Outcome of Lower-Risk Patients With Myelodysplastic Syndromes Without 5q Deletion After Failure of Erythropoiesis-Stimulating Agents

被引：75

作者：

Park, Sophie ^{[1
]}

Hamel, Jean-Francois ^{[2
]}

Toma, Andrea ^{[5
]}

Kelaidi, Charikleia ^{[8
]}

Thepot, Sylvain ^{[2
,3
,4
]}

Campelo, Maria Diez ^{[18
]}

Santini, Valeria ^{[23
,24
]}

Sekeres, Mikkael A. ^{[31
]}

Balleari, Enrico ^{[25
]}

Kaivers, Jennifer ^{[35
]}

Sapena, Rosa ^{[6
]}

Goetze, Katharina ^{[36
]}

Mueller-Thomas, Catharina ^{[36
]}

Beyne-Rauzy, Odile ^{[9
]}

Stamatoullas, Aspasia ^{[10
]}

Kotsianidis, Ioannis ^{[37
]}

Komrokji, Rami ^{[32
]}

Steensma, David P. ^{[33
]}

Fensterl, Jaime ^{[31
]}

Roboz, Gail J. ^{[34
]}

Bernal, Teresa ^{[19
]}

Ramos, Fernando ^{[20
]}

Calabuig, Marisa ^{[21
]}

Guerci-Bresler, Agnes ^{[11
]}

Bordessoule, Dominique ^{[12
]}

Cony-Makhoul, Pascale ^{[13
]}

Cheze, Stephane ^{[14
]}

Wattel, Eric ^{[15
]}

Rose, Christian ^{[16
]}

Vey, Norbert ^{[17
]}

Gioia, Daniela ^{[26
]}

Ferrero, Dario ^{[27
]}

Gaidano, Gianluca ^{[28
]}

Cametti, Giovanni ^{[29
]}

Pane, Fabrizio ^{[30
]}

Sanna, Alessandro ^{[23
,24
]}

Germing, Ulrich ^{[35
]}

Sanz, Guillermo F. ^{[22
]}

Dreyfus, Francois ^{[7
]}

Fenaux, Pierre ^{[6
]}

机构：

[1] Univ Grenoble Alpes, CHU Grenoble Alpes, Grenoble, France

[2] CHU Angers, Angers, France

[3] Univ Angers, Inst Natl Sante & Rech Med, Unite 1232, Angers, France

[4] LabEx IGO, Angers, France

[5] Univ Paris Est Creteil, CHU Henri Mondor, AP HP, Creteil, France

[6] Univ Paris 10, CHU St Louis, AP HP, Nanterre, France

[7] Univ Paris 05, CHU Cochin, AP HP, Paris, France

[8] CHU Avicenne, Avicenne, France

[9] Inst Univ Canc Toulouse Oncopole, Toulouse, France

[10] Ctr Henri Becquerel, Rouen, France

[11] CHU Nancy, Nancy, France

[12] CHU Dupuytren, Limoges, France

[13] Ctr Hosp Annecy Genevois, Epagny Metz Tessy, France

[14] CHU Caen, Caen, France

[15] Ctr Hosp Lyon Sud, Lyon, France

[16] Univ Catholique Lille, Lille, France

[17] Inst Paoli Calmettes Marseille, Marseille, France

[18] Hosp Univ Salamanca, Salamanca, Spain

[19] Hosp Cent Asturias, Oviedo, Spain

[20] Hosp Leon, Leon, Spain

[21] Hosp Clin Valencia, Valencia, Spain

[22] Hosp Univ & Politecn Le Fe, Valencia, Spain

[23] Azienda Osped Univ, Florence, Italy

[24] Univ Florence, Florence, Italy

[25] Ist Ricovero Cura Carattere Sci AOU San Martino G, Genoa, Italy

[26] Azienda Osped SS Antonio Biagio Cesare Arrigo, Alessandria, Italy

[27] Univ Studi Torino, Turin, Italy

[28] Amedeo Avogadro Univ Eastern Piedmont, Turin, Italy

[29] Osped Maggiore Chieri, Chieri, Italy

[30] Univ Federico 2, Naples, Italy

[31] Cleveland Clin, Taussig Canc Inst, Cleveland, OH 44106 USA

[32] H Lee Moffitt Canc Ctr & Res Inst, Tampa, FL USA

[33] Dana Farber Canc Inst, Boston, MA 02115 USA

[34] Cornell Univ, Weill Med Coll, New York, NY 10021 USA

[35] Univ Dusseldorf, Dusseldorf, Germany

[36] Univ Munich, Munich, Germany

[37] Democritus Univ Thrace, Univ Hosp Alexandroupolis, Alexandroupolis, Greece

来源：

JOURNAL OF CLINICAL ONCOLOGY | 2017年 / 35卷 / 14期

关键词：

PHASE-II; SURVIVAL; SELECTION; MDS; GFM;

D O I：

10.1200/JCO.2016.71.3271

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 ;

摘要：

Purpose Most anemic patients with non-deleted 5q lower-risk myelodysplastic syndromes (MDS) are treated with erythropoiesis-stimulating agents (ESAs), with a response rate of approximately 50%. Secondline treatments, including hypomethylating agents (HMAs), lenalidomide (LEN), and investigational drugs, may be used after ESA failure in some countries, but their effect on disease progression and overall survival (OS) is unknown. Here, we analyzed outcome after ESA failure and the effect of second-line treatments. Patients and Methods We examined an international retrospective cohort of 1,698 patients with non-del(5q) lower-risk MDS treated with ESAs. Results Erythroid response to ESAs was 61.5%, and median response duration was 17 months. Of 1,147 patients experiencing ESA failure, 653 experienced primary failure and 494 experienced relapse after a response. Primary failure of ESAs was associated with a higher risk of acute myeloid leukemia (AML) progression, which did not translate into an OS difference. Of 450 patients (39%) who received second-line treatment, 194 received HMAs, 148 received LEN, and 108 received other treatments (MISC), whereas 697 received RBC transfusions only. Five-year AML cumulative incidence was 20.3%, 20.3%, and 11.3% for those receiving HMAs, LEN, and MISC, respectively (P = .05). Five-year OS for patients receiving HMA, LEN, and MISC was 36.5%, 41.7%, and 51%, respectively (P = .21). In a multivariable analysis adjusted for age, sex, revised International Prognostic Scoring System score, and progression at ESA failure, there was no significant OS difference among the three groups. Conclusion In this large, multicenter, retrospective cohort of patients with non-del(5q) lower-risk MDS treated with ESAs, none of the most commonly used second-line treatments (HMA and LEN) significantly improved OS. Early failure of ESAs was associated with a higher risk of AML progression. (C) 2017 by American Society of Clinical Oncology

引用

页码：1591 / +

页数：12

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