疾病的进展在研究和现实世界帕金森病患者群体中显著不同
Brett K Beaulieu-Jones1,2,3,4,5, Francesca Frau6, Sylvie Bozzi7
1Department of Biomedical Informatics, Harvard Medical School, Boston, MA, 02115, USA. beaulieujones@uchicago.edu.
NPJ Parkinson's disease
|March 14, 2024
概括
帕金森病的进展在研究队伍和现实世界的数据之间有显著差异,在现实世界的人口中观察到的进展更快. 了解这些差异对于临床试验设计和患者护理至关重要.
科学领域:
- 神经学 神经学
- 生物统计学 生物统计学
- 数据科学数据科学数据科学
背景情况:
- 使用现实世界的证据 (RWD) 描述帕金森病 (PD) 的进展对于改善临床试验设计和识别患者亚群至关重要.
- 自然语言处理 (NLP) 的进步,包括大语言模型 (LLM),使得比以前更细致的人口比较成为可能.
- 这项研究比较了两个已建立的研究队列和两个RWD衍生的队列,以界定人口差异.
研究的目的:
- 描述和比较帕金森病在不同研究和现实世界数据群体中的进展.
- 确定研究环境中的患者群体与现实世界临床实践中的患者群体之间的系统差异.
- 为利用多样化的数据源和减轻PD研究偏见的策略提供信息.
主要方法:
- 包括研究群体:哈佛生物标志物研究 (HBS) 和福克斯洞察.
- 包括真实世界数据 (RWD) 种群:Optum集成索赔-EHR和Mass General Brigham (MGB) 无标识数据.
- 利用结构化EHR数据,补充LLM增强的NLP,用于MGB的PD进展测量.
主要成果:
- 与HBS研究队伍相比,MGB RWD队列的运动和认知进展更快 (例如,到Hoehn & Yahr阶段3的中位时间:5.6年与>10年).
- 在RWD人群中,患者的诊断时间比研究人群 (平均年龄60.4) 晚得多 (平均年龄72.2).
- 与研究队伍相比,在RWD队列中 (诊断后平均2.3年) 推迟了PD药物的开始.
结论:
- 在研究和现实世界的人口之间,关于帕金森病的诊断,进展率和治疗开始方面存在显著的分歧.
- 这些差异可能受到研究队伍中选择偏差和现实世界患者群体内在差异的结合的影响.
- 强调需要利用多个来源的数据,并仔细考虑PD研究和临床试验设计中的潜在偏见.
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