A Turkish study indicates that CFTR modulator treatments could offer benefits beyond lung function, including reductions in anxiety and depression among children with cystic fibrosis and their parents, highlighting the need for further research into psychological impacts.
Continued treatment with CFTR modulators may do more than improve lung function in children with cystic fibrosis, according to a Turkish study published in the European Journal of Pediatrics. Researchers reported lower anxiety and depression scores in children taking the drugs, alongside better breathing measures, improved growth and signs of reduced strain on parents.
CFTR modulators work by targeting the faulty protein behind cystic fibrosis, a genetic condition that causes thick mucus to build up in the lungs and other organs. The Turkish team followed 24 children with the disease and their parents over 18 months to see whether ongoing treatment changed mental health, sleep and quality of life. Most of the children were taking Trikafta, while smaller numbers were using Kalydeco, Symdeko or Orkambi.
By the end of the study, children’s scores for anxiety and depression had fallen, and daytime sleepiness also eased. Spirometry results improved in the children who completed the breathing test, and median body mass index rose from 15.2 to 16.9, suggesting better nutritional status. Some quality-of-life measures improved as well, although not all remained statistically significant after adjustment for multiple testing.
Parents also appeared to benefit. The study found lower anxiety and depression scores among caregivers, along with less reported burden and better overall quality of life. The findings echo an earlier Turkish study and fit with editorial commentary in the Journal of Cystic Fibrosis calling for more research into the psychological effects of CFTR modulators for both patients and families.
The authors cautioned that the study was small and had no comparison group, so it cannot prove the medicines caused the changes. Even so, they said the results suggest CFTR modulators may offer benefits beyond physical symptoms and support the need for larger studies with control groups to better understand their wider impact.
Disclaimer: This content is for informational purposes only and is not intended to be a substitute for professional medical judgment, advice, diagnosis, or treatment.
