文章：

CPX-351作为急性髓系白血病患者一线治疗的现实世界经验

Real-world experience of CPX-351 as first-line treatment for patients with acute myeloid leukemia

原文发布日期：2021-10-04

DOI: 10.1038/s41408-021-00558-5

类型: Article

开放获取: 是

英文摘要：

To investigate the efficacy and toxicities of CPX-351 outside a clinical trial, we analyzed 188 patients (median age 65 years, range 26–80) treated for therapy-related acute myeloid leukemia (t-AML, 29%) or AML with myelodysplasia-related changes (AML-MRC, 70%). Eighty-six percent received one, 14% two induction cycles, and 10% received consolidation (representing 22% of patients with CR/CRi) with CPX-351. Following induction, CR/CRi rate was 47% including 64% of patients with available information achieving measurable residual disease (MRD) negativity (<10−3) as measured by flow cytometry. After a median follow-up of 9.3 months, median overall survival (OS) was 21 months and 1-year OS rate 64%. In multivariate analysis, complex karyotype predicted lower response (p = 0.0001), while pretreatment with hypomethylating agents (p = 0.02) and adverse European LeukemiaNet 2017 genetic risk (p < 0.0001) were associated with lower OS. Allogeneic hematopoietic cell transplantation (allo-HCT) was performed in 116 patients (62%) resulting in promising outcome (median survival not reached, 1-year OS 73%), especially in MRD-negative patients (p = 0.048). With 69% of patients developing grade III/IV non-hematologic toxicity following induction and a day 30-mortality of 8% the safety profile was consistent with previous findings. These real-world data confirm CPX-351 as efficient treatment for these high-risk AML patients facilitating allo-HCT in many patients with promising outcome after transplantation.
 

摘要翻译： 

为探究CPX-351在临床试验之外的有效性和毒性，我们分析了188例接受治疗的患者（中位年龄65岁，范围26-80岁），这些患者包括治疗相关急性髓系白血病（t-AML，占29%）或伴骨髓增生异常相关改变的急性髓系白血病（AML-MRC，占70%）。其中86%的患者接受了一个诱导周期治疗，14%接受两个诱导周期，10%的患者（占达到CR/CRi患者的22%）使用CPX-351进行了巩固治疗。诱导治疗后，完全缓解/血细胞计数不完全恢复的完全缓解（CR/CRi）率为47%，在可获得信息的患者中，64%通过流式细胞术检测达到可测量残留病（MRD）阴性（<10⁻³）。中位随访9.3个月后，中位总生存期（OS）为21个月，1年OS率为64%。多变量分析显示，复杂核型预示较低缓解率（p=0.0001），而既往接受过低甲基化药物治疗（p=0.02）及欧洲白血病网2017年遗传学风险分级为高危（p<0.0001）与较低总生存期相关。116例患者（62%）接受了异基因造血细胞移植（allo-HCT），结果良好（中位生存期未达到，1年OS率73%），尤其在MRD阴性患者中表现显著（p=0.048）。安全性方面，69%的患者在诱导治疗后出现III/IV级非血液学毒性，30天死亡率为8%，这与既往研究结果一致。这些真实世界数据证实CPX-351是治疗这类高危AML患者的有效方案，能为许多患者接受异基因移植创造条件，并获得良好的移植后结局。

原文链接：

Real-world experience of CPX-351 as first-line treatment for patients with acute myeloid leukemia