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

Abstract

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.

• Authors: 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
• Journal: Haematologica (Haematologica) Vol. 97 Issue 9 Pg. 1364-71 (Sep 2012) ISSN: 1592-8721 [Electronic] Italy
• PMID: 22419577 (Publication Type: Clinical Trial, Phase III, Journal Article, Multicenter Study, Research Support, Non-U.S. Gov't)

Chemical References

• Benzoates
• Hemoglobins
• Iron Chelating Agents
• Triazoles
• Deferasirox

Topics

• Adolescent
• Adult
• Aged
• Aged, 80 and over
• Benzoates (therapeutic use)
• Blood Platelets (drug effects, pathology)
• Child
• Deferasirox
• Female
• Follow-Up Studies
• Hemoglobins (analysis)
• Humans
• Iron Chelating Agents (therapeutic use)
• Iron Overload (drug therapy, etiology, mortality)
• Male
• Middle Aged
• Myelodysplastic Syndromes (complications, therapy)
• Neoplasm Recurrence, Local (drug therapy, etiology, mortality)
• Neutrophils (drug effects, pathology)
• Prognosis
• Prospective Studies
• Survival Rate
• Transfusion Reaction
• Triazoles (therapeutic use)
• Young Adult