewi Sharon Simorangkir, Nafrialdi Nafrialdi, Pustika Amalia Wahidiyat, vivian soetikno

Last modified: 2019-06-16


Background:Regular blood transfusion is the main supportive therapy for thalassemia major patients, to maintaining haemoglobin 9 – 10 gr%, and suppresses the ineffective erythropoiesis process. But repeated transfusions are followed by new problem, namely the burden of excess iron which accumulates in the body's tissues. Iron chelation is the only way to maintain and balance levels of iron. Objective:This study aims to evaluate effectiveness, safety and to analyse cost of iron chelation combination (deferoxamine - deferiprone and deferipron-deferasirox) with deferiprone mono-therapy dose ≥ 90 mg/kg/day in pediatric thalassemia major patient with serum ferritin levels ≥ 2500 µg/L. Material and Methods: Subjects' medical records were observed within 6 or 12 months to analyze the relationship of therapeutic effectiveness, safety between two regimens. The outcome effectiveness was assessed by a decrease in serum ferritin ≥ 500 µg/L. Pharmacoeconomic analysis depends on the results of the statistical analysis of the effectiveness therapy. Results:Medical records from 46 subjects using the DFO-DFP and DFP-DFX combination regimens and 80 subjects using DFP ≥ 90 mg/kg/day were evaluated. After 6 or 12 months there was a decrease in serum ferritin of 16 subjects in the combination group (34.7%) and 23 subjects in the mono-therapy group. The effectiveness of the therapy did not show a significant difference between the two regimens (P = 0.481). In the combination group 17 subjects experienced adverse effect (21.2%) and in the mono-therapy group 9 subjects (19.5%, P = 0.822). Cost minimization analysis was chosen as a pharmacoeconomic study because statistically the effectiveness of the two interventions was assumed to be equal. The average cost per patient within 6 and 12 months mono-therapy treatment was cheaper compared to combination regimen. Conclusion: There were no significant differences in effectiveness and safety between two regimens.