随机侧面硬化症临床试验中的消耗和停止:一个元分析
Ruben P A van Eijk1,2, Floris T van Loon3, Jordi W J van Unnik3
1Department of Neurology, UMC Utrecht Brain Center, University Medical Center Utrecht, Heidelberglaan 100, 3584 CX, Utrecht, The Netherlands. r.p.a.vaneijk-2@umcutrecht.nl.
Journal of neurology
|December 12, 2024
概括
随着研究持续时间的增加,amyotrophic lateral sclerosis (ALS) 临床试验中的高消耗率会增加. 需要制定战略,以提高ALS患者的试验完整性和概括性.
科学领域:
- 神经学 神经学
- 临床试验 临床试验
- 生物统计学 生物统计学
背景情况:
- 临床试验中的患者消耗损害了数据完整性和概括性.
- 肌缩侧面硬化症 (ALS) 临床试验特别容易因疾病进展和不良事件而受损.
研究的目的:
- 在ALS临床试验中提供基于证据的消耗率估计.
- 为了确定与研究相关的消耗预测因素.
- 开发一个临床试验中止的预测模型.
主要方法:
- 随机,安慰剂控制的ALS临床试验的系统审查和元分析.
- 量化每个研究手臂的患者停药率.
- 进行元回归分析,以确定研究水平消耗的预测因素.
- 开发一个用于预测研究中断的Web应用程序.
主要成果:
- 包括60项试验,随机分类14493名ALS患者;综合消耗率为32.0%.
- 磨损率差异很大 (3.1%75.7%) 在干预和安慰剂手臂之间是相似的 (或1.08).
- 随访持续时间是唯一重要的预测因素;较长的试验显示出更高的消耗率 (例如,18个月试验的55.6%).
结论:
- 在ALS临床试验中,经历了大量的消耗,受研究持续时间的重大影响.
- 随访时间的延长与患者退休率的增加直接相关.
- 精细的策略至关重要,以减轻消耗,并确保ALS临床试验结果的可靠性.
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