Long-term efficacy and safety of deferasirox

Blood Rev. 2008 Dec:22 Suppl 2:S35-41. doi: 10.1016/S0268-960X(08)70007-9.

Abstract

Deferasirox is an oral iron chelator, with a long half-life, that can be given once daily, because it provides a 24-hour chelation. Several phase II trials and a pivotal phase III trial have established that, in transfusion-dependent patients with beta-thalassaemia major, deferasirox has a similar efficacy to previously available deferoxamine. A deferasirox dose of 20 mg/kg/day stabilizes serum ferritin levels and liver iron concentration, while a dose of 30 mg/kg/day reduces serum ferritin and liver iron concentration and achieves negative iron balance. Efficacy has also been shown across various transfusion-dependent anaemias including myelodysplastic syndromes, sickle cell disease, and other rare transfusion-dependent anaemias. Deferasirox is generally well tolerated, with the most common adverse events being gastrointestinal disturbances and rash. Longer-term studies with a median follow-up of 3.5 years have confirmed the efficacy and safety of deferasirox. It is recommended that patients treated with deferasirox are monitored regularly for iron status and adverse events, to ensure that an effective and tolerable iron chelation regimen is established for each individual patient.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Adolescent
  • Benzoates / administration & dosage
  • Benzoates / adverse effects*
  • Benzoates / therapeutic use
  • Child
  • Child, Preschool
  • Clinical Trials, Phase II as Topic
  • Clinical Trials, Phase III as Topic
  • Deferasirox
  • Humans
  • Iron Chelating Agents / administration & dosage
  • Iron Chelating Agents / adverse effects*
  • Iron Chelating Agents / therapeutic use
  • Iron Overload / drug therapy*
  • Triazoles / administration & dosage
  • Triazoles / adverse effects*
  • Triazoles / therapeutic use

Substances

  • Benzoates
  • Iron Chelating Agents
  • Triazoles
  • Deferasirox