第58届美国血液学会(ASH)年会介绍了低危骨髓增生异常综合征(MDS)患者的治疗。贫血是低危MDS最常见的单一症状,应用红细胞生成素(ESA)治疗可能有效。ESA剂量和治疗时间的选择对起效有关键作用。若ESA治疗无效,其他治疗选择包括来那度胺(针对del5q-综合征)、去甲基化治疗和临床试验。二线治疗的选择必须考虑到患者的生物学、细胞遗传学个体特点以及易感性和合并疾病。白细胞和血小板减少很少单独出现。针对单纯血小板减少症的促血小板生成药物已经进入临床试验,但是面临一些安全问题。针对中性粒细胞减少症的治疗中,粒细胞刺激因子是对症支持治疗,免疫抑制治疗主要应用于各类血细胞减少和低危MDS。造血干细胞移植也是低危MDS的一种治疗选择,但需要仔细评估相关不良反应和治疗风险。
New progress of treatment of" low-risk myelodysplastic syndromes (MDS) reported in the 58th American Socienty of Hematology (ASH) Annual Meetings was reviewed. Anemia is a single common symptom of low-risk MDS, and erythropoietic-stinlulating agents (ESA) may be effective. The dose and duration of erythropoietic-stimulating agents (ESA) are critical to determine efficacy. If the treatment of ESA failed, the available options may include lenalidomide (approved for delSq positive cases), hypomethylating agents and a rather large number of experimental agents. The choice for the second-line treatment must consider the biologic, cytogenetic and molecular-identified characteristics of individual patient, as well as frailty and comorbidities. Other cytopenias rarely appear alone. Thrombomimetic agents for thrombocytopenia has been proposed in clinical trials, but there were some safety issues. Although neutropenia is targeted symptomatically with growth factor supportive care, the immunosuppressive therapy is indicated mainly for pancytopenic and hypoplastic low-risk MDS. Finally, hematopoietic stem cell transplantion is the curative option also for low-risk MDS, but it should be carefully evaluated to balancing toxicity and the possibility of survival advantage.