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

Gattermann, Norbert; Finelli, Carlo; Della Porta, Matteo; Fenaux, Pierre; Ganser, Arnold; Guerci-Bresler, Agnes; Schmid, Mathias; Taylor, Kerry; Vassilieff, Dominique; Habr, Dany; Domokos, Gabor; Roubert, Bernard; Rose, Christian; Agaoglu, L.; Alimena, G.; Alonso, D.; Ame, S.; Angelucci, E.; 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-Pignatti, C.; 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.; GÜMRÜK, FATMA; Ha, S.Y.; 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.; Li, C.-K.; 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.C.; Zweegman, S.

doi:10.1016/j.leukres.2010.03.009

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

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Gattermann N., Finelli C., Della Porta M., Fenaux P., Ganser A., Guerci-Bresler A., ...More

Leukemia Research, vol.34, no.9, pp.1143-1150, 2010 (SCI-Expanded)

Publication Type: Article / Article
Volume: 34 Issue: 9
Publication Date: 2010
Doi Number: 10.1016/j.leukres.2010.03.009
Journal Name: Leukemia Research
Journal Indexes: Science Citation Index Expanded (SCI-EXPANDED), Scopus
Page Numbers: pp.1143-1150
Keywords: Deferasirox, Iron chelation therapy, Labile plasma iron, Myelodysplastic syndromes, Serum ferritin
Hacettepe University Affiliated: Yes

Abstract

The prospective 1-year EPIC study enrolled 341 patients with myelodysplastic syndromes (MDS); although baseline iron burden was >2500. ng/mL, ∼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. © 2010 Elsevier Ltd.