Hematologic responses to deferasirox therapy in transfusion-dependent patients with myelodysplastic syndromes

Norbert Gattermann, Carlo Finelli, Matteo Della Porta, Pierre Fenaux, Michael Stadler, Agnes Guerci-Bresler, Mathias Schmid, Kerry Taylor, Dominique Vassilieff, Dany Habr, Andrea Marcellari, Bernard Roubert, Christian Rose

Research output: Contribution to journalArticlepeer-review


Background Reductions in transfusion requirements/improvements in hematologic parameters have been associated with iron chelation therapy in transfusion-dependent patients, including those with myelodysplastic syndromes; data on there reductions/improvements have been limited to case reports and small studies. Design and Methods To explore this observation in a large population of patients, we report a post-hoc analysis evaluating hematologic response to deferasirox in a cohort of iron-overloaded patients with myelodysplastic syndromes enrolled in the Evaluation of Patients' Iron Chelation with Exjade® (EPIC) study using International Working Group 2006 criteria. Results Two-hundred and forty-seven, 100 and 50 patients without concomitant medication for myelodysplastic syndromes were eligible for analysis of erythroid, platelet and neutrophil responses, respectively. Erythroid, platelet and neutrophil responses were observed in 21.5% (53/247), 13.0% (13/100) and 22.0% (11/50) of the patients after a median of 109, 169 and 226 days, respectively. Median serum ferritin reductions were greater in hematologic responders compared with non-responders at end of study, although these differences were not statistically significant. A reduction in labile plasma iron to less than 0.4 μmol/L was observed from week 12 onwards; this change did not differ between hematologic responders and non-responders. Conclusions This analysis suggests that deferasirox treatment for up to 1 year could lead to improvement in hematologic parameters in some patients with myelodysplastic syndromes.

Original languageEnglish
Pages (from-to)1364-1371
Number of pages8
Issue number9
Publication statusPublished - 2012


  • Deferasirox
  • Hematologic response
  • Iron chelation therapy
  • Iron overload
  • Myelodysplastic syndromes

ASJC Scopus subject areas

  • Hematology
  • Medicine(all)

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