基于临床试验替代结果的可解释的机器学习模型,用于预测头癌的总生存率
W Hwang1, H A Jung2, L J Worth3
1Department of Medicine, Massachusetts General Hospital, Boston, USA; Lunit Inc., Seoul, Republic of Korea.
ESMO open
|September 4, 2025
概括
这项研究使用替代结果,如应答率和患者因子,开发了头癌整体生存的预测模型. 该模型有助于解释复发性或转移性脑甲状腺癌的早期临床试验结果.
科学领域:
- 癌症学
- 临床试验
- 生物统计学
背景情况:
- 复发性或转移性头皮状细胞癌 (R/ M HNSCC) 存在重大治疗挑战.
- 整体存活率 (OS) 是一个关键的终点,但替代疗效结果通常用于早期试验.
- 根据早期代用标记和患者特征预测OS对于R/M HNSCC药物开发至关重要.
研究的目的:
- 在R/ M HNSCC临床试验中评估代用疗效结果与OS之间的关系.
- 开发和验证一个包含替代结果和患者基线特征的OS预测模型.
- 改善R/M HNSCC早期临床试验数据的解释.
主要方法:
- 从一线R/M HNSCC试验系统地收集数据 (2010年1月至2025年3月).
- 通过替代结果 (ORR,PFS等) 预测OS的五种机器学习模型的评估 和患者因子 (HPV,ECOG,PD- L1).
- 使用单一机构的追溯数据和模拟数据集进行验证.
主要成果:
- 从52个出版物中分析了90个治疗臂 (26个基于ICI,64个非ICI).
- 一年生存率与平均生存率的相关性最强 (r=0. 87).
- 弹性网模型准确预测了操作系统 (测试组r=0.74,验证组r=0.75),其中1年操作系统率和ORR是关键预测因素.
结论:
- 弹性网模型有效地将代用疗效终点与R/ M HNSCC中的中位数OS联系起来.
- 这种模型有助于解释早期临床试验结果.
- 它有助于预测R/ M HNSCC患者的OS益处,特别是考虑到HPV状态和ECOG性能状态等因素.
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