CFTR modulator use was associated with lower anxiety scores in children with cystic fibrosis and lower anxiety and depression scores among their parents, though causal conclusions cannot be drawn from this observational study.
Key Findings
Results
Children with cystic fibrosis who did not use CFTR modulator therapy had significantly higher anxiety scores than those who did use modulator therapy.
Anxiety was measured using the Screen for Child Anxiety Related Emotional Disorders (SCARED) scale
SCARED scores were significantly higher in patients who did not use modulator therapy (p = 0.016)
Group 1 (modulator users) included n = 24 children; Group 2 (non-users) included n = 29 children
No significant difference in depression scores (Children's Depression Inventory) between groups was reported
Results
Parents of children with cystic fibrosis who did not use CFTR modulator therapy had significantly higher depression scores than parents of children who did use modulator therapy.
Depression was measured using the Beck Depression Inventory (BDI)
BDI scores were statistically significantly higher in parents of patients who did not use modulator therapy (p = 0.006)
Total parent sample was n = 53
The study design was prospective and observational
Results
Parents of children with cystic fibrosis who did not use CFTR modulator therapy had significantly higher anxiety scores than parents of children who did use modulator therapy.
Anxiety was measured using the Beck Anxiety Inventory (BAI)
BAI scores were statistically significantly higher in parents of patients who did not use modulator therapy (p = 0.002)
This finding was more statistically robust than the depression finding (p = 0.002 vs p = 0.006)
Results
Quality of life scores were higher among parents of patients who used CFTR modulator therapy, but the difference did not reach statistical significance.
Quality of life was measured using the World Health Organization Quality of Life Scale-Short Form (WHOQOL-BREF)
WHOQOL-BREF scores were higher among parents of patients who used modulator therapy, but the difference was not statistically significant (p = 0.060)
The result approached but did not meet the conventional threshold for statistical significance
Results
There were no significant demographic differences between the CFTR modulator-using group and the non-using group of children with cystic fibrosis.
Group 1 (modulator users): n = 24; Group 2 (non-users): n = 29
No significant difference between the groups in demographics was reported
The Cystic Fibrosis Quality of Life Questionnaire (CFQ-R) was also administered to patients as part of the assessment battery
What This Means
This research suggests that children with cystic fibrosis (CF) who were taking a newer class of medications called CFTR modulators had lower levels of anxiety compared to CF patients who were not taking these medications. CFTR modulators work by targeting the underlying protein defect that causes CF, rather than just treating symptoms, and have been shown to improve lung function and other physical health outcomes. This study, conducted in Turkey with 53 children with CF and their parents, also assessed mental health using standardized questionnaires for both patients and caregivers.
The study also found that parents of children taking CFTR modulators reported significantly lower levels of both anxiety and depression compared to parents of children not on these medications. While quality of life scores were also higher for parents in the modulator group, that difference narrowly missed statistical significance. No significant difference in depression scores was found between the two groups of children themselves, though anxiety scores did differ.
This research suggests a potential psychological benefit of CFTR modulator therapy that extends beyond the patient to their family members, possibly reflecting reduced worry about disease progression when effective treatment is in place. However, because this was an observational study — meaning the researchers observed existing groups rather than randomly assigning treatments — it is not possible to conclude that the modulators directly caused the mental health improvements. Other factors, such as overall disease severity, could also explain the differences between groups. Larger, longer-term studies would be needed to confirm these findings.
Yetişgin H, Akyan Soydaş &, Özkan Tabakçı S, Bilgiç I, Kürtül Çakar M, Akca Dinç G, et al.. (2026). Early effect of CFTR modulators on the mental health of patients with cystic fibrosis and parents.. European journal of pediatrics. https://doi.org/10.1007/s00431-026-07130-y