Long-term efficacy and safety of deferasirox

Research output: Contribution to journalArticle

53 Citations (Scopus)

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 β-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.

Original languageEnglish
JournalBlood Reviews
Volume22
Issue numberSUPPL. 2
DOIs
Publication statusPublished - Dec 2008

Fingerprint

Iron
Safety
Ferritins
Anemia
Deferoxamine
Liver
beta-Thalassemia
Myelodysplastic Syndromes
Sickle Cell Anemia
Chelating Agents
Exanthema
Serum
Half-Life
deferasirox

Keywords

  • Deferasirox
  • Iron chelation therapy
  • Iron overload
  • Long-term efficacy
  • Safety

ASJC Scopus subject areas

  • Hematology
  • Oncology

Cite this

Long-term efficacy and safety of deferasirox. / Cappellini, Maria Domenica.

In: Blood Reviews, Vol. 22, No. SUPPL. 2, 12.2008.

Research output: Contribution to journalArticle

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