瘤学患者偏好试验:范围审查和报告建议
Denice Kamp1, Rob Kessels1, Nadia Haj Mohammad2
1Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands.
Journal of clinical epidemiology
|May 25, 2025
概括
在瘤学中,患者偏好试验 (PPT) 为随机对照试验 (RCT) 提供了一个替代方案,当患者偏好影响参与时. 本综述强调了针对瘤学研究的PPT设计和报告方面的挑战.
科学领域:
- 在瘤学瘤学.
- 临床试验 临床试验
- 生物统计学 生物统计学
背景情况:
- 随机对照试验 (RCT) 面临的挑战是患者偏好影响可行性,概括性和有效性.
- 患者偏好试验 (PPT),将患者偏好纳入手臂分配,被提议作为一种替代设计.
- 本综述侧重于PPT在瘤学研究中的应用和报告,特别是对于干预有效性研究.
研究的目的:
- 在瘤学研究中提供患者偏好试验 (PPT) 设计的概述.
- 分析PPT在评估干预有效性的应用.
- 为瘤学中PPT报告提供建议.
主要方法:
- 对使用PPT设计的前性瘤学研究进行了范围审查.
- 如果根据患者的偏好来分配至少一组患者的试验组,则将研究纳入.
- 提取的数据包括研究目标,设计选择的理由,共变量调整和样本大小计算.
主要成果:
- 确定了44个PPT,其中34个专注于干预的有效性.
- 选择PPT设计的主要原因是RCT的不可行性 (50%的研究).
- 在53%的研究中进行了基线共变量调整,而50%的已完成试验报告了未预期的不平等分配比率.
结论:
- 瘤学患者偏好试验存在几个设计和报告挑战.
- 未来的PPT必须解决因优先分配而导致的混杂和不平等的组大小的潜在偏差.
- 仔细考虑和报告这些因素对于PPT的有效性和统计能力至关重要.
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