Deferasirox in iron-overloaded patients with transfusion-dependent myelodysplastic syndromes: Results from the large 1-year EPIC study.

Gattermann, N.; Finelli, C.; Porta, M. D.; Fenaux, P.; Ganser, A.; Guerci Bresler, A.; Schmid, M.; Taylor, K.; Vassilieff, D.; Habr, D.; Domokos, G.; Roubert, B.; Rose, C.; Agaoglu, L.; Alimena, G.; Alonso, D.; Ame, S.; Angelucci, E.; Businco, A.; Arrizabalaga, B.; Athanasiou Metaxa, M.; Augustson, B.; Aydinok, Y.; Baba, A.; Baccarani, M.; Beck, J.; Beris, P.; Beyne Rauzy, O.; Birgens, H.; Bordessoule, D.; Borgna, Caterina; Bosly, A.; Bouabdallah, K.; Bowden, D.; Bowen, D.; Bron, D.; Cappellini, M. D.; Capra, M.; Cartron, G.; Cazzola, M.; Chalkias, C.; Chan, L. L.; Chancharunee, S.; Chapman, C.; Charoenkwan, P.; Chasapopoulou, E.; Cheze, S.; Chuansumrit, A.; Cianciulli, P.; Dauriac, C.; Delforge, M.; Dölken, G.; Dombret, H.; Duyster, J.; Economopoulos, T.; Ehninger, G.; Elalfy, M.; El Beshlawy, A.; Enggaard, L.; Fillet, G.; Filosa, A.; Forni, G.; Galanello, R.; Gastl, G.; Giraudier, S.; Goldfarb, A.; Grigg, A.; Gumruk, F.; S. Y., Ha; Haase, D.; Heinrich, B.; Hertzberg, M.; Ho, J.; Hsu, H. C.; Huang, S.; Hunault Berger, M.; Inusa, B.; Jaulmes, D.; Jensen, J.; Kattamis, A.; Kilinc, Y.; Kim, K. H.; Kinsey, S.; Kjeldsen, L.; Koren, A.; Lai, M. E.; Lai, Y.; Lee, J. W.; Lee, K. H.; Lee, S. H.; Legros, L.; Li, C.; C. K., Li; Li, Q.; Lin, K. H.; Linkesch, W.; Lübbert, M.; Lutz, D.; Mohamed Thalha, A. J.; Mufti, G.; Muus, P.; Nobile, F.; Papadopoulos, N.; Perrotta, S.; Petrini, M.; Pfeilstöcker, M.; Piga, A.; Poole, J.; Porter, J. B.; Pungolino, E.; Quarta, G.; Ravoet, C.; Jolimont Lobbes, H. H.; Remacha, A. F.; Roy, L.; Saglio, G.; Sanz, G.; Schmugge, M.; Schots, H.; Secchi, G.; Seymour, J. F.; Shah, F.; Shah, H.; Shen, Z.; Slama, B.; Sutcharitchan, P.; Taher, A.; Tamary, H.; Tesch, H. J.; Thein, S. L.; Troncy, J.; Villegas, A.; Viprakasit, V.; Wainwright, L.; Wassmann, B.; Wettervald, M.; Will, A.; Wörmann, B.; Wright, J.; Yeh, S. P.; Yoon, S. S.; Zoumbos, N. S.; Zweegman, S.

doi:10.1016/j.leukres.2010.03.009

The prospective 1-year EPIC study enrolled 341 patients with myelodysplastic syndromes (MDS); although baseline iron burden was >2500ng/mL, approximately 50% were chelation-naïve. Overall median serum ferritin decreased significantly at 1 year (p=0.002). Decreases occurred irrespective of whether patients were chelation-naïve or previously chelated; changes were dependent on dose adjustments and ongoing iron intake. Sustained reductions in labile plasma iron were observed. Discontinuation rate (48.7%) and adverse event profile were consistent with previously reported deferasirox data in MDS. Alanine aminotransferase levels decreased significantly; change correlated significantly with reduction in serum ferritin (p<0.0001). This large dataset prospectively confirms the efficacy and well characterizes the safety profile of deferasirox in MDS.