人工智能引导的患者分层提高了AMARANTH阿尔茨海默病临床试验的结果和效率
Delshad Vaghari1, Gayathri Mohankumar2, Keith Tan3
1Department of Psychology, University of Cambridge, Cambridge, UK.
Nature communications
|July 17, 2025
概括
在临床试验中,一个人工智能工具精确地分层了阿尔茨海默病 (AD) 患者. 这种方法揭示了lanabecestat在减缓特定患者亚组的认知衰退方面的有效性.
科学领域:
- 神经科学是一个神经科学.
- 人工智能的人工智能
- 临床试验 临床试验
背景情况:
- 阿尔茨海默病 (AD) 药物发现面临挑战,原因是患者异质性和不精确的分层方法.
- 现有的工具缺乏对临床试验中准确的患者选择的敏感性,这可能导致试验结果负面.
- 尽管粉样蛋白减少,但BACE1抑制剂lanabecestat的AMARANTH试验未能显示出认知益处.
研究的目的:
- 开发和验证人工智能引导的工具 (预测性预后模型,PPM),用于在阿尔茨海默病临床试验中精确地分层患者.
- 用人工智能引导的患者分层重新评估lanabecestat的疗效.
- 展示AI在提高AD药物开发效率和成功率方面的潜力.
主要方法:
- 开发一个强大且可解释的人工智能引导的预测预后模型 (PPM).
- 从AMARANTH试验中使用基线数据和PPM的患者重新分层.
- 在不同患者小组中分析认知衰退轨迹,这些小组接受了lanabecestat治疗.
主要成果:
- 通过人工智能引导的重新分层确定了一小组进展缓慢的患者,他们经历了lanabecestat的认知衰退减缓46%.
- 快速进展的患者没有从治疗中获得显著的认知益处.
- 与标准方法 (如β-粉样蛋白阳性) 相比,PPM在患者分层方面表现出更高的精度.
结论:
- 人工智能引导的患者分层显著提高了准确性,并可以揭示传统方法错过的治疗疗效.
- PPM有可能提高未来阿尔茨海默病临床试验的效率和成功.
- 使用人工智能的精确分层可以优化患者选择,从而导致更有针对性和有效的治疗干预措施.
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