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<p>The cystic fibrosis transmembrane conductance regulator (CFTR) modulators were shown to improve clinical symptoms in patients with cystic fibrosis (CF). However, the effects of modulator therapy on the mental health of patients with CF remain uncertain. We aimed to investigate the impact of the CFTR modulator therapy on the mental health of children with CF and their parents. This prospective observational study was conducted on children with CF who used modulator therapy (Group 1, <i>n</i> = 24) and those who did not (Group 2, <i>n</i> = 29) and parents (<i>n</i>, 53). Cystic Fibrosis Quality of Life Questionnaire (CFQ-R), Children’s Depression Inventory (CDI), and Screen for Child Anxiety Related Emotional Disorders (SCARED) were applied to patients. Beck Depression Inventory (BDI), Beck Anxiety Inventory (BAI), and World Health Organization Quality of Life Scale-Short Form (WHOQOL-BREF) were administered to parents. There was no significant difference between the groups in demographics. SCARED scores were higher in patients who did not use modulator therapy (<i>p</i> = 0.016). Anxiety and depression scores of BDI and BAI scales applied to parents were found to be statistically significantly higher in parents of patients who did not use modulator therapy (<i>p</i> = 0.006, <i>p</i> = 0.002, respectively). WHOQOL-BREF scores were higher among parents of patients who used modulator therapy, but the difference was not statistically significant (<i>p</i> = 0.060).</p><p><i>Conclusion</i>: These findings suggest a possible association between CFTR modulator use and lower anxiety and depression scores in both patients and parents, though causal conclusions cannot be drawn from this observational study.</p><p><Table Float="No" ID="Taba"> <tgroup cols="2"> <colspec align="left" colname="c1" colnum="1" /> <colspec align="left" colname="c2" colnum="2" /> <tbody> <row> <entry nameend="c2" namest="c1"> <p><b>What is Known:</b></p> <p>• <i>CFTR modulator therapies have been increasingly used in recent years with demonstrated beneficial effects on clinical outcomes</i>.</p> <p>• <i>The effects of CFTR modulator therapies on mental health are not yet clearly defined</i>.</p> </entry> </row> <row> <entry nameend="c2" namest="c1"> <p><b>What is New:</b></p> <p>• <i>CFTR modulator use was associated with lower anxiety scores in children with cystic fibrosis and lower anxiety and depression scores among their parents</i>.</p> <p>• <i>These findings suggest a potential early psychosocial benefit of CFTR modulator therapy for both patients and caregivers; however, they should be interpreted cautiously because the observational design does not allow causal conclusions</i>.</p> </entry> </row> </tbody> </tgroup> </Table></p>

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Early effect of CFTR modulators on the mental health of patients with cystic fibrosis and parents

  • 期刊论文

  • 发表日期：

    2026年6月3日

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  • Hande Yetişgin,
  • Şule Selin Akyan Soydaş,
  • Satı Özkan Tabakçı,
  • Işıl Bilgiç,
  • Meltem Kürtül Çakar,
  • Gamze Akca Dinç,
  • Ayyüce Ünlü,
  • Çelebi Yıldırım,
  • Muhammet Ali Çetin,
  • Gökçen Dilşa Tuğcu,
  • Dilber Ademhan Tural,
  • Sanem Eryılmaz Polat,
  • Gülser Şenses Dinç,
  • Esra Çöp,
  • Güzin Cinel

摘要

The cystic fibrosis transmembrane conductance regulator (CFTR) modulators were shown to improve clinical symptoms in patients with cystic fibrosis (CF). However, the effects of modulator therapy on the mental health of patients with CF remain uncertain. We aimed to investigate the impact of the CFTR modulator therapy on the mental health of children with CF and their parents. This prospective observational study was conducted on children with CF who used modulator therapy (Group 1, n = 24) and those who did not (Group 2, n = 29) and parents (n, 53). Cystic Fibrosis Quality of Life Questionnaire (CFQ-R), Children’s Depression Inventory (CDI), and Screen for Child Anxiety Related Emotional Disorders (SCARED) were applied to patients. Beck Depression Inventory (BDI), Beck Anxiety Inventory (BAI), and World Health Organization Quality of Life Scale-Short Form (WHOQOL-BREF) were administered to parents. There was no significant difference between the groups in demographics. SCARED scores were higher in patients who did not use modulator therapy (p = 0.016). Anxiety and depression scores of BDI and BAI scales applied to parents were found to be statistically significantly higher in parents of patients who did not use modulator therapy (p = 0.006, p = 0.002, respectively). WHOQOL-BREF scores were higher among parents of patients who used modulator therapy, but the difference was not statistically significant (p = 0.060).

Conclusion: These findings suggest a possible association between CFTR modulator use and lower anxiety and depression scores in both patients and parents, though causal conclusions cannot be drawn from this observational study.

What is Known:

• CFTR modulator therapies have been increasingly used in recent years with demonstrated beneficial effects on clinical outcomes.

• The effects of CFTR modulator therapies on mental health are not yet clearly defined.

What is New:

• CFTR modulator use was associated with lower anxiety scores in children with cystic fibrosis and lower anxiety and depression scores among their parents.

• These findings suggest a potential early psychosocial benefit of CFTR modulator therapy for both patients and caregivers; however, they should be interpreted cautiously because the observational design does not allow causal conclusions.