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

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

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

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

^*Corresponding author for this work

Research output: Contribution to journal › Article › peer-review

163 Scopus citations

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.

Original language	English
Pages (from-to)	1143-1150
Number of pages	8
Journal	Leukemia Research
Volume	34
Issue number	9
DOIs	https://doi.org/10.1016/j.leukres.2010.03.009
State	Published - Sep 2010
Externally published	Yes

Funding

Funders	Funder number
Novartis Pharma

Keywords

Deferasirox
Iron chelation therapy
Labile plasma iron
Myelodysplastic syndromes
Serum ferritin

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10.1016/j.leukres.2010.03.009

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Gattermann, N., Finelli, C., Della Porta, M., 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., Arrizabalaga, B., ... Zweegman, S. (2010). Deferasirox in iron-overloaded patients with transfusion-dependent myelodysplastic syndromes: Results from the large 1-year EPIC study. Leukemia Research, 34(9), 1143-1150. https://doi.org/10.1016/j.leukres.2010.03.009

@article{a6997680625443b0ba54c057260bbc15,

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

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-na{\"i}ve. Overall median serum ferritin decreased significantly at 1 year (p=0.002). Decreases occurred irrespective of whether patients were chelation-na{\"i}ve 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.",

keywords = "Deferasirox, Iron chelation therapy, Labile plasma iron, Myelodysplastic syndromes, Serum ferritin",

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

note = "Funding Information: Funding : The study was sponsored by Novartis Pharma AG (Basel, Switzerland) and designed by the sponsor in consultation with a board of expert hematologists. The sponsor and research organization working under contract for the sponsor monitored the investigational sites, collected and managed the data, and conducted the statistical analysis. An independent Study Monitoring Committee (SMC) oversaw the execution of the study and the lead investigators and SMC had access to the final study data and provided significant contributions to the data interpretation. Dr Gattermann prepared the manuscript with the assistance of a medical writer. Financial support for medical editorial assistance was provided by Novartis. All other named authors contributed to the interpretation of the results, critically reviewed and commented on the manuscript, and approved the final version before submission. Dr Gattermann made all final decisions about all aspects of publication. We thank Andrew Jones, PhD for medical editorial assistance with this manuscript. ",

year = "2010",

month = sep,

doi = "10.1016/j.leukres.2010.03.009",

language = "אנגלית",

volume = "34",

pages = "1143--1150",

journal = "Leukemia Research",

issn = "0145-2126",

publisher = "Elsevier Ltd.",

number = "9",

}

Gattermann, N, Finelli, C, Della Porta, M, 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, 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, MD, Capra, M, Cartron, G, Cazzola, M, Chalkias, C, Chan, LL, 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, Ha, SY, Haase, D, Heinrich, B, Hertzberg, M, Ho, J, Hsu, HC, Huang, S, Hunault-Berger, M, Inusa, B, Jaulmes, D, Jensen, J, Kattamis, A, Kilinc, Y, Kim, KH, Kinsey, S, Kjeldsen, L, Koren, A, Lai, ME, Lai, Y, Lee, JW, Lee, KH, Lee, SH, Legros, L, Li, C, Li, CK, Li, Q, Lin, KH, Linkesch, W, Lübbert, M, Lutz, D, Mohamed Thalha, AJ, Mufti, G, Muus, P, Nobile, F, Papadopoulos, N, Perrotta, S, Petrini, M, Pfeilstöcker, M, Piga, A, Poole, J, Porter, JB, Pungolino, E, Quarta, G, Ravoet, C, Jolimont Lobbes, HH, Remacha, AF, Roy, L, Saglio, G, Sanz, G, Schmugge, M, Schots, H, Secchi, G, Seymour, JF, Shah, F, Shah, H, Shen, Z, Slama, B, Sutcharitchan, P, Taher, A, Tamary, H, Tesch, HJ, Thein, SL, Troncy, J, Villegas, A, Viprakasit, V, Wainwright, L, Wassmann, B, Wettervald, M, Will, A, Wörmann, B, Wright, J, Yeh, SP, Yoon, SS, Zoumbos, NC & Zweegman, S 2010, 'Deferasirox in iron-overloaded patients with transfusion-dependent myelodysplastic syndromes: Results from the large 1-year EPIC study', Leukemia Research, vol. 34, no. 9, pp. 1143-1150. https://doi.org/10.1016/j.leukres.2010.03.009

TY - JOUR

T1 - Deferasirox in iron-overloaded patients with transfusion-dependent myelodysplastic syndromes

T2 - Results from the large 1-year EPIC study

AU - Gattermann, Norbert

AU - Finelli, Carlo

AU - Della Porta, Matteo

AU - Fenaux, Pierre

AU - Ganser, Arnold

AU - Guerci-Bresler, Agnes

AU - Schmid, Mathias

AU - Taylor, Kerry

AU - Vassilieff, Dominique

AU - Habr, Dany

AU - Domokos, Gabor

AU - Roubert, Bernard

AU - Rose, Christian

AU - Agaoglu, L.

AU - Alimena, G.

AU - Alonso, D.

AU - Ame, S.

AU - Angelucci, E.

AU - Arrizabalaga, B.

AU - Athanasiou-Metaxa, M.

