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

• Published in: Haematologica (Roma) 2012-09, Vol.97 (9), p.1364-1371
• Main Authors: GATTERMANN, Norbert, FINELLI, Carlo, MARCELLARI, Andrea, ROUBERT, Bernard, ROSE, Christian, DELLA PORTA, Matteo, FENAUX, Pierre, STADLER, Michael, GUERCI-BRESLER, Agnes, SCHMID, Mathias, TAYLOR, Kerry, VASSILIEFF, Dominique, HABR, Dany
• Format: Article
• Language: English
• Subjects: Adolescent; Adult; Aged; Aged, 80 and over; Benzoates - therapeutic use; Biological and medical sciences; Blood Platelets - drug effects; Blood Platelets - pathology; Child; Female; Follow-Up Studies; Hematologic and hematopoietic diseases; Hemoglobins - analysis; Humans; Iron Chelating Agents - therapeutic use; Iron Overload - drug therapy; Iron Overload - etiology; Iron Overload - mortality; Leukemias. Malignant lymphomas. Malignant reticulosis. Myelofibrosis; Male; Medical sciences; Metabolic diseases; Metals (hemochromatosis...); Middle Aged; Myelodysplastic Syndromes - complications; Myelodysplastic Syndromes - therapy; Neoplasm Recurrence, Local - drug therapy; Neoplasm Recurrence, Local - etiology; Neoplasm Recurrence, Local - mortality; Neutrophils - drug effects; Neutrophils - pathology; Original and Brief Reports; Other metabolic disorders; Prognosis; Prospective Studies; Survival Rate; Transfusion Reaction; Triazoles - therapeutic use; Young Adult
• ISSN: 0390-6078; 1592-8721
• DOI: 10.3324/haematol.2011.048546

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. 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. 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. This analysis suggests that deferasirox treatment for up to 1 year could lead to improvement in hematologic parameters in some patients with myelodysplastic syndromes.