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Changing Epidemiology of Pediatric Pulmonary Exacerbations in Cystic Fibrosis

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
PubMed

Insights

Hospitalizations for cystic fibrosis (CF) pulmonary exacerbations significantly decreased after the introduction of elexacaftor/tezacaftor/ivacaftor (ETI) therapy. The study observed shifts in patient demographics and microbiology, indicating evolving treatment landscapes and persistent health disparities.

Area of Science:

  • Pediatric pulmonology
  • Cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies
  • Infectious disease epidemiology in CF

Background:

  • Elexacaftor/tezacaftor/ivacaftor (ETI) is a highly effective CFTR modulator therapy.
  • Introduction of ETI to younger populations and the COVID-19 pandemic impacted CF care.
  • Significant reductions in pulmonary exacerbations requiring hospitalization were observed in children with CF.

Purpose of the Study:

  • To evaluate demographic and clinical characteristics of hospitalized pediatric and young adult CF patients.
  • To compare these characteristics before and after the approval of pediatric ETI.
  • To analyze changes in hospitalization rates and patient profiles.

Main Methods:

  • Retrospective chart review across five US CF Foundation-accredited care centers.
  • Analysis of hospitalization data for children and young adults with CF in 2018 and 2022.
  • Comparison of patient demographics, clinical features, and microbiological data between the two years.

Main Results:

  • Hospitalizations decreased from 471 cases in 2018 to 163 cases in 2022.
  • Increased proportion of hospitalized patients identifying as people of color in 2022 (28% vs. 14%).
  • Higher percentage of hospitalized children in 2022 had two non-F508del mutations and reduced infections with key pathogens like Pseudomonas aeruginosa.

Conclusions:

  • The decline in hospitalizations is likely attributed to ETI therapy benefits.
  • A higher proportion of hospitalized patients in 2022 were not eligible for ETI, indicating treatment gaps.
  • Observed shifts in racial demographics and microbiology highlight ongoing health disparities and evolving CF care challenges.
Abstract

Keywords:
airway infectionchildrenepidemiologyhealth disparitiespulmonary exacerbations

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