Stone Richard, Sekeres Mikkael, Garcia-Manero Guillermo, Lyons Roger M
Department of Medicine, Harvard Medical School, Boston, Massachusetts, USA.
Clin Adv Hematol Oncol. 2008 Dec;6(12):1-15.
Myelodysplastic syndrome (MDS) is relatively common, with an incidence estimated as high as 50 cases per 100,000 people per year. This cancer mainly affects older (>or=60 years) patients. MDS refers to a collection of hematologic malignancies that share an ineffective production, or hematopoiesis, of normal bone marrow or myeloid cells. As progressive bone marrow failure occurs, patients generally display gradually worsening cytopenias specific to the type of bone marrow cell affected, such as thrombocytopenia or neutropenia. MDS patients often develop disease-related anemia requiring chronic blood transfusion; this can lead to complications including iron overload. As MDS progresses and the number of bone marrow blasts increases, the disease transforms into acute myelogenous leukemia (AML). Several classification systems have been developed to identify and differentiate particular types of MDS. Proper identification is essential, allowing the oncologist to determine prognosis, as well as the optimal therapeutic strategy. Several agents have been developed or are under investigation for the treatment of MDS, with the therapeutic goal of increasing survival and decreasing the rate of AML transformation. Currently, 3 agents are FDA-approved: azacitidine, decitabine, and lenalidomide. This clinical roundtable will discuss the optimal management of patients with each of these approved therapies, as well as the various classification systems used to differentiate MDS subtypes for treatment.
骨髓增生异常综合征(MDS)相对常见,估计发病率高达每年每10万人中有50例。这种癌症主要影响老年(≥60岁)患者。MDS指的是一组血液系统恶性肿瘤,其共同特征是正常骨髓或髓细胞的生成无效,即造血功能异常。随着进行性骨髓衰竭的发生,患者通常会出现因受影响的骨髓细胞类型而异的逐渐加重的血细胞减少,如血小板减少或中性粒细胞减少。MDS患者常出现与疾病相关的贫血,需要长期输血;这可能导致包括铁过载在内的并发症。随着MDS的进展以及骨髓原始细胞数量的增加,该疾病会转化为急性髓系白血病(AML)。已经开发了几种分类系统来识别和区分特定类型的MDS。正确识别至关重要,这能让肿瘤学家确定预后以及最佳治疗策略。已经开发了几种药物或正在对其进行治疗MDS的研究,治疗目标是提高生存率并降低AML转化率。目前,有3种药物已获得美国食品药品监督管理局(FDA)批准:阿扎胞苷、地西他滨和来那度胺。本次临床圆桌会议将讨论这些已批准疗法中每种疗法对患者的最佳管理,以及用于区分MDS亚型以进行治疗的各种分类系统。