Remacha Ángel F, Arrizabalaga Beatriz, Villegas Ana, Durán María Soledad, Hermosín Lourdes, de Paz Raquel, Garcia Marta, Diez Campelo Maria, Sanz Guillermo
Hematology Department, Complejo Hospitalario de Toledo, Avenida Barber, 30, 45004, Toledo, Spain,
Ann Hematol. 2015 May;94(5):779-87. doi: 10.1007/s00277-014-2274-y. Epub 2014 Dec 18.
This study aimed to evaluate the evolution of iron overload, assessed by serum ferritin (SF), in transfusion-dependent lower risk patients with myelodysplastic syndrome (MDS), as well as to describe the occurrence of organ complications, and to analyze its relationship with iron chelation therapy. This observational retrospective study was conducted from March 2010 to March 2011 in 47 Spanish hospitals. A total of 263 patients with lower risk MDS (International Prognostic Scoring System [IPSS] low/intermediate-1 risk or Spanish Prognostic Index [SPI] 0-1 risk), transfusion-dependent, and who had received ≥10 packed red blood cells (PRBC) were included. At MDS diagnosis, patients received a mean of 2.8 ± 3.9 PRBC/month, and 8.7% of patients showed SF ≥1000 μg/L. Over the course of the disease, patients received a mean of 83.4 ± 83.3 PRBC, and 36.1% of patients presented SF ≥2500 μg/L. Cardiac, hepatic, endocrine, or arthropathy complications appeared/worsened in 20.2, 11.4, 9.9, and 3.8% of patients, respectively. According to investigator, iron overload was a main cause of hepatic (70.0%) and endocrine (26.9%) complications. A total of 96 (36.5%) patients received iron chelation therapy for ≥6 months, being deferasirox the most frequent first chelation treatment (71.9%). Chelation-treated patients showed longer overall survival (p < 0.001), leukemia-free survival (p = 0.007), and cardiac event-free survival (p = 0.017) than non-chelated patients. In multivariable analyses, age (p = 0.011), IPSS (p < 0.001), and chelation treatment (p = 0.015) were predictors for overall survival; IPSS (p = 0.014) and transfusion frequency (p = 0.001) for leukemia-free survival; and chelation treatment (p = 0.040) and Sorror comorbidity index (p = 0.039) for cardiac event-free survival. In conclusion, these results confirm the potential survival benefit of iron chelation therapy and provide additional evidence on the deleterious effect of iron overload in lower risk MDS patients.
本研究旨在评估血清铁蛋白(SF)所评估的铁过载在输血依赖型低危骨髓增生异常综合征(MDS)患者中的演变情况,描述器官并发症的发生情况,并分析其与铁螯合治疗的关系。这项观察性回顾性研究于2010年3月至2011年3月在47家西班牙医院进行。共纳入263例输血依赖型低危MDS患者(国际预后评分系统[IPSS]低/中-1危或西班牙预后指数[SPI]0-1危),且接受过≥10单位浓缩红细胞(PRBC)输血。在MDS诊断时,患者每月平均接受2.8±3.9单位PRBC输血,8.7%的患者SF≥1000μg/L。在疾病过程中,患者平均接受83.4±83.3单位PRBC输血,36.1%的患者SF≥2500μg/L。心脏、肝脏、内分泌或关节病并发症分别在20.2%、11.4%、9.9%和3.8%的患者中出现/加重。根据研究者判断,铁过载是肝脏并发症(70.0%)和内分泌并发症(26.9%)的主要原因。共有96例(36.5%)患者接受了≥6个月的铁螯合治疗,去铁斯若为最常用的初始螯合治疗药物(71.9%)。接受螯合治疗的患者总生存期(p<0.001)、无白血病生存期(p=0.007)和无心脏事件生存期(p=0.017)均长于未接受螯合治疗的患者。在多变量分析中,年龄(p=0.011)、IPSS(p<0.001)和螯合治疗(p=0.015)是总生存期的预测因素;IPSS(p=0.014)和输血频率(p=0.001)是无白血病生存期的预测因素;螯合治疗(p=0.040)和索罗尔合并症指数(p=0.039)是无心脏事件生存期的预测因素。总之,这些结果证实了铁螯合治疗对生存的潜在益处,并为铁过载在低危MDS患者中的有害影响提供了更多证据。