Efficacy of deferasirox in pediatric patients with transfusion-dependent thalassemia: Single- center 2 year experience.

Abstract

Background: Children with thalassemia, dependent on regular blood transfusions, often have high serum ferritin levels due to the frequent transfusions, which can lead to long-term organ damage. Iron chelation therapy is necessary to reduce iron levels in the body. Lamphun Hospital has been supported with deferasirox from the National Health Security Project since 2018, but no studies have been conducted on the drug’s effectiveness.

Objectives:  To evaluate the effectiveness of deferasirox in reducing serum ferritin and the changes in serum ferritin levels in patients who had previously received other types of iron chelation drugs, compared with patients who received deferasirox as the first iron chelation therapy.

Methods:  This retrospective study collected data from thalassemia patients, dependent on blood transfusions, at Lamphun Hospital, with serum ferritin levels greater than 1000 ng/mL, who had received deferasirox for 2 years. A total of 37 patients were included, with dosage and serum ferritin levels measured every 3 months, along with monitoring of adverse effects from the medication.

Results:  After 12 and 24 months of receiving deferasirox, the serum ferritin levels in all patients did not significantly differ from baseline (p values 0.962 and 0.724, respectively). In patients who had previously received other iron chelation drugs, the serum ferritin levels did not differ significantly from baseline (p values 0.501 and 0.686, respectively). Similarly, patients receiving deferasirox for the first time showed no significant difference in serum ferritin levels from baseline (p values 0.301 and 0.936, respectively). The most common adverse effect was transaminitis.

Conclusion:  Deferasirox is effective in maintaining serum ferritin levels without increasing them during the first 24 months of use in pediatric thalassemia patients dependent on regular blood transfusions.