Stone Richard, Sekeres Mikkael, Garcia-Manero Guillermo
Harvard University School of Medicine, Boston, Massachusetts, USA.
Clin Adv Hematol Oncol. 2009 Aug;7(8):1-14; quiz 2 p following 14.
Myelodysplastic syndrome (MDS) is a clonal hematopoietic disorder characterized by a hyperproliferative bone marrow, cellular dysplasia, and ineffective hematopoiesis. This heterogeneous malignancy is composed of several subtypes, the classification of which has evolved over several years. The treatment of MDS involves improving patient survival and quality of life while decreasing the likelihood of progression to acute myelogenous leukemia (AML). In addition to supportive care with transfusions and hematopoietic growth factors as well as stem cell transplantation, three chemotherapeutic agents have been approved to treat MDS--lenalidomide, azacitidine, and decitabine. In addition, multiple agents and novel combinations are currently in development to treat both MDS AML. Several clinical studies which have investigated these therapeutic approaches, as well as the incorporation of new tools used in the diagnosis of MDS, have been published since the 2008 American Society of Hematology (ASH) Annual Meeting and Exposition, and are discussed here. By becoming familiar with these studies, the physician will be better able to provide the optimal treatment for their patients, as well as become aware of novel therapeutic strategies to offer their patients in ongoing clinical trials.
骨髓增生异常综合征(MDS)是一种克隆性造血疾病,其特征为骨髓增生过度、细胞发育异常以及造血无效。这种异质性恶性肿瘤由几种亚型组成,其分类在数年中不断演变。MDS的治疗旨在提高患者生存率和生活质量,同时降低进展为急性髓系白血病(AML)的可能性。除了通过输血和造血生长因子进行支持性治疗以及干细胞移植外,三种化疗药物已被批准用于治疗MDS——来那度胺、阿扎胞苷和地西他滨。此外,目前有多种药物和新型联合疗法正在研发中,用于治疗MDS和AML。自2008年美国血液学会(ASH)年会及展览会以来,已经发表了几项研究这些治疗方法以及MDS诊断中使用的新工具的临床研究,本文将对此进行讨论。通过熟悉这些研究,医生将能够更好地为患者提供最佳治疗,并了解在正在进行的临床试验中为患者提供的新型治疗策略。