低甲基化剂加上修改后的原始治疗方案与基于venetoclax的治疗方案相比,基于急性髓性白血病新诊断患者的分子特征:一个多中心队列研究
Guangyang Weng1, Jingya Huang2, Xin He3
1Department of Hematology, The First Affiliated Hospital of Shenzhen University, Shenzhen Second People's Hospital, Shenzhen, China.
Annals of hematology
|September 18, 2023
概括
低甲基化剂 (HMAs) 加上原始化疗法显示为新诊断的急性髓性白血病 (AML) 基于Venetoclax (VEN) 的治疗方法的替代方案. 虽然总体反应率相似,但VEN疗法在特定的分子子组中优越.
科学领域:
- 血液学 血液学 血液学
- 在瘤学瘤学.
- 临床研究 临床研究
背景情况:
- 基于Venetoclax (VEN) 的治疗方案是新诊断的老年或身体不适的患者急性髓性白血病 (ND AML) 的标准.
- 低甲基化剂 (HMA) 加上原始化方案正在被探索为替代疗法,因为在单臂研究中有希望的疗效.
研究的目的:
- 在ND AML患者中,比较HMA加原始化疗法与VEN基疗法的疗效和整体存活率 (OS).
- 进行分子子组分析,以确定治疗反应的潜在差异.
主要方法:
- 追溯的多中心队列研究.
- 招募了294名ND AML患者:HMA+原始组167名,基于VEN的组127名.
- 比较治疗反应 (ORR,CR,CRi,MLFS) 和OS,随访时间中位数为10.1个月.
主要成果:
- 总体响应率 (ORR) 类似:HMA+原始剂65.3%,基于VEN的疗法70.9%.
- 完全缓解 (CR) 和CR与不完全的血液恢复 (CRi) 率在各组之间没有显著差异 (p > 0.05).
- 在患有FLT3,IDH1/2和NPM1突变的患者中,VEN疗法显示出优异的CR/CRi,与HMA+原始化 (p ≤0.02) 相比.
结论:
- HMA加上修改后的初始化疗法可以作为ND AML的可行的替代治疗方法.
- 基于VEN的疗法在特定的分子子组 (FLT3,IDH1/2,NPM1突变) 中表现出优势.
- 分子特征对于指导ND AML治疗选择至关重要.
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