Mental health, sleep, and quality of life following CFTR modulator therapy: a longitudinal study of children with cystic fibrosis and caregivers
European Journal of Pediatrics, cilt.185, sa.9, 2026 (SCI-Expanded, Scopus)
- Yayın Türü: Makale / Tam Makale
- Cilt numarası: 185 Sayı: 9
- Basım Tarihi: 2026
- Doi Numarası: 10.1007/s00431-026-07331-5
- Dergi Adı: European Journal of Pediatrics
- Derginin Tarandığı İndeksler: Science Citation Index Expanded (SCI-EXPANDED), Scopus, BIOSIS, CINAHL, EMBASE, MEDLINE, Academic Search Ultimate (EBSCO), Biomedical Reference Collection: Corporate Edition (EBSCO), Health Research Premium Collection (ProQuest)
- Anahtar Kelimeler: Anxiety, CFTR modulator therapy, Cystic fibrosis, Depression, Quality of life
- Ankara Yıldırım Beyazıt Üniversitesi Adresli: Evet
Özet
Modulator therapies have been shown to improve clinical outcomes, particularly respiratory outcomes, in children with cystic fibrosis (CF). However, their potential effects on sleep and mental health remain unclear. This study aimed to evaluate the impact of longitudinal modulator therapy on mental health, sleep, and quality of life (QOL) in children with CF and their caregivers. This prospective observational study is a longitudinal follow-up of our previously published cross-sectional study. Twenty-four children with CF receiving modulator therapy and their parents were enrolled. Participants were assessed at baseline and reassessed following an 18-month follow-up period. Patients completed the Cystic Fibrosis Quality of Life Questionnaire (CFQ-R), Child Depression Inventory (CDI), Screen for Child Anxiety Related Emotional Disorders (SCARED), and Epworth Sleepiness Scale (ESS). Parents completed the Beck Depression Inventory (BDI), Beck Anxiety Inventory (BAI), Zarit Burden Interview (ZBI), World Health Organization Quality of Life Scale – Short Form (WHOQOL-BREF), and CFQ-R parent-proxy questionnaires. At the end of the 18-month period, significant decreases were observed in patients' SCARED, CDI, and ESS scores (p = 0.002, p < 0.001, and p = 0.025, respectively) relative to baseline. Significant improvements were observed in BDI, BAI, and ZBI scores among parents, while significant increases were seen in WHOQOL-BREF scores (p = 0.001, p = 0.001, p < 0.001, and p < 0.001, respectively). Improvements were observed in the physical, social, and respiratory domains of the CFQ-R (p = 0.018, p = 0.025, and p = 0.043, respectively). Conclusion: Over an 18-month period, modulator therapy was associated with a reduced risk of anxiety, depression, and daytime sleepiness and improved QoL in children with CF. Additionally, improvements in parental QoL and reduced caregiving burden were observed. These results suggest that the benefits of modulator therapies may extend beyond clinical outcomes to the psychosocial well-being of families. (Table presented.)