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

• Main Authors: Gattermann,N., Finelli,C., Della Porta,M., Fénaux,P., Ganser,A.G., Guerci-Bresler,A., Schmid,M.L., Taylor,K.M., Vassilieff,D., Habr,D., Domokos,G., Roubert,B., Rose,C.S., Aǧaoǧlu,L., Alimena,G., Alonso,D., Amé,S., Angelucci,E., Arrizabalaga,B., Athanasiou-Metaxa,M., Augustson,B.M., Aydınok,Y., Baba,A.A., Baccarani,M., Beck,J.T., Béris,P.H., Beyne-Rauzy,O., Birgens,H.S., B́ordessoule,D., Borgna-Pignatti,C., Bosly,A.E., Bouabdallah,K., Bowden,D.K., Bowen,D.T., Bron,D.G., Cappellini,M.D.O., Capra,M.E.Z., Cartron,G., Cazzola,M., Chalkias,C., Chan,L., Chancharunee,S., Chapman,C.S., Charoenkwan,P., Chasapopoulou,E., Chèze,S., Chuansumrit,A., Cianciulli,P., Dauriac,C., Delforge,M., Dölken,G., Dombret,H., Duyster,J., Economopoulos,T.H., Ehninger,G.E., Elalfy,M.S., El Beshlawy,A., Enggaard,L., Fillet,G., Filosa,A., Forni,G.L., Galanello,R., Gastl,G.A., Giraudier,S., Goldfarb,A.W., Grigg,A.P., Gümrük,F.U., Ha,S.Y., Haase,D., Heinrich,B., Hertzberg,M.S., Ho,J., Hsu,H., Huang,S., Hunault-Berger,M., Inusa,B.P.D., Jaulmes,D., Jensen,J.M., Kattamis,A.C., Kilinç,Y., Kim,K., Kinsey,S.E., Kjeldsen,L., Koren,A., Lai,M., Lai,Y., Lee,J., Lee,K., Lee,S., Legros,L., Li,C., Li,Q., Lin,K., Linkesch,W.M., Lübbert,M., Lutz,D., Mohamed Thalha,A.J., Mufti,G.J., Muus,P.K., Nobile,F., Papadopoulos,N.T., Perrotta,S., Petrini,M.T., Pfeilstöcker,M., Piga,A.M., Poole,J.E., Porter,J.B., Pungolino,E.M., Quarta,G., Ravoet,C., Jolimont Lobbes,H.H., Remacha,A.F., Roy,L., Saglio,G.G., Sanz,G.F., Schmugge,M., Schots,H., Secchi,G., Seymour,J.F., Shah,F.T., Shah,H.N., Shen,Z., Slama,B., Sutcharitchan,P., Taher,A.T., Tamary,H., Tesch,H.J., Thein,S.L., Troncy,J., Villegas,A.M., Viprakasit,V., Wainwright,L., Waßmann,B., Wettervald,M., Will,A.M., Wörmann,B.J., Wright,J.G., Yeh,S., Yoon,S., Zoumbos,N.C., Zweegman,S.
• Format: Article
• Published: Elsevier Limited 2015
• Subjects: Cancer Research; Hematology; Oncology
• Online Access: http://www.scopus.com/inward/record.url?partnerID=HzOxMe3b&scp=77955268192&origin=inward; http://cmuir.cmu.ac.th/handle/6653943832/38178
• Institution: Chiang Mai University

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.