Department of Haematology, University Hospital Geelong, Geelong, 3220, Australia.
Australian Centre for Blood Diseases, Monash University, Melbourne, 3004, Australia.
Future Oncol. 2021 Jul;17(20):2563-2571. doi: 10.2217/fon-2020-1318. Epub 2021 Mar 26.
Two oral hypomethylating agents, oral azacitidine (CC-486) and decitabine/cedazuridine (ASTX727), have recently entered the clinical domain. CC-486 has been shown to improve overall survival as maintenance therapy for older patients with acute myeloid leukemia in complete remission, whereas the combination of decitabine with cedazuridine, a cytidine deaminase inhibitor, is indicated for the treatment of adult patients with myelodysplastic syndromes and chronic myelomonocytic leukemia with intermediate-1, or higher, International Prognostic Scoring System risk. This article briefly summarizes the clinical development of both drugs, the pivotal studies that led to their approval and some of the issues faced in extending the use of these drugs to other indications.
两种口服低甲基化剂，口服阿扎胞苷（CC-486）和地西他滨/西他滨（ASTX727），最近已进入临床领域。CC-486 已被证明可改善完全缓解的老年急性髓系白血病患者的总生存期，而地西他滨与胞嘧啶脱氨酶抑制剂西他滨的联合应用，适用于治疗中危-1 或更高危国际预后评分系统的成人骨髓增生异常综合征和慢性粒单核细胞白血病患者。本文简要总结了这两种药物的临床开发情况，以及导致其获批的关键性研究，以及在将这些药物扩展用于其他适应证时所面临的一些问题。
