The Efficacy and Safety of Deferasirox Monotherapy as a Second-Line Treatment in Transfusion-Dependent Thalassemia with Iron Overload

Deferasirox (DFX) is an oral iron chelator for thalassemia patients with iron overload. DFX was FDA-approved as a first-line treatment for chronic iron overload. In Thailand, DFX was indicated as second-line therapy for patients unresponsive to deferiprone. <b>Objectives</b>: This study aimed to investigate the efficacy and safety of DFX monotherapy. <b>Methods</b>: All transfusion-dependent thalassemia patients who received second-line DFX monotherapy were identified from the thalassemia registry between May 2007 and May 2022. The primary endpoint was the change in body iron stores, measured by serum ferritin at week 24. At treatment end, patients with a serum ferritin (SF) level < 1000 ng/mL in transfusion-dependent thalassemia (TDT) were categorized as the ferritin response group. Multivariate analysis identified factors driving group differences. <b>Results</b>: Forty-two patients were enrolled with a mean age of 35.5 (13-57) years. Of these, 73.81% had beta-thalassemia. The median initial DFX dose was 20.26 (17.85-22.22) mg/kg/day, with a median treatment follow-up of 2 (1.80-2.45) years. Median SF was decreased from 2516 (1712 to 3065) ng/mL to 1027.5 (598-1867) ng/mL (<i>p</i> < 0.001). Of 21 (50%) patients in the ferritin response group, independent factors were age > 15 years and lower initial SF, with OR = 7.13 (95% CI 1.05-48.49, <i>p</i> = 0.045) and OR = 0.93 (95% CI 0.87-1.00, <i>p</i> = 0.039). The most common adverse events were gastric irritation symptoms (11.90%). <b>Conclusions</b>: Deferasirox is an effective oral iron chelator for thalassemia, with manageable side effects. Half of patients reached target SF levels. Adults (>15 years) with lower initial SF levels had a better response to DFX.