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

被引：7

作者：

Buccisano, Francesco ^{[1
,2
]}

Piccioni, Anna Lina ^{[3
]}

Nobile, Carolina ^{[4
]}

Criscuolo, Marianna ^{[5
]}

Niscola, Pasquale ^{[6
]}

Tatarelli, Caterina ^{[7
,8
]}

Fianchi, Luana ^{[5
]}

Villiva, Nicoletta ^{[9
]}

Neri, Benedetta ^{[6
]}

Carmosino, Ida ^{[10
]}

Gumenyuk, Svitlana ^{[11
]}

Mancini, Stefano ^{[12
]}

Voso, Maria Teresa ^{[1
]}

Maurillo, Luca ^{[1
]}

Breccia, Massimo ^{[10
]}

Zini, Gina ^{[5
]}

Venditti, Adriano ^{[1
]}

Fenu, Susanna ^{[13
]}

Spiriti, Maria Antonietta Aloe ^{[7
]}

Latagliata, Roberto ^{[10
]}

机构：

[1] Univ Roma Tor Vergata, Dept Biomed & Prevent, Hematol, Rome, Italy

[2] Univ Roma Tor Vergata, Ematol, Dipartimento Biomed & Prevenz, Via Montpellier 1, I-00133 Rome, Italy

[3] Sandro Pertini Hosp, Div Hematol, Rome, Italy

[4] Univ Campus Biomed, Hematol, Rome, Italy

[5] Univ Cattolica Sacro Cuore, Inst Hematol, I-00168 Rome, Italy

[6] St Eugenio Hosp, Hematol, Rome, Italy

[7] Div Hematol, Rome, Italy

[8] St Andrea Hosp, Hematol, Rome, Italy

[9] Nuovo Regina Margherita Hosp, Hematol, Rome, Italy

[10] Univ Roma La Sapienza, Dept Cellular Biotechnol & Hematol, Piazzale Aldo Moro 5, I-00185 Rome, Italy

[11] IFO San Gallicano Hosp, UOC Hematol, Rome, Italy

[12] Azienda Osped San Camillo Forlanini, Div Hematol & Bone Marrow Transplantat, Rome, Italy

[13] San Giovanni Hosp, Div Hematol, Rome, Italy

来源：

ANNALS OF HEMATOLOGY | 2016年 / 95卷 / 07期

关键词：

MDS; ESAs; Real-life study; Overall survival; Leukemia progression-free survival; QUALITY-OF-LIFE; RISK; ANEMIA; EPIDEMIOLOGY; SURVIVAL; EFFICACY; CRITERIA;

D O I：

10.1007/s00277-016-2667-1

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

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.

引用

页码：1059 / 1065

页数：7

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