Department of Biomedical and Clinical Sciences Luigi Sacco, Luigi Sacco Hospital Milan, University of Milan, Milan, Italy.
Hematology and Transfusion Medicine, Department of Oncology and Onco-Hematology, L. Sacco University Hospital, University of Milan, Milan, Italy.
Med Oncol. 2018 Apr 19;35(5):76. doi: 10.1007/s12032-018-1135-2.
The myelodysplastic syndromes (MDSs) are clonal hematopoietic stem cell disorders. The International Prognostic Score System (IPSS) groups MDS in lower-risk (IPSS low and intermediate-1) and higher-risk disease (IPSS intermediate-2 and high). AML transformation is the main concern in higher-risk MDS, while anemia and transfusion dependency represent the major issues for low-risk MDS patients. Improving erythropoiesis, and eliminating fatigue and symptoms, is the main therapeutic goal for low-risk MDS patients. Around 50% of MDS patients present with anemia with an Hb level < 100 g/L. Severe anemia increases the negative effects of comorbidities, such as heart and lung failure. Erythropoiesis-stimulating agents (ESAs), with or without granulocyte colony-stimulating factor, induce erythroid response rates in 40-50% of lower-risk anemic MDS patients. The median response duration of 24 months. Apoptosis of erythroid cells is inhibited by ESAs leading to erythrocyte production. Our paper considers the state of the art of treatment of anemia in low-risk MDS patients and the treatment options in MDS resistant or refractory to ESAs.
骨髓增生异常综合征(MDS)是一种克隆性造血干细胞疾病。国际预后评分系统(IPSS)将 MDS 分为低危(IPSS 低和中-1)和高危疾病(IPSS 中-2 和高)。AML 转化是高危 MDS 的主要关注点,而贫血和输血依赖是低危 MDS 患者的主要问题。改善红细胞生成、消除疲劳和症状是低危 MDS 患者的主要治疗目标。大约 50%的 MDS 患者出现贫血,Hb 水平<100g/L。严重的贫血会增加合并症的负面影响,如心肺衰竭。红细胞生成刺激剂(ESA),联合或不联合粒细胞集落刺激因子,可诱导 40-50%低危贫血性 MDS 患者的红细胞反应率。中位反应持续时间为 24 个月。ESA 抑制红细胞细胞凋亡,导致红细胞生成。我们的论文考虑了低危 MDS 患者贫血治疗的最新进展,以及对 ESA 耐药或难治性 MDS 的治疗选择。