Mies Anna, Platzbecker Uwe
Department of Internal Medicine I, University Hospital Carl Gustav Carus, Technische Universität Dresden, Germany, German Cancer Consortium (DKTK), and German Cancer Research Center (DKFZ), Heidelberg, Germany.
Department of Internal Medicine I, University Hospital Carl Gustav Carus, Technische Universität Dresden, Germany, German Cancer Consortium (DKTK), and German Cancer Research Center (DKFZ), Heidelberg, Germany.
Semin Hematol. 2017 Jul;54(3):141-146. doi: 10.1053/j.seminhematol.2017.06.004. Epub 2017 Jul 4.
Patients with lower-risk myelodysplastic syndromes (MDS) are mainly affected by chronic anemia and fatigue. Treatment strategies aim to improve anemia and quality of life, as well as iron overload due to red blood cell transfusion support. To promote proliferation and differentiation of erythropoiesis, erythropoiesis-stimulating agents (ESAs) such as erythropoietin (EPO) and mimetics are applied as first-line therapy in a large fraction of lower-risk MDS patients. In general, ESAs yield favorable responses in about half of the patients, although responses are often short-lived. In fact, many ESA-refractory patients harbor defects in late-stage erythropoiesis downstream of EPO action. Novel transforming growth factor (TGF)-β superfamily inhibitors sotatercept and luspatercept represent a promising approach to alleviate anemia by stimulating erythroid differentiation.
低危骨髓增生异常综合征（MDS）患者主要受慢性贫血和疲劳影响。治疗策略旨在改善贫血和生活质量，以及因红细胞输血支持导致的铁过载。为促进红细胞生成的增殖和分化，促红细胞生成素（ESA）如促红细胞生成素（EPO）及其模拟物在大部分低危MDS患者中作为一线治疗应用。总体而言，ESA在约一半的患者中产生良好反应，尽管反应通常是短暂的。事实上，许多对ESA难治的患者在EPO作用下游的晚期红细胞生成中存在缺陷。新型转化生长因子（TGF）-β超家族抑制剂索他西普和罗沙司他是通过刺激红系分化来缓解贫血的一种有前景的方法。
