文章：

CPX-351治疗继发性急性髓系白血病有效，并提高了异基因造血干细胞移植的可行性：意大利同情用药项目结果

CPX-351 treatment in secondary acute myeloblastic leukemia is effective and improves the feasibility of allogeneic stem cell transplantation: results of the Italian compassionate use program

原文发布日期：2020-10-06

DOI: 10.1038/s41408-020-00361-8

类型: Article

开放获取: 是

英文摘要：

Secondary acute myeloid leukemia (sAML) poorly responds to conventional treatments and allogeneic stem cell transplantation (HSCT). We evaluated toxicity and efficacy of CPX-351 in 71 elderly patients (median age 66 years) with sAML enrolled in the Italian Named (Compassionate) Use Program. Sixty days treatment-related mortality was 7% (5/71). The response rate at the end of treatment was: CR/CRi in 50/71 patients (70.4%), PR in 6/71 (8.5%), and NR in 10/71 (19.7%). After a median follow-up of 11 months relapse was observed in 10/50 patients (20%) and 12 months cumulative incidence of relapse (CIR) was 23.6%. Median duration of response was not reached. In competing risk analysis, CIR was reduced when HSCT was performed in first CR (12 months CIR of 5% and 37.4%, respectively, for patients receiving (=20) or not (=30) HSCT, p = 0.012). Twelve-months OS was 68.6% (median not reached). In landmark analysis, HSCT in CR1 was the only significant predictor of longer survival (12 months OS of 100 and 70.5%, for patients undergoing or not HSCT in CR1, respectively, p = 0.011). In conclusion, we extend to a real-life setting, the notion that CPX is an effective regimen for high risk AML patients and may improve the results of HSCT.
 

摘要翻译： 

继发性急性髓系白血病（sAML）对常规治疗和异基因干细胞移植（HSCT）反应不佳。我们评估了CPX-351在71名老年sAML患者（中位年龄66岁）中的毒性和疗效，这些患者参与了意大利命名（同情）使用计划。60天治疗相关死亡率为7%（5/71）。治疗结束时的反应率为：50/71名患者（70.4%）达到完全缓解/完全缓解伴不完全血细胞恢复，6/71名患者（8.5%）达到部分缓解，10/71名患者（19.7%）为无缓解。中位随访11个月后，10/50名患者（20%）观察到复发，12个月累积复发率（CIR）为23.6%。中位反应持续时间未达到。在竞争风险分析中，当在首次完全缓解时进行HSCT时，CIR降低（接受HSCT的患者（=20）和未接受HSCT的患者（=30）的12个月CIR分别为5%和37.4%，p=0.012）。12个月总生存期（OS）为68.6%（中位未达到）。在里程碑分析中，CR1时进行HSCT是较长生存期的唯一显著预测因子（在CR1时接受或未接受HSCT的患者12个月OS分别为100%和70.5%，p=0.011）。总之，我们在真实世界环境中扩展了CPX是高风险AML患者有效方案的观点，并且可能改善HSCT的结果。

原文链接：

CPX-351 treatment in secondary acute myeloblastic leukemia is effective and improves the feasibility of allogeneic stem cell transplantation: results of the Italian compassionate use program