文章：

骨髓增生异常综合征2018年当前治疗算法

Myelodysplastic syndromes current treatment algorithm 2018

原文发布日期：2018-05-24

DOI: 10.1038/s41408-018-0085-4

类型: Current Treatment Algorithm

开放获取: 是

英文摘要：

Myelodysplastic syndromes (MDS) include a group of clonal myeloid neoplasms characterized by cytopenias due to ineffective hematopoiesis, abnormal blood and marrow cell morphology, and a risk of clonal evolution and progression to acute myeloid leukemia (AML). Because outcomes for patients with MDS are heterogeneous, individual risk stratification using tools such as the revised International Prognostic Scoring System (IPSS-R) is important in managing patients—including selecting candidates for allogeneic hematopoietic stem cell transplantation (ASCT), the only potentially curative therapy for MDS. The IPSS-R can be supplemented by molecular genetic testing, since certain gene mutations such as TP53 influence risk independent of established clinicopathological variables. For lower risk patients with symptomatic anemia, treatment with erythropoiesis-stimulating agents (ESAs) or lenalidomide (especially for those with deletion of chromosome 5q) can ameliorate symptoms. Some lower risk patients may be candidates for immunosuppressive therapy, thrombopoiesis-stimulating agents, or a DNA hypomethylating agent (HMA; azacitidine or decitabine). Among higher risk patients, transplant candidates should undergo ASCT as soon as possible, with HMAs useful as a bridge to transplant. Non-transplant candidates should initiate HMA therapy and continue if tolerated until disease progression. Supportive care with transfusions and antimicrobial drugs as needed remains important in all groups.

摘要翻译： 

骨髓增生异常综合征（MDS）是一组克隆性髓系肿瘤，其特征包括无效造血导致的血细胞减少、外周血与骨髓细胞形态异常，以及存在克隆性进展为急性髓系白血病（AML）的风险。由于MDS患者的预后具有异质性，采用修订版国际预后评分系统（IPSS-R）等工具进行个体化风险分层对临床管理至关重要——包括筛选适合异基因造血干细胞移植（ASCT）的候选者，这是目前MDS唯一可能根治的疗法。IPSS-R可结合分子遗传学检测进行补充，因为TP53等基因突变对风险的影响独立于既定临床病理学指标。对于伴症状性贫血的低危患者，使用促红细胞生成剂（ESA）或来那度胺（尤其适用于5号染色体长臂缺失患者）可改善症状。部分低危患者可考虑免疫抑制治疗、血小板生成刺激剂或DNA低甲基化剂（HMA，如阿扎胞苷或地西他滨）。在高危患者中，符合移植条件者应尽快接受ASCT，HMA可作为移植前的桥接治疗。非移植候选者应启动HMA治疗，若可耐受则持续用药直至疾病进展。所有患者群体均需根据病情接受输血及抗微生物药物等支持治疗。

原文链接：

Myelodysplastic syndromes current treatment algorithm 2018