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在临床试验中用于现场风险预测的机器学习:开发,外部验证和在现场资格认证中的操作应用
1Development Operations, AstraZeneca Global R&D (China) Co., Ltd., 88 Xizang Road, Jing'an District, Shanghai 200085, China.
International journal of medical informatics
|February 25, 2026
概括
一个新的机器学习工具准确地预测了罕见疾病的临床试验场所风险,如西氨基酸心肌病 (ATTR-CM). 这种健康信息学方法改善了地点的选择和资格,证明了在不同疾病中广泛的适用性.
科学领域:
- 医疗信息学 医疗信息学
- 临床研究中的机器学习
- 临床试验操作 临床试验操作
背景情况:
- 临床试验地点的选择和资格是具有挑战性的,特别是对于罕见疾病,如西氨基酸心肌病 (ATTR-CM).
- 目前的风险评估方法缺乏系统的验证,临床试验数据在主动风险预测中仍未得到充分利用.
- 需要数据驱动的工具来优化临床试验操作和现场管理.
研究的目的:
- 开发和验证基于机器学习 (ML) 的临床决策支持工具,用于预测ATTR-CM临床试验中的场所风险.
- 通过使用杜氏肌肉发育不良 (DMD) 试验数据,评估该工具的通用性和跨疾病适用性.
- 设计一个操作工作流程,将ML工具集成到现有的场地资格风险评估 (SQRA) 流程中.
主要方法:
- 分析了460个ATTR-CM和761个DMD试验地点,使用来自ClinicalTrials.gov.gov的数据.
- 开发一种混合风险评分系统,将专家共识和 LASSO 优化结合起来.
- 在DMD研究中,使用5倍交叉验证,时间验证和外部验证来训练和验证四个ML算法.
- 利用SHAP分析来分析模型的解释性,并确定关键的风险预测因素.
主要成果:
- 该ML工具在ATTR-CM站点风险预测中达到98.91%的准确性,在DMD外部验证中达到81.21%的准确性.
- SHAP分析确定了数据质量风险,屏幕失效率和入学风险作为重要的预测因素.
- 在ATTR-CM和DMD之间的特征重要性排名中,高一致性 (斯皮尔曼 ρ = 0.905) 证明了跨疾病的概括性.
结论:
- 一个运行准备好的基于ML的工具,用于临床试验场所风险预测,已成功开发和验证.
- 该工具在ATTR-CM中表现出高准确度和跨不同疾病的概括性,支持其整合到SQRA工作流程中.
- 虽然有希望,但与实地真相监测结果和作战部署的进一步验证对于确认预计的好处至关重要.
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