{"title":"Elexacaftor/Tezacaftor/Ivacaftor Treatment Accessibility and Mental Health: Reducing Anxiety in People With Cystic Fibrosis.","authors":"Burcu Uzunoglu, Merve Selcuk Balci, Mine Kalyoncu, Seyda Karabulut, Neval Metin Cakar, Ceren Ayca Yildiz, Gamze Tastan, Damla Kocaman, Almala Pinar Ergenekon, Yasemin Gökdemir, Ela Erdem Eralp, Fazilet Karakoc, Bülent Karadag","doi":"10.1002/ppul.71037","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>Although modulator therapies have proven effective in cystic fibrosis (CF) access is limited due to reimbursement issues in Turkey. We aimed to examine anxiety and depression levels of people with CF (pwCF) and their caregivers according to their access to modulator treatment.</p><p><strong>Methods: </strong>Participants genetically eligible for elexacaftor/tezacaftor/ivacaftor (ETI) were divided into Group 1 (access via court decision, not yet on treatment) and Group 2 (unable to access due to reimbursement issues). Genetically ineligible participants formed Group 3. All pwCF and parents of those under 18 were screened for depression by the Patient Health Questionnaire-9 (PHQ-9) and for anxiety by the Generalized Anxiety Disorder-7 (GAD-7). Surveys for Group 1 patients were conducted just before starting ETI. Binary logistic regression analysis was performed to evaluate the effects of independent variables on anxiety and depression in pwCF and their primary caregivers.</p><p><strong>Results: </strong>A total of 389 pwCF and 285 caregivers were included. Group 3 (ineligible) had the highest depression rate (72.9%, n = 35), while Group 1 (pre-ETI) had the lowest (50.0%, n = 35). Median PHQ-9 scores were significantly lower in Group 1 (p < 0.006). Anxiety rates were higher in Groups 2 and 3 compared to Group 1 (p = 0.011 and p = 0.003, respectively). Access to ETI reduced the odds of anxiety by 67.7% (p = 0.029). Caregiver GAD-7 scores showed a weak negative correlation with pwCF age (r = -0.117).</p><p><strong>Conclusion: </strong>Limited access to modulator therapies is associated with higher depression and anxiety symptoms among pwCF. Addressing these barriers is critical to improving their well-being.</p>","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":"60 3","pages":"e71037"},"PeriodicalIF":2.7000,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11892081/pdf/","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Pediatric Pulmonology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1002/ppul.71037","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"PEDIATRICS","Score":null,"Total":0}
引用次数: 0
Abstract
Background: Although modulator therapies have proven effective in cystic fibrosis (CF) access is limited due to reimbursement issues in Turkey. We aimed to examine anxiety and depression levels of people with CF (pwCF) and their caregivers according to their access to modulator treatment.
Methods: Participants genetically eligible for elexacaftor/tezacaftor/ivacaftor (ETI) were divided into Group 1 (access via court decision, not yet on treatment) and Group 2 (unable to access due to reimbursement issues). Genetically ineligible participants formed Group 3. All pwCF and parents of those under 18 were screened for depression by the Patient Health Questionnaire-9 (PHQ-9) and for anxiety by the Generalized Anxiety Disorder-7 (GAD-7). Surveys for Group 1 patients were conducted just before starting ETI. Binary logistic regression analysis was performed to evaluate the effects of independent variables on anxiety and depression in pwCF and their primary caregivers.
Results: A total of 389 pwCF and 285 caregivers were included. Group 3 (ineligible) had the highest depression rate (72.9%, n = 35), while Group 1 (pre-ETI) had the lowest (50.0%, n = 35). Median PHQ-9 scores were significantly lower in Group 1 (p < 0.006). Anxiety rates were higher in Groups 2 and 3 compared to Group 1 (p = 0.011 and p = 0.003, respectively). Access to ETI reduced the odds of anxiety by 67.7% (p = 0.029). Caregiver GAD-7 scores showed a weak negative correlation with pwCF age (r = -0.117).
Conclusion: Limited access to modulator therapies is associated with higher depression and anxiety symptoms among pwCF. Addressing these barriers is critical to improving their well-being.
期刊介绍：
Pediatric Pulmonology (PPUL) is the foremost global journal studying the respiratory system in disease and in health as it develops from intrauterine life though adolescence to adulthood. Combining explicit and informative analysis of clinical as well as basic scientific research, PPUL provides a look at the many facets of respiratory system disorders in infants and children, ranging from pathological anatomy, developmental issues, and pathophysiology to infectious disease, asthma, cystic fibrosis, and airborne toxins. Focused attention is given to the reporting of diagnostic and therapeutic methods for neonates, preschool children, and adolescents, the enduring effects of childhood respiratory diseases, and newly described infectious diseases.
PPUL concentrates on subject matters of crucial interest to specialists preparing for the Pediatric Subspecialty Examinations in the United States and other countries. With its attentive coverage and extensive clinical data, this journal is a principle source for pediatricians in practice and in training and a must have for all pediatric pulmonologists.
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