Department of Hematology/Oncology, University of Freiburg Medical Center, Freiburg, Germany.
Semin Hematol. 2012 Oct;49(4):330-41. doi: 10.1053/j.seminhematol.2012.08.001.
Patients with myelodysplastic syndromes (MDS), especially those with high-risk disease, other comorbidities, or of advanced age, still have a limited prognosis. In addition to cytotoxic chemotherapies, hypomethylating agents such as decitabine (5-aza-2'-deoxycytidine) and azacitidine (5-azacytidine), have been approved during the past decade and represent a very important option for the treatment of MDS today. Due to their lower toxicity compared to conventional chemotherapy, hypomethylating agents are often a safe and feasible alternative also for frail patients. Decitabine has been shown to be active in numerous studies including International Prognostic Scoring System (IPSS) intermediate-1 to high risk patients, in secondary acute myeloid leukemia (AML) arising from MDS, and in MDS with poor-risk cytogenetics. Furthermore, decitabine has been studied in the allograft setting and in combination treatment regimens, and may play a role in epigenetic "priming" before conventional chemotherapy. This review summarizes the results of clinical trials and experiences with decitabine not only in first-line and single-agent treatment regimens but also as second-line treatment after prior treatment failure, in the context of the allograft setting and as part of combination treatment regimens.
骨髓增生异常综合征（MDS）患者，尤其是高危疾病、其他合并症或高龄患者，预后仍然有限。除细胞毒性化疗外，过去十年中批准了低甲基化剂，如地西他滨（5-氮杂-2'-脱氧胞苷）和阿扎胞苷（5-氮杂胞苷），它们是当今 MDS 治疗的非常重要的选择。由于与传统化疗相比毒性较低，低甲基化剂通常也是脆弱患者的安全且可行的替代方案。地西他滨在许多研究中均显示出活性，包括国际预后评分系统（IPSS）中危-高危患者、源自 MDS 的继发性急性髓系白血病（AML）以及伴有不良细胞遗传学的 MDS。此外，地西他滨已在同种异体移植环境中和联合治疗方案中进行了研究，并且可能在常规化疗前的表观遗传“启动”中发挥作用。这篇综述总结了地西他滨的临床试验结果和经验，不仅在一线和单药治疗方案中，而且在先前治疗失败后的二线治疗中，在同种异体移植环境中和联合治疗方案中都有应用。
