在子宫内膜异位症疼痛试验中,随机选择的安慰剂对照组与历史的安慰剂对照组的替换:一个用另一项试验的历史对照数据重新评估试验数据的案例研究
Marius Sieverding1,2, Christoph Gerlinger2,3, Christian Seitz4,5
1Berlin School of Public Health, Charite Medical Faculty Berlin, Berlin, Germany.
BMJ open
|July 21, 2023
概括
使用与倾向分数 (PS) 匹配的历史对照 (HC) 可以减少患者在子宫内膜异位症相关的骨盆疼痛 (EAPP) 研究中的招募. 这种方法,当仔细应用时,可以复制原始研究结果并提高可比性.
科学领域:
- 药理学 药理学是指药理学的学科.
- 临床试验 临床试验
- 疼痛管理 疼痛管理
背景情况:
- 历史对照 (HC) 提供了一种减少临床试验患者招募的策略.
- 在子宫内膜异位症相关的骨盆疼痛 (EAPP) 研究中HC的适用性需要仔细评估,因为可能存在偏差.
研究的目的:
- 评估在EAPP研究中使用历史对照 (HC) 臂引入的潜在偏差.
- 通过HC和倾向分数 (PS) 匹配来评估dienogest在治疗EAPP中的疗效.
主要方法:
- 重新评估了使用dienogest对EAPP进行随机,安慰剂对照试验的数据.
- 倾向性得分 (PS) 匹配用于根据基线变量对准治疗和HC组.
- 分析了有效性参数,使用完整和匹配的对照组来评估偏差.
主要成果:
- 治疗和聚合HC之间的疼痛减轻 (视觉模拟量表) 的差异与研究中的对照组有所不同.
- 在PS匹配后,平均值的差异与研究中的对照组的结果非常接近.
- PS匹配成功地重现了原始研究的主要终点结果.
结论:
- 具有倾向分数 (PS) 匹配的历史控制 (HC) 对EAPP研究有价值,只要仔细考虑选择机制和假设.
- 尽管研究设计有相似之处,但研究之间存在异质性和差异.
- PS匹配显著提高了HC手臂与实验中的对照组的可比性,使原始研究结果的重现成为可能.
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