摘要低危骨髓增生异常综合征(MDS)是指国际预后积分系统修订版(IPSS-R)评分≤3.5分的MDS,支持治疗仍是主要的治疗手段.对于染色体5q缺失[del(5q)]MDS患者,如果内源性促红细胞生成素(EPO)水平<500 U/L(最好<200 U/L),红系造血刺激剂(ESA)仍是最佳选择.对应用EPO无效或脱离输血后复发的患者,可以选择免疫抑制剂、转化生长因子抑制剂和来那度胺.del(5q)患者可以从来那度胺长期受益.血小板减少造成严重出血的情况较少,血小板生成素受体类似物可以减少出血,提高血小板数,可用于骨髓原始细胞比例<0.05的患者.治疗失败或有进展为高危MDS和急性髓系白血病风险者,可能需要造血干细胞移植治疗.文章结合第59届美国血液学会(ASH)年会报道,介绍有关低危MDS的治疗策略.

abstractsThe support treatment of low-risk myelodysplastic syndromes (MDS) patients whose revised international prognostic score system (IPSS-R) is defined as 3.5 points remains the main treatment. The erythropoiesis stimulating agent (ESA) is the best choice for patients with del (5q) MDS as long as the endogenous erythropoietin (EPO) level is less than 500 U/L (preferably<200 U/L). The application of EPO for patients with treatment failure or relapsed after transfusion, chooses are immunosuppressant, transforming growth factor beta inhibitors and lenalidomide. Del (5q) syndrome could benefit from lenalidomide, and some patients after discontinuation of treatment still have been stable in peripheral blood. Thrombocytopenia caused serious hemorrhage rarely; thrombopoietin receptor analogs could reduce bleeding and improve the platelet count. These drugs can be used in patients with bone marrow blast counts of < 0.05. If treatment failed or advanced to high-risk MDS or transformed to acute myeloid leukemia, allogeneic hematopoietic stem cell transplantation might be required. Combined with reports in the 59th American Society of Hematology (ASH) Annual Meeting, this article describes the treatment strategies for low-risk MDS.