| Literature DB >> 28350519 |
Sophie Park1, Jean-François Hamel1, Andrea Toma1, Charikleia Kelaidi1, Sylvain Thépot1, Maria Diez Campelo1, Valeria Santini1, Mikkael A Sekeres1, Enrico Balleari1, Jennifer Kaivers1, Rosa Sapena1, Katharina Götze1, Catharina Müller-Thomas1, Odile Beyne-Rauzy1, Aspasia Stamatoullas1, Ioannis Kotsianidis1, Rami Komrokji1, David P Steensma1, Jaime Fensterl1, Gail J Roboz1, Teresa Bernal1, Fernando Ramos1, Marisa Calabuig1, Agnès Guerci-Bresler1, Dominique Bordessoule1, Pascale Cony-Makhoul1, Stéphane Cheze1, Eric Wattel1, Christian Rose1, Norbert Vey1, Daniela Gioia1, Dario Ferrero1, Gianluca Gaidano1, Giovanni Cametti1, Fabrizio Pane1, Alessandro Sanna1, Ulrich Germing1, Guillermo F Sanz1, François Dreyfus1, Pierre Fenaux1.
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.Entities:
Mesh：
Substances：
Year: 2017 PMID： 28350519 DOI： 10.1200/JCO.2016.71.3271
Source DB: PubMed Journal: J Clin Oncol ISSN： 0732-183X Impact factor: 44.544