@article{3089133, title = "Deferasirox in iron-overloaded patients with transfusion-dependent myelodysplastic syndromes: Results from the large 1-year EPIC study", author = "Gattermann, N. and Finelli, C. and Della Porta, M. and Fenaux, P. and Ganser, A. and Guerci-Bresler, A. and Schmid, M. and Taylor, K. and Vassilieff, D. and Habr, D. and Domokos, G. and Roubert, B. and Rose, C. and Agaoglu, L. and Alimena, G. and Alonso, D. and Ame, S. and Angelucci, E. and Arrizabalaga, B. and Athanasiou-Metaxa, M. and Augustson, B. and Aydinok, Y. and Baba, A. and Baccarani, M. and Beck, J. and Beris, P. and Beyne-Rauzy, O. and Birgens, H. and Bordessoule, D. and Borgna-Pignatti, C. and Bosly, A. and Bouabdallah, K. and Bowden, D. and Bowen, D. and Bron, D. and Cappellini, M.D. and Capra, M. and Cartron, G. and Cazzola, M. and Chalkias, C. and Chan, L.L. and Chancharunee, S. and Chapman, C. and Charoenkwan, P. and Chasapopoulou, E. and Cheze, S. and Chuansumrit, A. and Cianciulli, P. and Dauriac, C. and Delforge, M. and Dölken, G. and Dombret, H. and Duyster, J. and Economopoulos, T. and Ehninger, G. and Elalfy, M. and El-Beshlawy, A. and Enggaard, L. and Fillet, G. and Filosa, A. and Forni, G. and Galanello, R. and Gastl, G. and Giraudier, S. and Goldfarb, A. and Grigg, A. and Gumruk, F. and Ha, S.Y. and Haase, D. and Heinrich, B. and Hertzberg, M. and Ho, J. and Hsu, H.-C. and Huang, S. and Hunault-Berger, M. and Inusa, B. and Jaulmes, D. and Jensen, J. and Kattamis, A. and Kilinc, Y. and Kim, K.-H. and Kinsey, S. and Kjeldsen, L. and Koren, A. and Lai, M.E. and Lai, Y. and Lee, J.-W. and Lee, K.-H. and Lee, S.-H. and Legros, L. and Li, C. and Li, C.-K. and Li, Q. and Lin, K.-H. and Linkesch, W. and Lübbert, M. and Lutz, D. and Mohamed Thalha, A.J. and Mufti, G. and Muus, P. and Nobile, F. and Papadopoulos, N. and Perrotta, S. and Petrini, M. and Pfeilstöcker, M. and Piga, A. and Poole, J. and Porter, J.B. and Pungolino, E. and Quarta, G. and Ravoet, C. and Jolimont Lobbes, H.H. and Remacha, A.F. and Roy, L. and Saglio, G. and Sanz, G. and Schmugge, M. and Schots, H. and Secchi, G. and Seymour, J.F. and Shah, F. and Shah, H. and Shen, Z. and Slama, B. and Sutcharitchan, P. and Taher, A. and Tamary, H. and Tesch, H.-J. and Thein, S.L. and Troncy, J. and Villegas, A. and Viprakasit, V. and Wainwright, L. and Wassmann, B. and Wettervald, M. and Will, A. and Wörmann, B. and Wright, J. and Yeh, S.-P. and Yoon, S.-S. and Zoumbos, N.C. and Zweegman, S.", journal = "Leukemia Research", year = "2010", volume = "34", number = "9", pages = "1143-1150", publisher = "Elsevier Ireland Ltd", issn = "0145-2126", doi = "10.1016/j.leukres.2010.03.009", keywords = "alanine aminotransferase; deferasirox; ferritin; iron, abdominal pain; adolescent; adult; aged; anemia; article; chelation therapy; child; clinical trial; colitis; constipation; creatinine blood level; cytopenia; dementia; diarrhea; dose response; drug dose increase; drug dose reduction; drug efficacy; drug fatality; drug induced headache; drug safety; drug withdrawal; duodenitis; erythrocyte transfusion; esophagitis; female; ferritin blood level; fever; gastritis; gastrointestinal symptom; hearing impairment; heart failure; human; hyperthermia; iron blood level; iron intake; iron overload; leukocyte count; major clinical study; male; multicenter study; myelodysplastic syndrome; neutropenia; pancytopenia; preschool child; priority journal; prospective study; rash; school child; side effect; thrombocytopenia; upper abdominal pain; vomiting; weight reduction", 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." }