Fireizen Yaron, Ahmed Mohamoud, Vigers Timothy, Akong Kathryn, Ryu Julie, Hahn Andrea, Fanous Hani, Koumbourlis Anastassios, Tirakitsoontorn Pornchai, Arrieta Antonio, Burgener Elizabeth B, Koff Jonathan, Cogen Jonathan D, Bouzek Drake C, Hanley Elin, Keck Allison, Stout Dayna, Bradley John, Sagel Scott D
Department of Pediatrics, Rady Children's Hospital, University of California San Diego, San Diego, California, USA.
University of Colorado School of Medicine, Aurora, Colorado, USA.
Pediatr Pulmonol. 2025 Mar;60(3):e71019. doi: 10.1002/ppul.71019.
The introduction of elexacaftor/tezacaftor/ivacaftor (ETI), a highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy, to younger ages and the COVID-19 pandemic have significantly reduced pulmonary exacerbations requiring hospitalization among children with CF.
To assess demographic and clinical characteristics of children and young adults with CF hospitalized for pulmonary exacerbations before and after pediatric ETI approval.
A retrospective chart review was conducted at five United States CF Foundation-accredited care centers. Hospitalization data from children and young adults with CF in 2018 and 2022 were analyzed.
Hospitalizations decreased from 471 cases (241 individuals) in 2018 to 163 cases (110 individuals) in 2022. The racial distribution shifted, with more hospitalized patients identifying as people of color in 2022 (28% vs. 14%; p = 0.018). A greater proportion of hospitalized children in 2022 had two non-F508del mutations compared with children hospitalized in 2018 (38% vs. 19%) and were less likely to be infected with methicillin-resistant Staphylococcus aureus (MRSA). Comparing 2022-2018, children on CFTR modulator therapy, including ETI (76%), showed reduced infections with Pseudomonas aeruginosa and Achromobacter xylosoxidans.
The decline in hospitalizations for pulmonary exacerbations likely reflects the benefits of ETI therapy, as a higher proportion of children and young adults hospitalized in 2022 had two non-F508del mutations and were not eligible for ETI. A greater percentage of those hospitalized in 2022 identified as belonging to minority racial groups, highlighting ongoing health disparities in the ETI era. Additionally, there were notable changes in the microbiological characteristics between 2018 and 2022.
将艾列卡福托/替扎卡福托/依伐卡托（ETI）这种高效的囊性纤维化跨膜传导调节因子（CFTR）调节剂疗法应用于更年轻的患者，以及新冠疫情，显著减少了囊性纤维化（CF）患儿中需要住院治疗的肺部加重发作。
评估小儿ETI获批前后因肺部加重发作住院的CF儿童和青年的人口统计学和临床特征。
在美国五个经囊性纤维化基金会认可的护理中心进行了一项回顾性病历审查。分析了2018年和2022年CF儿童和青年的住院数据。
住院病例从2018年的471例（241人）降至2022年的163例（110人）。种族分布发生了变化，2022年更多住院患者为有色人种（28%对14%；p = 0.018）。与2018年住院儿童相比，2022年住院儿童中具有两个非F508del突变的比例更高（38%对19%），且感染耐甲氧西林金黄色葡萄球菌（MRSA）的可能性更小。与2018年相比，2022年接受CFTR调节剂治疗（包括ETI，76%）的儿童感染铜绿假单胞菌和木糖氧化无色杆菌的情况减少。
肺部加重发作住院人数的下降可能反映了ETI疗法的益处，因为2022年住院的儿童和青年中，有更高比例具有两个非F508del突变且不符合ETI治疗条件。2022年住院患者中更大比例属于少数种族群体，这凸显了ETI时代持续存在健康差异。此外，2018年至2022年微生物特征发生了显著变化。
