在CF亚群中肺部恶化风险的差异
D R VanDevanter1, C O'Rourke2, M W Konstan3
1Case Western Reserve University School of Medicine, Cleveland, OH, USA.
概括
在患有囊性纤维化 (pwCF) 患者中,以前的肺恶化 (PEx) 强烈预测了未来的PEx风险,无论调节剂治疗如何. 即使使用elexacaftor/tezacaftor/ivacaftor (ETI),更高的先前PEx也表明未来风险更大.
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.
- 流行病学 流行病学
背景情况:
- 肺部恶化 (PEx) 是囊性纤维化 (CF) 管理中的一个关键问题.
- 评估未来的PEx风险对于治疗策略至关重要.
- 较新的CFTR调节器疗法,如elexacaftor/tezacaftor/ivacaftor (ETI),对PEx风险的影响需要进一步调查.
研究的目的:
- 评估前一年PEx频率与CF患者 (pwCF) 未来PEx风险之间的关联.
- 为了确定这种关联是否在接受ETI的pwCF中持续存在.
- 为新的CF疗法进行临床试验的设计提供信息.
主要方法:
- 来自CF基金会患者登记处 (CFFPR) 对12岁以上 (2022-2023) 的pwCF数据的分析.
- 2022年PEx频率的分层 (零,一,二,三+).
- 基于2022年PEx频率,疾病阶段,人口统计,感染状况和调节器使用 (没有与ETI) 的2023年PEx危险的建模.
主要成果:
- 更高的2022年PEx频率与增加的2023年PEx平均值和较短的2023年PEx的时间密切相关.
- 与没有PEx的PwCF相比,在2022年具有3+PEx的PwCF在2023年具有7.11倍的PEx危险.
- 普华永道在2022年有一两次PEx的PwCF在2023年有2.89倍的PEx风险.
结论:
- 前一年的PEx频率是pwCF中未来PEx风险的重要独立预测因素.
- ETI 降低了 PEx 率,但具有较高 PEx 史的人仍然面临高风险.
- 未来的治疗干预可以研究在ETI治疗的pwCF中,有频繁PEx的病史.
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