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

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 beta-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 microg/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 beta-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.

• Authors: Ali Taher, Maria D Cappellini, Elliott Vichinsky, Renzo Galanello, Antonio Piga, Tomasz Lawniczek, Joan Clark, Dany Habr, John B Porter
• Journal: British journal of haematology (Br J Haematol) Vol. 147 Issue 5 Pg. 752-9 (Dec 2009) ISSN: 1365-2141 [Electronic] England
• PMID: 19764988 (Publication Type: Journal Article, Meta-Analysis)

Chemical References

• Benzoates
• Iron Chelating Agents
• Triazoles
• Ferritins
• Creatinine
• Deferasirox

Topics

• Adolescent
• Adult
• Anemia (therapy)
• Benzoates (administration & dosage, adverse effects, therapeutic use)
• Child
• Child, Preschool
• Creatinine (blood)
• Deferasirox
• Drug Administration Schedule
• Female
• Ferritins (blood)
• Humans
• Iron Chelating Agents (administration & dosage, adverse effects, therapeutic use)
• Iron Overload (blood, drug therapy, etiology)
• Male
• Middle Aged
• Retrospective Studies
• Transfusion Reaction
• Treatment Outcome
• Triazoles (administration & dosage, adverse effects, therapeutic use)
• Young Adult