Department of Leukemia, University of Texas MD Anderson Cancer Center, Houston, TX, United States.
Leuk Lymphoma. 2020 Feb;61(2):288-297. doi: 10.1080/10428194.2019.1660970. Epub 2019 Sep 24.
For decades, the standard induction for patients with acute myeloid leukemia (AML) has been the combination of cytarabine with anthracycline (7 + 3 regimen). In August 2017 the US FDA approved CPX-351 (vyxeos), a liposomal formulation of cytarabine and daunorubicin at a fixed 5:1 molar ratio, for the treatment of adults with newly diagnosed AML with myelodysplasia-related changes (AML-MRC) and therapy-related AML (t-AML). This is the first approved treatment specifically for patients with this subgroup of AML. The approval was based on findings from a multicenter, randomized, open-label, phase III study of CPX-351 Versus 7 + 3 in patients 60-75 years old with newly diagnosed AML-MRC or t-AML. In this study CPX-351 had a higher median OS than 7 + 3 (9.56 vs 5.95 months, HR 0.69; 95% CI: 0.52 to 0.90, = 0.005). In this profile, we review preclinical and clinical data, and discuss limitations and future directions with CPX-351 use in AML.
几十年来，急性髓系白血病 (AML) 患者的标准诱导治疗一直是阿糖胞苷联合蒽环类药物（7+3 方案）。2017 年 8 月，美国食品药品监督管理局 (FDA) 批准 CPX-351（Vyxeos），即阿糖胞苷和柔红霉素以固定 5:1 摩尔比的脂质体制剂，用于治疗新诊断的伴骨髓增生异常相关改变的 AML (AML-MRC) 和治疗相关 AML (t-AML) 成人患者。这是第一种专门针对 AML 亚组患者的批准治疗药物。该批准基于 CPX-351 与 7+3 在 60-75 岁新诊断的 AML-MRC 或 t-AML 患者中进行的多中心、随机、开放标签、III 期研究的结果。在这项研究中，CPX-351 的中位 OS 高于 7+3（9.56 与 5.95 个月，HR 0.69；95%CI：0.52 至 0.90，P=0.005）。在这篇综述中，我们回顾了 CPX-351 在 AML 中的临床前和临床数据，并讨论了其使用的局限性和未来方向。
