在新诊断的急性髓性白血病中,瓜迪西塔与治疗选择:全球第三阶段随机研究
Pierre Fenaux1, Marco Gobbi2, Patricia L Kropf3
1Hôpital Saint-Louis, Paris, France.
Blood advances
|June 5, 2023
概括
这一第三阶段试验发现,瓜迪西塔和标准治疗选择对急性髓性白血病患者同样有效,这些患者不适合接受密集化疗. 在整体患者群体中,没有观察到完全缓解或总生存时间的显著差异.
科学领域:
- 血液学 血液学 血液学
- 在瘤学瘤学.
- 临床药理学 临床药理学
背景情况:
- 急性髓性白血病 (AML) 患者不适合接受密集化疗,治疗选择有限.
- 低甲基化剂是这种患者群体的标准治疗方法.
- 瓜德西塔是一种用于AML治疗的新型低甲基化剂.
研究的目的:
- 为了评估与预先选择的治疗选择 (TC) 相比,瓜迪西塔的疗效和安全性,在AML患者中不适合进行密集的诱导化疗.
- 为了比较瓜迪西塔和TC手臂之间的完全缓解率和整体存活率.
主要方法:
- 第三阶段,随机性研究,将瓜迪西塔 (n=408) 与阿扎西蒂丁,迪西塔或低剂量细胞 (n=407) 的治疗选择 (TC) 进行比较.
- 包括AML不适合强化化疗的患者,其中50%的患者表现不佳 (ECOG 2-3).
- 共同初级终点:完全缓解和整体存活率.
主要成果:
- 在完全缓解率 (19%与17%,P=.48) 或整体存活率 (中位数7.1与8.5个月,P=.73) 中没有显著的差异.
- 对接受≥4个循环的患者进行的一次特设后分析显示,瓜迪西塔的中位生存时间较长 (15.6 vs 13.0个月,P=.02).
- 类似的等级≥3不良事件率 (92% vs 88%),但高发烧性中性质疏松症,中性质疏松症和肺炎的比率更高.
结论:
- 与治疗选择相比,瓜德西塔在AML患者的整体人群中没有显著的疗效差异,这些患者不适合进行密集化疗.
- 安全性概况是可比的,尽管特定的不良事件与瓜迪西塔更频繁.
- 对于特定的子组,如完成至少4个治疗周期的子组,可能需要进一步调查.
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