复杂的Mycobacterium avium肺病的核心结果领域:一个MACCOR研究
Cara D Varley1,2, Sarah A R Siegel2, Naomi A DeBacker2
1Oregon Health and Science University, School of Medicine, Portland, OR, USA.
ERJ open research
|December 24, 2025
概括
研究人员确定了Mycobacterium avium复杂肺病 (MAC-PD) 临床试验的核心结果领域. 这种共识将改善这种慢性肺部疾病的研究和治疗评估.
科学领域:
- 肺部医学 肺部医学
- 临床试验设计 临床试验设计
- 患者报告的结果
背景情况:
- 飞菌复杂肺病 (MAC-PD) 是一种具有重大患者影响的慢性炎症状况.
- 在MAC-PD试验中,目前的结果测量通常不足以反映疾病和治疗效应.
- 在MAC-PD研究中需要标准化结果领域.
研究的目的:
- 为MAC-PD临床试验建立一套基于国际共识的核心结果领域 (COD).
- 制定一个评估MAC-PD的框架,更好地反映患者的经验和疾病负担.
- 为未来的研究指导,开发适合MAC-PD的测量仪器.
主要方法:
- 采用了经过修改的Delphi共识方法.
- 包括患者,临床医生和研究人员在内的国际利益相关者参与了电子Delphi过程.
- 参与者评价了初步结果领域对于达成共识的重要性.
主要成果:
- 来自17个国家的306名参与者参与了共识进程.
- 在经过多轮协商一致后,九个核心结果领域符合纳入标准.
- 包括的领域包括症状,微生物学,治疗副作用,成像,身体功能,治疗负担,活力,复发和生物标志物.
结论:
- 确定的核心结果领域为MAC-PD临床试验设计提供了基础.
- 这些领域将促进对疾病活动的复合措施的开发.
- 这些COD的传播将有助于优先考虑MAC-PD测量仪器的研究.
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