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

Ali Taher, Maria D. Cappellini, Elliott Vichinsky, Renzo Galanello, Antonio Piga, Tomasz Lawniczek, Joan Clark, Dany Habr, John B. Porter

Research output: Contribution to journalArticlepeer-review

Abstract

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 g/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.

Original languageEnglish
Pages (from-to)752-759
Number of pages8
JournalBritish Journal of Haematology
Volume147
Issue number5
DOIs
Publication statusPublished - Dec 2009

Keywords

  • Deferasirox
  • Efficacy
  • Safety
  • Transfusion-dependent

ASJC Scopus subject areas

  • Hematology

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