测量患者偏好以告知临床试验设计:风湿性关节炎的一个例子
Gillian R Currie1, Jan Storek2, Karen V MacDonald3
1Department of Community Health Sciences, University of Calgary, Calgary, AB, Canada. currie@ucalgary.ca.
The patient
|December 12, 2024
概括
患有类风湿性关节炎 (RA) 的患者愿意承担重大风险,包括增加死亡或慢性移植对宿主疾病 (cGVHD) 的风险,以阻止异构骨髓移植 (BMT) 的疾病进展. 这表明,在RA中BMT的临床试验可以成功地招募参与者.
科学领域:
- 类风湿病学 类风湿病学
- 移植免疫学 移植免疫学
- 临床试验设计 临床试验设计
背景情况:
- 全基性骨髓移植 (BMT) 提供了对类风湿性关节炎 (RA) 的潜在治疗方法,但存在重大风险,包括死亡率.
- 患者愿意接受BMT风险和RA治疗的好处仍然不确定,影响临床试验的可行性.
研究的目的:
- 量化患者的风险耐受性和益处风险权衡,用于类基骨髓移植 (BMT) 在类风湿性关节炎 (RA).
- 通过了解患者的偏好,为在RA中BMT进行以患者为中心的临床试验的设计提供信息.
主要方法:
- 采用了离散选择实验 (DCE),为患者提供了涉及不同程度疾病进展,死亡风险和慢性移植对宿主疾病 (cGVHD) 的选择.
- 参与者从炎症性关节炎登记册中招募,并使用逻辑模型分析选择数据,以评估风险-收益偏好.
主要成果:
- 患者表现出愿意接受增加的风险 (例如,3%更高的死亡率,6%更高的cGVHD) 以增加阻止疾病进展的机会.
- 对于临床相关的BMT情景,显著多数患者 (72-91%) 表示他们会选择BMT,特别是那些患有严重,治疗不良的RA患者.
结论:
- 患有RA的患者愿意承担巨大的风险,因为他们有可能通过BMT实现疾病缓解.
- 偏好研究对于设计以患者为中心的临床试验至关重要,支持 RA 试点 BMT 试验的潜在成功.
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