伊瓦卡夫托开始与肺功能变异之间的现实世界关联:一个注册研究.
Rhonda D Szczesniak1, Eleni-Rosalina Andrinopoulou2, Hancheng Li1
1Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA; University of Cincinnati, Cincinnati, OH, USA.
伊瓦卡夫托尔治疗显著减少了囊性纤维化 (CF) 患者的肺功能变异性. 这种强迫呼气体积在1秒内减少预测的百分比 (FEV1pp) 变化可能表明治疗的有效性.
科学领域:
- 肺部医学 肺部医学
- 药理学 药理学是指药理学的学科.
- 遗传学 是一个遗传学.
背景情况:
- 增加的肺功能变异性,特别是预测的1秒内强迫呼气体积的1% (FEV1pp),与囊性纤维化 (CF) 的更快下降有关.
- 伊瓦卡夫托对FEV1pp变异性的影响以前没有被描述.
研究的目的:
- 为了研究 ivacaftor 启动和FEV1pp 变异性在患有CF的个体之间的联系.
- 为了确定与FEV1pp变异性增加相关的风险因素.
主要方法:
- 对囊性纤维化基金会患者登记 (2008-2020) 数据的分析.
- 利用线性混合效应模型来比较前和后债务或FEV1pp变化,使用最大/中位数偏差和趋势线标准偏差等措施.
- 包括527个人在分析队列中.
主要成果:
- 开始Ivacaftor与FEV1pp变化率的降低有关 (中位数降低:预测1.85%).
- 从最佳FEV1pp最大偏差使用最明显的减少,而趋势线措施,特别是中位偏差,显示了一致的发现.
- 儿科和成人患者之间变化的风险因素有所不同,但在G551D和R117H亚组中一致. F508del同卵性患者显示的变化很小.
结论:
- 有证据表明,ivacaftor的启动有效地减少了CF患者的FEV1pp变化.
- 测量FEV1pp变异性可以作为评估ivacaftor治疗疗效的有价值标记.
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