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骨髓增生异常综合征的当前和未来的治疗选择。

Current and future management options for myelodysplastic syndromes.

机构信息

Department of Leukemia, The University of Texas, M.D. Anderson Cancer Center, Houston, Texas 77030, USA.

出版信息

Drugs. 2010 Jul 30;70(11):1381-94. doi: 10.2165/11537920-000000000-00000.

DOI:10.2165/11537920-000000000-00000
PMID:20614946
Abstract

The management of the myelodysplastic syndromes (MDS) requires insight into the complex biology of the disease. Despite this challenge, two recent developments have contributed significantly to advancements in the treatment of MDS: (i) improvements in classification systems and prognostic models; and (ii) the emergence of US FDA-approved agents such as lenalidomide, azacitidine and decitabine. Prior to these developments, supportive care measures consisting of blood and platelet transfusions, haematopoietic growth factors and antimicrobials remained standard of care for the treatment of MDS. As a result of these developments, clinicians are able to provide patient-tailored therapy for specific MDS subgroups. Clinical trials addressing combination therapies with multiple investigational agents as well as novel combination regimens are ongoing. This review focuses on supportive care modalities, the approved agents indicated for the treatment of MDS and future directions for the treatment of MDS, including agents under clinical investigation.

摘要

骨髓增生异常综合征(MDS)的治疗需要深入了解该疾病的复杂生物学特性。尽管存在这一挑战,但最近的两项进展极大地推动了 MDS 的治疗进展:(i)分类系统和预后模型的改进;(ii)美国食品和药物管理局(FDA)批准的药物如来那度胺、阿扎胞苷和地西他滨的出现。在此之前,输血和血小板输注、造血生长因子和抗生素等支持性护理措施仍是 MDS 治疗的标准护理方法。由于这些进展,临床医生能够为特定 MDS 亚组患者提供个体化治疗。目前正在进行针对多种研究性药物联合治疗以及新型联合方案的临床试验。这篇综述重点介绍支持性护理方式、FDA 批准用于治疗 MDS 的药物以及 MDS 治疗的未来方向,包括正在进行临床试验的药物。

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