Importance of optimal dosing ≥30mg/kg/d during deferasirox treatment: 2.7-yr follow-up from the ESCALATOR study in patients with β-thalassaemia

Ali Taher*, Mohsen S. Elalfy, Kusai Al Zir, Shahina Daar, Abdullah Al Jefri, Dany Habr, Ulrike Kriemler-Krahn, Ali El-Ali, Bernard Roubert, Amal El-Beshlawy

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

31 Citations (Scopus)

Abstract

Following 1-yr deferasirox therapy in the ESCALATOR study, 57% of previously chelated patients with β-thalassaemia achieved treatment success (maintenance of or reduction in liver iron concentration (LIC) vs. baseline LIC). Seventy-eight per cent had dose increases at median of 26wk, suggesting that 1-yr results may not have reflected full deferasirox efficacy. Extension data are presented here. Deferasirox starting dose was 20mg/kg/d (increases to 30/40mg/kg/d permitted in the core/extension, respectively). Efficacy was primarily assessed by absolute change in LIC and serum ferritin. Overall, 231 patients received deferasirox in the extension; 67.4% (P<0.0001) achieved treatment success. By the end of the extension, 66.2% of patients were receiving doses ≥30mg/kg/d. By the end of the 1-yr extension, mean LIC had decreased by 6.6±9.4mg Fe/g dw (baseline 19.6±9.2; P<0.001) and median serum ferritin by 929ng/mL (baseline 3356; P<0.0001). There was a concomitant improvement in liver function markers (P<0.0001). Fewer drug-related adverse events were reported in extension than core study (23.8% vs. 44.3%). Doses ≥30mg/kg/d were generally required because of high transfusional iron intake and high baseline serum ferritin levels, highlighting the importance of administering an adequate dose to achieve net negative iron balance.

Original languageEnglish
Pages (from-to)355-365
Number of pages11
JournalEuropean Journal of Haematology
Volume87
Issue number4
DOIs
Publication statusPublished - Oct 2011
Externally publishedYes

Keywords

  • Deferasirox
  • Efficacy
  • Iron chelation therapy
  • Iron overload
  • β-thalassaemia

ASJC Scopus subject areas

  • Hematology

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