在严重α1抗素缺乏症 (RAPID) 中静脉增强治疗和肺密度:一个随机,双盲,安慰剂控制的随机试验
Kenneth R Chapman1, Jonathan G W Burdon2, Eeva Piitulainen3
1Asthma and Airway Centre, University Health Network, Toronto Western Hospital, and Division of Respiratory Medicine, Department of Medicine, University of Toronto, Toronto, ON, Canada.
Lancet (London, England)
|June 1, 2015
概括
阿尔法1蛋白酶抑制剂 (A1PI) 增强治疗减缓了阿尔法1抗素缺乏症患者的肺气进展. 这一发现基于CT肺密度和肺总容量,支持A1PI治疗,以保护肺组织.
科学领域:
- 肺部病理学 肺部病理学
- 放射学 放射学是指放射学
- 药理学 药理学是指药理学的学科.
背景情况:
- 阿尔法1抗素缺乏症 (AATD) 是一种导致肺气的遗传性疾病.
- 没有随机的安慰剂对照试验证明A1PI增强疗效.
- 通过CT测量肺密度是AATD肺瘤进展的敏感标志物.
研究的目的:
- 在AATD中评估α1蛋白酶抑制剂 (A1PI) 增强疗法的疗效.
- 用CT评估的肺密度作为主要结果指标.
- 进行一项严格的,随机的,安慰剂对照试验 (RAPID研究).
主要方法:
- 一个多中心,双盲,随机,安慰剂对照试验,涉及180名严重AATD患者.
- 患者在24个月内接受静脉A1PI (60毫克/千克/周) 或安慰剂.
- 主要终点:CT肺密度变化总肺容量 (TLC) 和功能残留容量 (FRC) 组合和单独.
主要成果:
- 在结合TLC和FRC测量时,A1PI治疗没有显著改变肺密度损失 (p=0.06).
- 然而,A1PI显著降低了仅在TLC的肺密度损失率 (差异为每年0.74 g/L,p=0.03).
- 在A1PI和安慰剂组之间,不良事件是相似的;A1PI被很好地容忍.
结论:
- 单独在TLC进行CT肺密度测量表明,A1PI增强疗法减缓了AATD中肺的进展.
- 这一发现支持考虑A1PI增强,以保护AATD相关的肺瘤中的肺瘤.
- 单独FRC或组合测量的肺密度没有显示显著差异.
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