Efficacy and safety of deferasirox doses of >30 mg/kg per d in patients with transfusion‐dependent anaemia and iron overload

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Summary: The highest approved dose of deferasirox is currently 30 mg/kg per d in many countries; however, some patients require escalation above 30 mg/kg per d to achieve their therapeutic goals. This retrospective analysis investigated the efficacy (based on change in serum ferritin levels) and safety of deferasirox >30 mg/kg per d in adult and paediatric patients with transfusion‐dependent anaemias, including β‐thalassaemia, sickle cell disease and the myelodysplastic syndromes. In total, 264 patients pooled from four clinical trials received doses of >30 mg/kg per d; median exposure to deferasirox >30 mg/kg per d was 36 weeks. In the overall population there was a statistically significant median decrease in serum ferritin of 440 μg/l (P < 0·0001) from pre‐dose‐escalation to the time‐of‐analysis; significant decreases were also observed in adult and paediatric patients, as well as β‐thalassaemia patients. The adverse event profile in patients who received deferasirox doses of >30 mg/kg per d was consistent with previously published data. There was no worsening of renal or liver function following dose escalation. Deferasirox >30 mg/kg per d effectively reduced iron burden to levels lower than those achieved prior to dose escalation in patients with transfusion‐dependent anaemias. This has important implications for patients who are heavily transfused and may require higher doses to reduce body iron burden.

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