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Forskolin-induced Swelling in Intestinal Organoids: An In Vitro Assay for Assessing Drug Response in Cystic Fibrosis Patients
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Cystic Fibrosis and CFTR Modulators: The Impact on Bone Density, Muscle Mass and Strength in Children and Young

Katerina Iordanidou1,2, Nikolaos D Karakousis1, Elpis Hatziagorou2

  • 1Department of Nutrition and Dietetics, School of Physical Education, Sport Science and Dietetics, University of Thessaly, 42100 Trikala, Greece.

Children (Basel, Switzerland)
|November 27, 2025
PubMed

Insights

Cystic fibrosis (CF) treatments called CFTR modulators may improve bone density in children. Their effects on muscle mass and strength require further investigation, highlighting the need for more research in CF care.

Area of Science:

  • Pediatric Health
  • Genetic Disorders
  • Pulmonology

Background:

  • Cystic fibrosis (CF) is a genetic disorder caused by CFTR gene mutations.
  • These mutations impair CFTR protein function, leading to multi-organ complications, primarily affecting the lungs.
  • Understanding CF's impact on non-pulmonary aspects is crucial for comprehensive patient care.

Purpose of the Study:

  • To investigate the impact of CFTR modulators on bone density, muscle mass, and strength in pediatric CF patients.
  • To synthesize current evidence regarding these effects through a narrative review.
  • To identify gaps in knowledge and areas for future research in CF management.

Main Methods:

  • Conducted a narrative (non-systematic) review of existing literature.
  • Focused on studies involving children and young adolescents with CF.
  • Analyzed data on the effects of CFTR modulators on bone mineral density, muscle mass, and strength.

Main Results:

  • CFTR modulators show potential for positive influence on bone mineral density in pediatric CF.
  • Evidence regarding the impact of CFTR modulators on muscle mass and strength is variable and requires further study.
  • Current findings suggest a nuanced effect of CFTR modulators on musculoskeletal health.

Conclusions:

  • Further research is necessary to validate the observed effects of CFTR modulators on bone density.
  • Additional studies are needed to clarify the inconsistent findings on muscle mass and strength.
  • Comprehensive assessment of CFTR modulators' long-term impact on overall health in CF patients is warranted.

Keywords:
bone densitycystic fibrosisdual energy X-ray absorptiometryivacaftormuscle mass

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