在囊性纤维化中,儿童肺部恶化病的流行病学正在发生变化
Yaron Fireizen1, Mohamoud Ahmed2, Timothy Vigers3,4
1Department of Pediatrics, Rady Children's Hospital, University of California San Diego, San Diego, California, USA.
Pediatric pulmonology
|February 28, 2025
概括
在引入elexacaftor/tezacaftor/ivacaftor (ETI) 治疗后,因囊性纤维化 (CF) 肺部恶化而住院的病例显著减少. 该研究观察到患者人口统计和微生物学的变化,表明治疗格局的变化和持续的健康差异.
科学领域:
- 儿科肺病学 儿科肺病学
- 囊性纤维化转膜导电调节器 (CFTR) 调节器疗法
- 在CF的传染病流行病学.
背景情况:
- 埃莱克萨卡夫托/特萨卡夫托/伊瓦卡夫托 (ETI) 是一种高效的CFTR调节器疗法.
- 在年轻人群中引入ETI和COVID-19大流行影响了CF护理.
- 在患有CF的儿童中,观察到需要住院治疗的肺部恶化显著减少.
研究的目的:
- 评估住院儿科和年轻成人CF患者的人口和临床特征.
- 为了比较这些特征,在批准儿科ETI之前和之后.
- 分析住院率和患者个人资料的变化.
主要方法:
- 五个美国CF基金会认可的护理中心的回顾性图表审查.
- 2018年和2022年对患有CF的儿童和年轻人住院数据的分析.
- 两年间患者人口统计,临床特征和微生物学数据的比较.
主要成果:
- 住院病例从2018年的471例减少到2022年的163例.
- 2022年,更多的住院患者将自己认定为有色人种 (28%对14%).
- 2022年住院儿童的比例更高,有两个非F508del突变,并减少了与Pseudomonas aeruginosa等关键病原体的感染.
结论:
- 住院病例的减少很可能归因于ETI治疗的好处.
- 在2022年住院患者中,更高比例的人没有资格接受ETI,这表明治疗缺口.
- 在种族人口统计和微生物学上观察到的变化凸显了持续的健康差异和不断变化的CF护理挑战.
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