A Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial of Deferasirox (Exjade®) in Patients with Low/Intermediate-1 Risk MDS and Transfusional Iron Overload

Abstract 4854 Background Approximately 60–80% of patients with myelodysplastic syndromes (MDS) require ongoing red blood cell (RBC) transfusions due to impaired hematopoiesis. Iron chelation therapy has been extensively studied in thalassemia major patients; however, there are fewer data available on the efficacy and safety in transfusion-dependent patients with MDS who have a completely different clinical scenario (Angelucci E and Di Tucci AA. Leuk Res 2009;33:743–4). Increasing evidence is being reported in the literature on the rate and site of iron accumulation (Di Tucci AA et al. Haematologica 2008;93:1385–8), on the impact of transfusion dependency and iron overload on survival (Malcovati L. Leuk Res 2007;31:S2–6, Sanz G et al. Blood 2008;112(11):Abst 640) and on the efficacy of deferasirox (Exjade®) in removing iron in MDS patients. Nevertheless, the possibility to modify clinical outcome by iron chelation in MDS patients is still debated (DeLoughery TG. Am J Hematol 2009;84:263–4). Recent retrospective and prospective non-randomized data (Rose C et al. Blood 2007:110(11);Abst 249) suggest that iron chelation could have a positive impact. A Phase III, prospective, randomized, double-blind, placebo-controlled, parallel-group design clinical trial has therefore been planned to assess the effects of iron chelation therapy with deferasirox on clinical outcomes in patients with MDS (Low/Int-1 risk) and transfusional iron overload. Methods For inclusion, patients must be chelation-naïve, aged ≥18 years with Low/Int-1 risk MDS and serum ferritin (SF) levels of >1000– Conclusions This prospective multicentre study has been designed to investigate the clinical benefit of chelation therapy with deferasirox in patients with MDS, a matter that has assumed increasing relevance during recent years. Disclosures Bowen: Novartis: Honoraria, Research Funding. Magalhães:Novartis: Honoraria, Membership on an entity's Board of Directors or advisory committees; Janssen-Cilag: Research Funding. Lawniczek:Novartis Pharma AG: Employment. Douma:Novartis Pharma AG: Employment. Jakobs:Novartis Pharma AG: Employment.