根据性别和种族参与美国ALS临床试验
Mark P Levine1, Sun Young Chung2, Kellen H Quigg3
1Department of Neurology, Emory University School of Medicine, Atlanta, Georgia, USA.
Muscle & nerve
|November 6, 2025
概括
美国肌缩性侧面硬化症 (ALS) 临床试验未招收非白人参与者,并显示女性略有不足. 提高ALS试验招生人数的多样性对于可概括的结果和更快的治疗开发至关重要.
科学领域:
- 临床研究是临床研究.
- 神经学 神经学
- 卫生公平性健康公平性
背景情况:
- 肌缩侧面硬化症 (ALS) 临床试验对于开发新疗法至关重要.
- 了解ALS试验中的参与者人口统计数据对于确保结果的概括性至关重要.
- 以前的研究表明,临床试验招生可能存在差异,但美国ALS试验的具体数据有限.
研究的目的:
- 量化美国的肌缩性侧面硬化症 (ALS) 临床试验中按性别和种族的参与者入学率.
- 将ALS试验中的参与者人口统计数据与人口流行数据进行比较.
- 确定特定的人口群体在ALS研究中的潜在代表性不足.
主要方法:
- 人口数据是从美国ALS临床试验的第2期和第3期收集的,这些临床试验通过文献和clinicaltrials.gov.gov被确定.
- 疾病控制和预防中心 (CDC) 国家ALS注册表和国家监测项目作为患病率基准.
- 参与率与流行率 (PPR) 的比率被计算出来,以评估性别和种族的入学公平性.
主要成果:
- 分析了11个美国ALS试验,1153名参与者.
- 与CDC注册表相比,女性的注册人数不足 (PPR=0.76),而白人参与者注册人数过多 (PPR=1.26).
- 黑人参与者 (PPR=0.34) 和其他/多种族背景的参与者 (PPR=0.35) 显著不足.
结论:
- 美国ALS临床试验显示,非白人参与者的代表性明显不足,女性代表性不足的趋势.
- 解决这些入学差异对于提高ALS临床研究的外部有效性和效率至关重要.
- 扩大ALS患者不同人群的试验参与是加速治疗进展的必要条件.
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