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Respiratory Muscle Function in Children and Adolescents with Cystic Fibrosis in the Era of CFTR Modulator Therapies

Guillermo García-Pérez-de-Sevilla1, Ángela Blanco Velasco2, Thomas Yvert2

  • 1Department of Physiotherapy, Faculty of Medicine, Health and Sports, European University of Madrid, 28670 Madrid, Spain.

Children (Basel, Switzerland)
|July 29, 2025
PubMed

Insights

Children with cystic fibrosis (CF) on Elexacaftor/Tezacaftor/Ivacaftor (ETI) show comparable respiratory muscle strength to healthy peers. This suggests CFTR modulators may positively impact lung function in young CF patients.

Area of Science:

  • Pediatric Pulmonology
  • Medical Genetics
  • Respiratory Medicine

Background:

  • Cystic Fibrosis (CF) is a genetic disorder affecting multiple organs, primarily the lungs.
  • CFTR modulators, like Elexacaftor/Tezacaftor/Ivacaftor (ETI), represent a significant advancement in CF treatment.
  • The impact of ETI on respiratory muscle function in pediatric populations requires further investigation.

Purpose of the Study:

  • To evaluate respiratory muscle strength (maximal inspiratory and expiratory pressures) in children and adolescents with CF treated with ETI.
  • To compare respiratory muscle function between CF patients on ETI and healthy controls.
  • To explore potential correlations between ETI treatment and improvements in respiratory muscle strength.

Main Methods:

  • A descriptive, observational, cross-sectional study involving 24 children/adolescents with CF (aged 6-18) on ETI and 24 healthy controls.
  • Assessment of lung function, maximal inspiratory pressure (MIP), maximal expiratory pressure (MEP), diet quality (KIDMED), and physical activity (PAQ).
  • Statistical analysis using student's t-test or Mann-Whitney U-test, with significance set at p < 0.05.

Main Results:

  • No significant differences were observed in maximal expiratory pressure (MEPmax, p=0.440) or maximal inspiratory pressure (MIPmax, p=0.206) between CF patients on ETI and healthy controls.
  • CF patients exhibited lower diet quality (KIDMED, p=0.022) and physical activity (PAQ, p=0.010) scores compared to controls.
  • Observed MIP and MEP values in CF participants were higher than historical data from before ETI introduction.

Conclusions:

  • Children and adolescents with CF treated with ETI demonstrate respiratory muscle strength comparable to healthy individuals.
  • While lifestyle factors (diet, physical activity) differed, ETI may contribute positively to respiratory muscle function in this cohort.
  • Further longitudinal and controlled studies are warranted to confirm the long-term effects of ETI on respiratory muscle strength.

Keywords:
CFTR modulatorscystic fibrosismaximal expiratory pressuremaximal inspiratory pressure

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