Hernández-Boluda Juan-Carlos, Correa Juan-Gonzalo, García-Delgado Regina, Martínez-López Joaquín, Alvarez-Larrán Alberto, Fox María-Laura, García-Gutiérrez Valentín, Pérez-Encinas Manuel, Ferrer-Marín Francisca, Mata-Vázquez María-Isabel, Raya José-María, Estrada Natalia, García Silvia, Kerguelen Ana, Durán María-Antonia, Albors Manuel, Cervantes Francisco
Hematology Department, Hospital Clínico Universitario, INCLIVA, Valencia, Spain.
Hematology Department, Hospital Clínic, IDIBAPS, University of Barcelona, Barcelona, Spain.
Eur J Haematol. 2017 Apr;98(4):407-414. doi: 10.1111/ejh.12846. Epub 2017 Jan 19.
Erythropoiesis-stimulating agents (ESAs) are commonly used to treat the anemia of myelofibrosis (MF), but information on the predictors of response is limited.
Results of ESA therapy were analyzed in 163 MF patients with severe anemia, most of whom had inadequate erythropoietin (EPO) levels (<125 U/L) at treatment start.
According to the revised criteria of the International Working Group for Myelofibrosis Treatment and Research, anemia response was achieved in 86 patients (53%). Median response duration was 19.3 months. In multivariate analysis, baseline factors associated with a higher response rate were female sex (P=.007), leukocyte count ≥10×10 /L (P=.033), and serum ferritin <200 ng/mL (P=.002). Patients with 2 or 3 of the above features had a significantly higher response rate than the remainder (73% vs 28%, respectively; P<.001). Over the 373 patient-years of follow-up on ESA treatment, nine patients developed thrombotic complications (six arterial, three venous), accounting for 2.41 events per 100 patient-years. Survival time from ESA start was longer in anemia responders than in non-responders (P=.011).
Besides the already established predictive value of EPO levels, these data can help to identify which MF patients are more likely to benefit from ESA treatment.
促红细胞生成素（ESA）常用于治疗骨髓纤维化（MF）所致贫血，但关于反应预测因素的信息有限。
分析了163例重度贫血MF患者的ESA治疗结果，其中大多数患者在治疗开始时促红细胞生成素（EPO）水平不足（<125 U/L）。
根据国际骨髓纤维化治疗和研究工作组的修订标准，86例患者（53%）实现了贫血反应。中位反应持续时间为19.3个月。多变量分析显示，与较高反应率相关的基线因素为女性（P = 0.007）、白细胞计数≥10×10⁹/L（P = 0.033）和血清铁蛋白<200 ng/mL（P = 0.002）。具有上述2项或3项特征的患者反应率显著高于其余患者（分别为73%和28%；P<0.001）。在ESA治疗的373患者年随访中，9例患者发生血栓并发症（6例动脉性，3例静脉性），每100患者年发生率为2.41次。贫血反应者从开始使用ESA起的生存时间长于无反应者（P = 0.011）。
除了已确定的EPO水平的预测价值外，这些数据有助于确定哪些MF患者更可能从ESA治疗中获益。
