急性严重性结肠炎:关于临床试验设计和终点的国际德尔菲共识
Sailish Honap1, Vipul Jairath2, Bruce E Sands3
1Department of Gastroenterology, INFINY Institute, CHRU Nancy, Nancy, France; Department of Gastroenterology, St George's University Hospitals NHS Foundation Trust, London, United Kingdom; School of Immunology and Microbial Sciences, King's College London, London, United Kingdom.
这一共识为设计急性严重性结肠炎 (ASUC) 临床试验提供了明确的指导方针. 它涉及诸如资格标准和终点等关键领域,以改善新疗法的研究.
科学领域:
- 胃肠病学 胃肠病学
- 临床试验设计 临床试验设计
- 炎症性肠道疾病研究研究
背景情况:
- 针对急性严重性结肠炎 (ASUC) 的临床试验缺乏标准化的设计,阻碍了新治疗方法的开发.
- 试验设计中的异质性阻碍了研究努力,以确定ASUC的有效疗法.
研究的目的:
- 在ASUC试验设计的关键方面建立第一个共识.
- 为优化ASUC的干预性临床试验提供明确的立场陈述.
主要方法:
- 使用了经过修改的Delphi共识方法.
- 一个由20名国际专家组成的ASUC试验设计小组参加了会议.
- 共识被定义为参与者之间至少75%的协议.
主要成果:
- 30个声明达到了共识,并被专家小组批准.
- 在资格标准上达成共识,包括疾病范围,严重程度和先前治疗.
- 优化试验设计元素,如随机化,分层化,皮质类固醇处理和评估时间被商定.
- 制定了对初级和二级终点的建议,包括临床和内镜反应/缓解,治疗失败和生活质量.
结论:
- 最近治疗性结肠炎的进展,包括Janus激酶抑制剂,重新引起了对ASUC疗法的兴趣.
- 在高风险ASUC群体中进行临床试验具有挑战性.
- 这种共识为指导未来ASUC临床试验和推进药物开发提供了关键的框架.
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