CFTR modulators may offer early mental health benefits for children with cystic fibrosis and their parents.
CFTR MODULATORS may offer early mental health benefits for children with cystic fibrosis and their parents, according to prospective observational findings that point to lower anxiety scores in treated children and lower anxiety and depression scores among parents.
The study evaluated children with cystic fibrosis who were receiving cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy and compared them with children not receiving modulator therapy. Although CFTR modulators have been shown to improve clinical symptoms in cystic fibrosis, their potential psychosocial effects remain less clearly defined.
Anxiety Scores Lower With CFTR Modulators
Investigators enrolled 53 children with cystic fibrosis, including 24 using modulator therapy and 29 not using modulator therapy, along with their parents. There were no significant demographic differences between the groups, supporting comparison of mental health and quality of life measures across treatment status.
Children completed the Cystic Fibrosis Quality of Life Questionnaire Revised, Children’s Depression Inventory, and Screen for Child Anxiety Related Emotional Disorders. Parents completed the Beck Depression Inventory, Beck Anxiety Inventory, and World Health Organization Quality of Life Scale Short Form.
Children who were not receiving CFTR modulators had significantly higher anxiety scores on the Screen for Child Anxiety Related Emotional Disorders than those receiving therapy. The finding suggests that modulator use may be associated with reduced anxiety burden early in treatment, although the observational design means causality cannot be established.
Parent Mental Health Also Shows Improvement
The psychosocial signal extended to caregivers. Parents of children who were not using CFTR modulators had significantly higher depression and anxiety scores on the Beck Depression Inventory and Beck Anxiety Inventory compared with parents of children receiving modulator therapy.
Quality of life scores among parents were higher in the modulator therapy group, but this difference did not reach statistical significance. Together, the results suggest that the early benefits of CFTR modulators may extend beyond clinical symptoms, potentially reducing emotional strain for both children and caregivers.
The findings underscore the need for continued mental health screening in cystic fibrosis care, even as disease modifying therapy improves clinical outcomes. Larger longitudinal studies are needed to determine whether CFTR modulators directly improve anxiety, depression, and quality of life over time.
Reference
Yetişgin H et al. Early effect of CFTR modulators on the mental health of patients with cystic fibrosis and parents. Eur J Pediatr. 2026;185:464.
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