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

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; these data have been limited to case reports and small studies. Design and methods: To explore this observation in a large patient population, we report a post-hoc analysis evaluating hematologic response to deferasirox in a cohort of iron-overloaded myelodysplastic syndromes patients enrolled in the Evaluation of Patients’ Iron Chelation with Exjade® (EPIC) study using International Working Group 2006 criteria. Results: 247, 100 and 50 patients without concomitant myelodysplastic syndromes medication were eligible for erythroid, platelet and neutrophil response analyses. Erythroid, platelet and neutrophil responses were observed in 21.5% (53/247), 13.0% (13/100) and 22.0% (11/50) of 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. Reduction in labile plasma iron to <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.