a Department of Hematology , Medical University of Lodz and Copernicus Memorial Hospital , Lodz , Poland.
Expert Opin Pharmacother. 2018 Nov;19(16):1835-1839. doi: 10.1080/14656566.2018.1524875. Epub 2018 Sep 24.
Acute myeloid leukemia (AML) remains a poor prognosis hematological malignancy. The introduction of aggressive chemotherapy with allogeneic stem cell transplantation has resulted in improved clinical outcomes in younger patients. However, the treatment results in unfit elderly AML population remain disappointing. New strategies should be introduced to improve the prognosis in this group of patients. Areas covered: This review presents and discusses the mechanism of action, safety and efficacy of sapacitabine in AML patients. Expert opinion: Sapacitabine, a novel nucleoside analog, seemed to be a promising new agent for AML treatment. Its oral bioavailability and tolerable toxicity profile allow the drug to be used in an outpatient setting, especially in elderly unfit patients. Sapacitabine is known to have antileukemic activity in randomized clinical trials. In AML patients, sapacitabine monotherapy offered no advantage over low-intensity cytarabine treatment, and the combination of sapacitabine with decitabine was not significantly more effective than decitabine alone. However, the oral administration of sapacitabine allows it to be used in AML maintenance therapy.
急性髓细胞白血病（AML）仍然是一种预后不良的血液系统恶性肿瘤。采用强化化疗联合异基因造血干细胞移植可改善年轻患者的临床结局。然而，治疗效果在不适合进行高强度治疗的老年 AML 患者中仍然令人失望。应该引入新的策略来改善这组患者的预后。
本综述介绍并讨论了沙柏西他滨在 AML 患者中的作用机制、安全性和疗效。
沙柏西他滨是一种新型核苷类似物，似乎是治疗 AML 的一种有前途的新药。其口服生物利用度和可耐受的毒性特征使其可在门诊环境中使用，尤其是在不适合进行高强度治疗的老年患者中。沙柏西他滨在随机临床试验中具有抗白血病活性。在 AML 患者中，沙柏西他滨单药治疗与低强度阿糖胞苷治疗相比没有优势，沙柏西他滨联合地西他滨治疗并不比单独用地西他滨更有效。然而，沙柏西他滨的口服给药使其可用于 AML 的维持治疗。
