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

Gina Zini, Marianna Criscuolo, Luana Fianchi, Maria Teresa Voso, Francesco Buccisano, Anna Lina Piccioni, Carolina Nobile, Pasquale Niscola, Caterina Tatarelli, Nicoletta Villivà, Benedetta Neri, Ida Carmosino, Svitlana Gumenyuk, Stefano Mancini, Luca Maurillo, Massimo Breccia, Adriano Venditti, Susanna Fenu, Maria Antonietta Aloe Spiriti, Roberto Latagliata

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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.
Original languageEnglish
Pages (from-to)1059-1065
Number of pages7
JournalAnnals of Hematology
Publication statusPublished - 2016


  • Aged
  • Aged, 80 and over
  • ESAs
  • Female
  • Follow-Up Studies
  • Hematinics
  • Hematology
  • Humans
  • Italy
  • Leukemia progression-free survival
  • MDS
  • Male
  • Myelodysplastic Syndromes
  • Overall survival
  • Real-life study
  • Retrospective Studies
  • Survival Rate


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