Combination therapy of deferasirox and deferoxamine shows significant improvements in markers of iron overload in a patient with β‐thalassemia major and severe iron burden

Abstract

Iron overload is a common complication of patients with β-thalassemia major (TM). Despite the availability of three iron chelators, deferoxamine (DFO), deferiprone (DFP), and deferasirox (DFX), some patients fail to respond adequately to monotherapy with any of them. We report a case of TM who had refractory severe iron overload and was successfully and safely chelated with the combination of DFX with DFO. A 40-year-old male with β-TM, who had been regularly transfused from the age of 2, had been administered in the past iron chelation with DFO, DFP, and DFX monotherapy, without major improvement on his iron overload status. Liver and cardiac magnetic resonance imaging (MRI) revealed severe iron overload, while serum ferritin was persistently greater than 2500 μg/L. After the patient gave informed consent, he was administered combination therapy of DFX at 30 mg/kg/day for 7 days per week and DFO at 2500 mg/day for 4 days every week and routinely followed up for compliance. Eighteen months later, serum ferritin was reduced to 680 μg/L, while both liver and cardiac MRI T2* values improved, reflecting lower iron overload. The combination regimen was well tolerated and no adverse events were documented. This is the first official report of simultaneous daily administration of the two iron chelators DFX and DFO that demonstrates the beneficial effect of the combination on heart and liver hemosiderosis. If our observation is confirmed in more patients, this combination could constitute a useful option in tailoring individual chelation therapy for β-TM patients with iron overload.