Outcome of Lower-Risk Patients With Myelodysplastic Syndromes Without 5q Deletion After Failure of Erythropoiesis-Stimulating Agents.
Fiche publication
Date publication
mars 2017
Journal
Journal of clinical oncology : official journal of the American Society of Clinical Oncology
Auteurs
Membres identifiés du Cancéropôle Est :
Dr GUERCI-BRESLER Agnès
Tous les auteurs :
Park S, Hamel JF, Toma A, Kelaidi C, Thépot S, Campelo MD, Santini V, Sekeres MA, Balleari E, Kaivers J, Sapena R, Götze K, Müller-Thomas C, Beyne-Rauzy O, Stamatoullas A, Kotsianidis I, Komrokji R, Steensma DP, Fensterl J, Roboz GJ, Bernal T, Ramos F, Calabuig M, Guerci-Bresler A, Bordessoule D, Cony-Makhoul P, Cheze S, Wattel E, Rose C, Vey N, Gioia D, Ferrero D, Gaidano G, Cametti G, Pane F, Sanna A, Germing U, Sanz GF, Dreyfus F, Fenaux P
Lien Pubmed
Résumé
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.
Mots clés
Aged, Aged, 80 and over, Anemia, etiology, Antilymphocyte Serum, therapeutic use, Antineoplastic Agents, therapeutic use, Arsenic, therapeutic use, Azacitidine, analogs & derivatives, Chromosome Deletion, Chromosomes, Human, Pair 5, Cyclosporine, therapeutic use, Cytarabine, therapeutic use, Decitabine, Disease Progression, Enzyme Inhibitors, therapeutic use, Erythrocyte Transfusion, Female, Hematinics, therapeutic use, Humans, Hydroxyurea, therapeutic use, Immunologic Factors, therapeutic use, Lenalidomide, Leukemia, Myeloid, Acute, etiology, Male, Middle Aged, Myelodysplastic Syndromes, complications, Recurrence, Retreatment, Retrospective Studies, Risk Factors, Survival Rate, Thalidomide, analogs & derivatives, Treatment Failure, Tretinoin, therapeutic use, Valproic Acid, therapeutic use
Référence
J. Clin. Oncol.. 2017 Mar;:JCO2016713271