专家德尔菲共识关于代用终点治疗评估在代谢功能障碍相关的型肝炎
Arun J Sanyal1, Atsushi Nakajima2, Elisabetta Bugianesi3
1Virginia Commonwealth University, Richmond, Virginia.
概括
专家们就评估代谢功能障碍相关的脂肪肝炎 (MASH) 治疗的关键措施达成共识. 组织学终点是有利于监管批准,而非侵入性测试,如肝硬度测量 (LSM),是首选的临床实践和疾病监测.
科学领域:
- 肝病学 肝病学是一种肝病学.
- 胃肠病学 胃肠病学
- 临床试验设计 临床试验设计
背景情况:
- 与代谢功能障碍相关的脂肪肝炎 (MASH) 是一个日益严重的全球健康挑战.
- 需要有效的MASH疗法需要标准化代用终点进行评估.
- 目前关于监管批准,临床实践和报销的最佳终点的共识有限.
研究的目的:
- 建立专家共识,以确定MASH治疗评估的适当代用终点.
- 在监管,报销和临床环境中确定MASH适合的替代结果措施.
主要方法:
- 一个经过修改的Delphi过程,涉及23名国际肝病学和胃肠病学专家.
- 两轮结构化调查由有针对性的文献综述提供信息.
- 共识定义为≥75%的同意,根据CREDES指南报告.
主要成果:
- 组织学终点 (纤维化改善或MASH解决) 被认为适合监管/补偿设置.
- 在临床实践中,非侵入性测试 (NIT) 是MASH监测的首选方法.
- 共识支持通过VCTE或MRE通过肝硬度测量 (LSM) 减少≥30%,作为有意义的治疗应答指标,支持在实践中替换肝活检.
结论:
- 基于组织学和NIT的测量对于MASH治疗评估至关重要.
- 支持转向使用NIT,特别是通过VCTE或MRE通过LSM进行常规MASH监测.
- 这些发现对MASH临床试验设计,补偿政策和临床指南有影响.
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