系统性硬化症患者报告的结果:对生活质量和疾病负担的见解
Alain Lescoat1,2,3, Yen T Chen3,4, Dinesh Khanna3
1Department of Internal Medicine and Clinical Immunology, Rennes University Hospital.
患者报告的结果测量 (PROM) 对于评估系统性硬化症 (SSc) 治疗至关重要. 新的以患者为中心的倡议和综合指数旨在改善在临床试验中评估治疗疗效的方式.
科学领域:
- 类风湿病学 类风湿病学
- 临床试验 临床试验
- 患者报告的结果
背景情况:
- 监管机构要求对患者的"感觉和功能"进行评估,以获得系统性硬化症 (SSc) 治疗批准.
- 患者报告的结果测量 (PROM) 对于在随机对照试验 (RCT) 中评估治疗疗效至关重要.
研究的目的:
- 审查SScRCT中使用的可用PROM.
- 突出SSc-PROMs最近的发展和正在进行的举措.
- 为未来在SSc研究中使用PROM提供考虑.
主要方法:
- 对SSc中的PROMs现有文献的叙述性综述.
- 专注于最近开发的PROM和以患者为中心的倡议.
- 分析正在进行的项目和SSc-PROMs的未来方向.
主要成果:
- 一些倡议 (ASRAP,Mawdsley Calcinosis问卷,COAST,CRISTAL) 让患者伙伴参与开发新的PROM.
- 这些举措强调了以患者为中心的结果评估方法.
- 最近开发的PROM和正在进行的倡议正在扩大SSc研究中患者报告数据的范围.
结论:
- 未来的SSc试验可以从组合响应指数中获益,将PROM作为主要结果.
- CRISS指数是扩散皮肤SSc的一个例子,整合了医生和患者的观点.
- 以患者为中心的PROM的开发和利用是准确评估SSc治疗疗效的关键.
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