Outcome of Lower-Risk Patients With Myelodysplastic Syndromes Without 5q Deletion After Failure of Erythropoiesis-Stimulating Agents
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Published:2017-05-10
Issue:14
Volume:35
Page:1591-1597
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ISSN:0732-183X
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Container-title:Journal of Clinical Oncology
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language:en
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Short-container-title:JCO
Author:
Park Sophie1, Hamel Jean-François1, Toma Andrea1, Kelaidi Charikleia1, Thépot Sylvain1, Campelo Maria Diez1, Santini Valeria1, Sekeres Mikkael A.1, Balleari Enrico1, Kaivers Jennifer1, Sapena Rosa1, Götze Katharina1, Müller-Thomas Catharina1, Beyne-Rauzy Odile1, Stamatoullas Aspasia1, Kotsianidis Ioannis1, Komrokji Rami1, Steensma David P.1, Fensterl Jaime1, Roboz Gail J.1, Bernal Teresa1, Ramos Fernando1, Calabuig Marisa1, Guerci-Bresler Agnès1, Bordessoule Dominique1, Cony-Makhoul Pascale1, Cheze Stéphane1, Wattel Eric1, Rose Christian1, Vey Norbert1, Gioia Daniela1, Ferrero Dario1, Gaidano Gianluca1, Cametti Giovanni1, Pane Fabrizio1, Sanna Alessandro1, Germing Ulrich1, Sanz Guillermo F.1, Dreyfus François1, Fenaux Pierre1
Affiliation:
1. Sophie Park, Centre Hospitalier Universitaire (CHU) Grenoble Alpes, Université Grenoble Alpes, Grenoble; Jean-François Hamel and Sylvain Thépot, CHU d’Angers; Sylvain Thépot, Université d’Angers, Institut National de la Santé et de la Recherche Médicale, Unité 1232, and LabEx IGO, Angers; Andrea Toma, CHU Henri Mondor, Assistance Publique Hôpitaux de Paris (AP-HP), Université Paris-Est Créteil; Rosa Sapena and Pierre Fenaux, CHU Saint Louis, AP-HP, Université Paris X; François Dreyfus, CHU Cochin, AP-HP,...
Abstract
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%. Second-line 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.
Publisher
American Society of Clinical Oncology (ASCO)
Subject
Cancer Research,Oncology
Cited by
74 articles.
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