在乳病中标准化随机对照试验:一项国际多学科适当性研究
Benjamin Lebwohl1, Christopher Ma2, Stephen M Lagana3
1Celiac Disease Center, Department of Medicine, Columbia University College of Physicians and Surgeons, New York, New York; Department of Epidemiology, Mailman School of Public Health, Columbia University, New York, New York.
Gastroenterology
|September 13, 2023
概括
开发新的乳病 (CeD) 治疗方法需要标准化的临床试验设计. 本研究为CeD随机对照试验 (RCT) 提供了共识建议,重点关注资格和结果措施.
科学领域:
- 胃肠病学 胃肠病学
- 临床试验设计 临床试验设计
- 结核病研究 结核病研究
背景情况:
- 需要安全有效的药理疗法治疗腹腔疾病 (CeD).
- 关于CeD随机对照试验 (RCT) 的最佳设计存在缺乏共识.
- 标准化方法对于推进CeD治疗开发至关重要.
研究的目的:
- 建立关于病RCT适当设计和配置的共识.
- 为 CeD 临床试验的关键方面提供标准化建议.
- 引导未来对CeD药理疗法的研究.
主要方法:
- 采用了两轮修改的研究和开发/加利福尼亚大学洛杉矶大学适当性方法研究.
- 十八位专家胃肠道学家和胃肠道病理学家对关于CeD RCT的陈述进行了投票.
- 评估了两种不同的RCT设计:包含质挑战的设计和评估组织学变化逆转的设计.
主要成果:
- 制定了参与者资格标准的建议,包括饮食遵守和基线组织学.
- 提供了具体的指导,用于进行质挑战 (例如,无FODMAP) 和选择结果措施.
- 组织学和患者报告的结局都被认为适合在CeD试验中评估治疗疗效.
结论:
- 已经制定了CeD临床试验设计,资格和结果措施的标准化建议.
- 这些建议旨在提高未来乳病RCT的一致性和严格性.
- 该共识为评估CeD新型药理干预措施提供了一个框架.
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