AU - Augustson, B.

AU - Aydinok, Y.

AU - Baba, A.

AU - Baccarani, M.

AU - Beck, J.

AU - Beris, P.

AU - Beyne-Rauzy, O.

AU - Birgens, H.

AU - Bordessoule, D.

AU - Borgna-Pignatti, C.

AU - Bosly, A.

AU - Bouabdallah, K.

AU - Bowden, D.

AU - Bowen, D.

AU - Bron, D.

AU - Cappellini, M. D.

AU - Capra, M.

AU - Cartron, G.

AU - Cazzola, M.

AU - Chalkias, C.

AU - Chan, L. L.

AU - Chancharunee, S.

AU - Chapman, C.

AU - Charoenkwan, P.

AU - Chasapopoulou, E.

AU - Cheze, S.

AU - Chuansumrit, A.

AU - Cianciulli, P.

AU - Dauriac, C.

AU - Delforge, M.

AU - Dölken, G.

AU - Dombret, H.

AU - Duyster, J.

AU - Economopoulos, T.

AU - Ehninger, G.

AU - Elalfy, M.

AU - El-Beshlawy, A.

AU - Enggaard, L.

AU - Fillet, G.

AU - Filosa, A.

AU - Forni, G.

AU - Galanello, R.

AU - Gastl, G.

AU - Giraudier, S.

AU - Goldfarb, A.

AU - Grigg, A.

AU - Gumruk, F.

AU - Ha, S. Y.

AU - Haase, D.

AU - Heinrich, B.

AU - Hertzberg, M.

AU - Ho, J.

AU - Hsu, H. C.

AU - Huang, S.

AU - Hunault-Berger, M.

AU - Inusa, B.

AU - Jaulmes, D.

AU - Jensen, J.

AU - Kattamis, A.

AU - Kilinc, Y.

AU - Kim, K. H.

AU - Kinsey, S.

AU - Kjeldsen, L.

AU - Koren, A.

AU - Lai, M. E.

AU - Lai, Y.

AU - Lee, J. W.

AU - Lee, K. H.

AU - Lee, S. H.

AU - Legros, L.

AU - Li, C.

AU - Li, C. K.

AU - Li, Q.

AU - Lin, K. H.

AU - Linkesch, W.

AU - Lübbert, M.

AU - Lutz, D.

AU - Mohamed Thalha, A. J.

AU - Mufti, G.

AU - Muus, P.

AU - Nobile, F.

AU - Papadopoulos, N.

AU - Perrotta, S.

AU - Petrini, M.

AU - Pfeilstöcker, M.

AU - Piga, A.

AU - Poole, J.

AU - Porter, J. B.

AU - Pungolino, E.

AU - Quarta, G.

AU - Ravoet, C.

AU - Jolimont Lobbes, H. H.

AU - Remacha, A. F.

AU - Roy, L.

AU - Saglio, G.

AU - Sanz, G.

AU - Schmugge, M.

AU - Schots, H.

AU - Secchi, G.

AU - Seymour, J. F.

AU - Shah, F.

AU - Shah, H.

AU - Shen, Z.

AU - Slama, B.

AU - Sutcharitchan, P.

AU - Taher, A.

AU - Tamary, H.

AU - Tesch, H. J.

AU - Thein, S. L.

AU - Troncy, J.

AU - Villegas, A.

AU - Viprakasit, V.

AU - Wainwright, L.

AU - Wassmann, B.

AU - Wettervald, M.

AU - Will, A.

AU - Wörmann, B.

AU - Wright, J.

AU - Yeh, S. P.

AU - Yoon, S. S.

AU - Zoumbos, N. C.

AU - Zweegman, S.

N1 - Funding Information: Funding : The study was sponsored by Novartis Pharma AG (Basel, Switzerland) and designed by the sponsor in consultation with a board of expert hematologists. The sponsor and research organization working under contract for the sponsor monitored the investigational sites, collected and managed the data, and conducted the statistical analysis. An independent Study Monitoring Committee (SMC) oversaw the execution of the study and the lead investigators and SMC had access to the final study data and provided significant contributions to the data interpretation. Dr Gattermann prepared the manuscript with the assistance of a medical writer. Financial support for medical editorial assistance was provided by Novartis. All other named authors contributed to the interpretation of the results, critically reviewed and commented on the manuscript, and approved the final version before submission. Dr Gattermann made all final decisions about all aspects of publication. We thank Andrew Jones, PhD for medical editorial assistance with this manuscript.

PY - 2010/9

Y1 - 2010/9

N2 - 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.

AB - 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.

KW - Deferasirox

KW - Iron chelation therapy

KW - Labile plasma iron

KW - Myelodysplastic syndromes

KW - Serum ferritin

UR - http://www.scopus.com/inward/record.url?scp=77955268192&partnerID=8YFLogxK

U2 - 10.1016/j.leukres.2010.03.009

DO - 10.1016/j.leukres.2010.03.009

M3 - ???researchoutput.researchoutputtypes.contributiontojournal.article???

C2 - 20451251

AN - SCOPUS:77955268192

SN - 0145-2126

VL - 34

SP - 1143

EP - 1150

JO - Leukemia Research

JF - Leukemia Research

IS - 9

ER -

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

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