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Real-life use of erythropoiesis-stimulating agents in myelodysplastic syndromes: a “Gruppo Romano Mielodisplasie (GROM)” multicenter study

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Abstract

The Gruppo Romano Mielodisplasie (GROM) conducted a retrospective study in 543 patients with myelodysplastic syndromes (MDS) to evaluate the safety and efficacy of erythropoiesis-stimulating agents (ESAs) in “real-life” clinical practice. The 40.000-UI/week erythropoietin (EPO)-alpha and 30.000-UI/week EPO-beta starting dose were defined “standard,” and 80,000 UI/week EPO-alpha and 60.000 UI/week EPO-beta were defined “high.” Response was defined according to International Working Group (IWG) 2006 criteria. At ESA’s start, median age was 74.2 years (interquartile range (IR) 67.8–79.5) and median hemoglobin was 8.9 g/dl (IR 8.2–9.6). Median time from diagnosis to ESAs start was 3.8 months (IR 0.8–13.2). ESA starting dose was “standard” in 361 patients (66.5 %) and “high” in 182 patients (33.5 %). Erythroid response was observed in 82/185 (44.3 %) transfusion dependent (TD) patients as compared with 226/329 (68.6 %) transfusion independent (TI) ones (p < 0.001). At multivariate analysis, in TD patients, only endogenous EPO levels <50 mU/l were significant (p = 0.046), whereas in TI patients, high-dose ESAs (p < 0.001), abnormal creatinine levels (0.009), and endogenous EPO levels <50 mU/l (p = 0.014) were predictors of response. Responders showed a higher 5-year overall survival (OS) (57.8 vs. 32.2 %, p < 0.001) and leukemia-free survival (76.0 vs. 49.8 %, p < 0.001). At multivariable analysis for OS, response to ESA, low International Prognostic Scoring System (IPSS), no transfusion need, and female sex showed an independent favorable prognostic role. Our results confirm that treatment with ESAs is effective in a real-life MDS setting, particularly at high dose and in TI patients. Prospective studies are needed to define the optimal starting dose.

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Acknowledgments

This work was supported by a grant from Regione Lazio: “Sindromi mielodisplastiche dell’adulto nell’area di Roma e nel Lazio: epidemiologia, caratteristiche diagnostiche e clinico-terapeutiche, analisi dei costi mediante un registro onco-ematologico regionale (Grant 2011 – progetti di farmacovigilanza – Area tematica 5). The authors thank Dr. Esther Natalie Oliva for the critical review of the manuscript.

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Authors and Affiliations

  1. Hematology, Department of Biomedicine and Prevention, Tor Vergata University, Rome, Italy

    Francesco Buccisano, Maria Teresa Voso, Luca Maurillo & Adriano Venditti

  2. Ematologia, Dipartimento di Biomedicina e Prevenzione, Università di Roma Tor Vergata, Via Montpellier 1, 00133, Rome, Italy

    Francesco Buccisano

  3. Division of Hematology, Sandro Pertini Hospital, Rome, Italy

    Anna Lina Piccioni

  4. Hematology, University “Campus Biomedico”, Rome, Italy

    Carolina Nobile

  5. Institute of Hematology, Catholic University of Sacred Heart, Rome, Italy

    Marianna Criscuolo, Luana Fianchi & Gina Zini

  6. Hematology, Sant’Eugenio Hospital, Rome, Italy

    Pasquale Niscola & Benedetta Neri

  7. Division of Hematology, Rome, Italy

    Caterina Tatarelli & Maria Antonietta Aloe Spiriti

  8. Hematology, Sant’Andrea Hospital, Rome, Italy

    Caterina Tatarelli

  9. Hematology, Nuovo Regina Margherita Hospital, Rome, Italy

    Nicoletta Villivà

  10. Department of Cellular Biotechnologies and Hematology, University “La Sapienza”, Rome, Italy

    Ida Carmosino, Massimo Breccia & Roberto Latagliata

  11. UOC of Hematology, IFO-San Gallicano Hospital, Rome, Italy

    Svitlana Gumenyuk

  12. Division of Hematology and Bone Marrow Transplantation, Azienda Ospedaliera San Camillo-Forlanini, Rome, Italy

    Stefano Mancini

  13. Division of Hematology, San Giovanni Hospital, Rome, Italy

    Susanna Fenu

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  1. Francesco Buccisano
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  2. Anna Lina Piccioni
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  3. Carolina Nobile
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  14. Luca Maurillo
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  19. Maria Antonietta Aloe Spiriti
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  20. Roberto Latagliata
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on behalf of GROM (Gruppo Romano Mielodisplasie)

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Correspondence to Francesco Buccisano.

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Buccisano, F., Piccioni, A.L., Nobile, C. et al. Real-life use of erythropoiesis-stimulating agents in myelodysplastic syndromes: a “Gruppo Romano Mielodisplasie (GROM)” multicenter study. Ann Hematol 95, 1059–1065 (2016). https://doi.org/10.1007/s00277-016-2667-1

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