Cystic Fibrosis (CF)
Conditions
Keywords
Cystic Fibrosis, Children, Mental health screening, Depression, Anxiety, CFTR modulators, Neuropsychiatric adverse events
Brief summary
This is a longitudinal, observational epidemiological study designed to estimate the prevalence of depression, anxiety, and behavior problems in children ages 18 months through 11 years with cystic fibrosis (CF).
Detailed description
Integration of mental health (MH) screening and treatment into cystic fibrosis (CF) care represents over 10 years of research and clinical progress, driven by elevated rates of depression and anxiety in the International Depression Epidemiological Study, MH guidelines, and CF Foundation implementation support to screen adolescents and adults in all CF Centers. Benefits of screening include earlier identification, greater access to care, reduced stigma, and positive uptake from the CF community. However, TIDES did not include children with CF under 12 years. Depression and anxiety have increased dramatically in young children, with new guidelines for MH screening of children in primary care. Given the pediatric MH crisis and the widespread adoption of cystic fibrosis fibrosis transmembrane conductance regulator (CFTR) modulator therapy, which have been associated with adverse events, there is an urgent need to gather MH data in children with CF \<12 years. Thus, the goals of this study are to evaluate the national, longitudinal prevalence of depression, anxiety, and behavior problems in children with CF 18 months through 11 years, evaluate and compare the performance of two widely used brief screeners (criterion validity, sensitivity, specificity) to identify the optimal measures for this population, and characterize neuropsychiatric adverse events (AEs) associated with CFTR modulator therapy in this age group. Purposive randomized sampling will be used to recruit 600 children (half 18 mos.-5 yrs. and half 6-11 yrs.) at 16 CF Centers across the US. This study will estimate the prevalence of children above the clinical cut-score on each symptom domain (depression, anxiety, behavior problems) and evaluate their longitudinal course and predictors. Rigorous mixed methods will be used to describe any potential AEs perceived by parents or children to be associated with CFTR modulator therapy. This study will provide the groundwork to extend mental health screening and care to younger children with CF.
Interventions
None listed
Sponsors
State University of New York at Buffalo
Cystic Fibrosis Foundation
Brown University
Study design
Observational model
OTHER
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Months to 11 Years
Healthy volunteers
No
Inclusion criteria
1. Child with a diagnosis of Cystic fibrosis (CF) actively followed by the CF care team at a participating site 2. Child is age 18 months thru 11 years 3. English and/or Spanish speaking 4. Parent/legal guardian willing and able to give informed consent, and for minor participants ages 7 thru 11 years able to give assent.
Exclusion criteria
* Unable or unwilling to participate in study procedures, or at Site PI discretion.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Prevalence of depression, anxiety, and behavioral problems in children with cystic fibrosis | 07/01/2024 - 06/30/2028 | Estimate the national prevalence of depression, anxiety, and behavioral problems among children with cystic fibrosis aged 18 months through 11 years using the Behavior Assessment System for Children, Third Edition (BASC-3). At the baseline assessment (Time 1), parents complete the age-appropriate BASC-3 Parent Rating Scale, and children aged 8-11 years complete the BASC-3 Self-Report of Personality. BASC-3 classifications of emotional and behavioral symptoms will be used to estimate the prevalence of clinically significant symptoms. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Diagnostic performance of brief behavioral and mental health screening instruments | 07/01/2024 - 06/30/2028 | Evaluate the diagnostic accuracy and clinical utility of the Pediatric Symptom Checklist (PSC-Preschool, PSC-17, and PSC-Y-17) and PROMIS short forms for anxiety, depressive symptoms, anger/irritability, sleep disturbance, cognitive functioning, flexibility, and persistence by comparing their performance with BASC-3 at baseline. |
| Longitudinal changes in child mental health symptoms | 07/01/2024 - 06/30/2028 | Assess changes in parent-reported and child self-reported mental health symptoms over three study assessments using PSC and PROMIS measures. BASC-3 is administered only at the first assessment. |
| Cystic fibrosis-specific health-related quality of life | 07/01/2024 - 06/30/2028 | Assess CF-specific health-related quality of life using age-appropriate versions of the Cystic Fibrosis Questionnaire-Revised (CFQ-R). Parent-proxy and child self-report versions are administered according to participant age, with the Preschool Pictorial CFQ-R administered once for eligible children. |
| Parent symptoms of depression and anxiety | 07/01/2024 - 06/30/2028 | Assess parent mental health using the Patient Health Questionnaire-8 (PHQ-8) and Generalized Anxiety Disorder-7 (GAD-7) at each assessment. |
| Neuropsychiatric symptoms associated with CFTR modulator therapy | 07/01/2024 - 06/30/2028 | Evaluate parent-reported new or worsening neuropsychiatric symptoms following initiation of elexacaftor/tezacaftor/ivacaftor (ETI) or vanzacaftor/tezacaftor/deutivacaftor (VTD) using the CFTR Modulator Survey. Symptoms include anxiety, depression, attention problems, behavioral dysregulation, irritability, and sleep disturbance. The Modified Naranjo Scale will be used to assess the likelihood that reported symptoms are related to CFTR modulator therapy. |
| Qualitative experiences related to CFTR modulator-associated neuropsychiatric symptoms | 07/01/2024 - 06/30/2028 | Conduct semi-structured qualitative interviews with a purposive sample of parents reporting worsening neuropsychiatric symptoms following CFTR modulator initiation or non-initiation because of mental health concerns. Eligible school-aged children may also participate. Interview transcripts will undergo thematic analysis. |
Countries
United States
Contacts
CONTACTBeth A Smith, MD
CONTACTMinu Mohan, MPH
Outcome results
None listed