Schuh Andre C, Döhner Hartmut, Pleyer Lisa, Seymour John F, Fenaux Pierre, Dombret Hervé
Princess Margaret Cancer Centre, Toronto, Ontario, Canada.
Universitätsklinikum Ulm, Ulm, Germany.
Crit Rev Oncol Hematol. 2017 Aug;116:159-177. doi: 10.1016/j.critrevonc.2017.05.010. Epub 2017 Jun 3.
Azacitidine is recommended front-line treatment for older patients with acute myeloid leukemia (AML) who are not candidates for intensive treatment regimens, and was recently granted approval in the European Union for treatment of adult AML. Reviewed here is azacitidine experience in AML, including: mechanistic and pharmacokinetic data; safety and efficacy in controlled trials; treatment effects in AML subpopulations defined by disease characteristics; experience in unselected patients treated in the community setting; clinical outcomes relative to other approved AML therapies; and experience with azacitidine-based combination treatment regimens. Collectively, these data suggest that (a) azacitidine may prolong overall survival to a similar or greater extent than do other approved AML treatments, but with less toxicity, (b) azacitidine may be the preferred treatment option for older patients with unfavorable cytogenetics, and (c) experience and outcomes with azacitidine in the clinic are similar to those seen in clinical trials. Continued investigation of combination regimens on an azacitidine backbone is warranted.
阿扎胞苷被推荐作为不适合接受强化治疗方案的老年急性髓系白血病（AML）患者的一线治疗药物，并且最近在欧盟获得了治疗成人AML的批准。本文回顾了阿扎胞苷治疗AML的经验，包括：作用机制和药代动力学数据；对照试验中的安全性和有效性；根据疾病特征定义的AML亚群中的治疗效果；在社区环境中接受治疗的未选择患者的经验；相对于其他已批准的AML疗法的临床结果；以及基于阿扎胞苷的联合治疗方案的经验。总体而言，这些数据表明：（a）阿扎胞苷可能比其他已批准的AML治疗方法在更大程度上延长总生存期，且毒性更低；（b）阿扎胞苷可能是细胞遗传学不良的老年患者的首选治疗选择；（c）阿扎胞苷在临床中的经验和结果与临床试验中所见相似。有必要继续研究以阿扎胞苷为基础的联合治疗方案。
