Real-life use of erythropoiesis-stimulating agents in myelodysplastic syndromes: a "Gruppo Romano Mielodisplasie (GROM)" multicenter study.
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.
Hematology, Department of Biomedicine and Prevention, Tor Vergata University, Rome, Italy. francesco.buccisano@uniroma2.it.|Ematologia, Dipartimento di Biomedicina e Prevenzione, Università di Roma Tor Vergata, Via Montpellier 1, 00133, Rome, Italy. francesco.buccisano@uniroma2.it.
Francesco
F
Buccisano
Division of Hematology, Sandro Pertini Hospital, Rome, Italy.
Anna Lina
AL
Piccioni
Hematology, University "Campus Biomedico", Rome, Italy.
Carolina
C
Nobile
Institute of Hematology, Catholic University of Sacred Heart, Rome, Italy.
Marianna
M
Criscuolo
Hematology, Sant'Eugenio Hospital, Rome, Italy.
Pasquale
P
Niscola
Division of Hematology, Rome, Italy.|Hematology, Sant'Andrea Hospital, Rome, Italy.
Caterina
C
Tatarelli
Institute of Hematology, Catholic University of Sacred Heart, Rome, Italy.
Luana
L
Fianchi
Hematology, Nuovo Regina Margherita Hospital, Rome, Italy.
Nicoletta
N
Villivà
Hematology, Sant'Eugenio Hospital, Rome, Italy.
Benedetta
B
Neri
Department of Cellular Biotechnologies and Hematology, University "La Sapienza", Rome, Italy.
Ida
I
Carmosino
UOC of Hematology, IFO-San Gallicano Hospital, Rome, Italy.
Svitlana
S
Gumenyuk
Division of Hematology and Bone Marrow Transplantation, Azienda Ospedaliera San Camillo-Forlanini, Rome, Italy.
Stefano
S
Mancini
Hematology, Department of Biomedicine and Prevention, Tor Vergata University, Rome, Italy.
Maria Teresa
MT
Voso
Hematology, Department of Biomedicine and Prevention, Tor Vergata University, Rome, Italy.
Luca
L
Maurillo
Department of Cellular Biotechnologies and Hematology, University "La Sapienza", Rome, Italy.
Massimo
M
Breccia
Institute of Hematology, Catholic University of Sacred Heart, Rome, Italy.
Gina
G
Zini
Hematology, Department of Biomedicine and Prevention, Tor Vergata University, Rome, Italy.
Adriano
A
Venditti
Division of Hematology, San Giovanni Hospital, Rome, Italy.
Susanna
S
Fenu
Division of Hematology, Rome, Italy.
Maria Antonietta Aloe
MA
Spiriti
Department of Cellular Biotechnologies and Hematology, University "La Sapienza", Rome, Italy.
Roberto
R
Latagliata
D000368:Aged; D000369:Aged, 80 and over; D005260:Female; D005500:Follow-Up Studies; D006397:Hematinics / Q000627:therapeutic use*; D006801:Humans; D007558:Italy / Q000453:epidemiology; D008297:Male; D009190:Myelodysplastic Syndromes / Q000175:diagnosis* / Q000188:drug therapy* / Q000401:mortality; D012189:Retrospective Studies; D015996:Survival Rate / Q000639:trends
D006397:Hematinics
ESAs; Leukemia progression-free survival; MDS; Overall survival; Real-life study