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).
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.
Study Type
OBSERVATIONAL
Enrollment
600
Children's Hospital of Orange County
Orange, California, United States
RECRUITINGChildren's Hospital Colorado
Aurora, Colorado, United States
Prevalence of depression, anxiety, and behavioral problems in children with cystic fibrosis
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.
Time frame: 07/01/2024 - 06/30/2028
Diagnostic performance of brief behavioral and mental health screening instruments
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.
Time frame: 07/01/2024 - 06/30/2028
Longitudinal changes in child mental health symptoms
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.
Time frame: 07/01/2024 - 06/30/2028
Cystic fibrosis-specific health-related quality of life
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.
Time frame: 07/01/2024 - 06/30/2028
Parent symptoms of depression and anxiety
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Joe DiMaggio
Hollywood, Florida, United States
RECRUITINGNemours Foundation
Orlando, Florida, United States
RECRUITINGIndiana University
Bloomington, Indiana, United States
RECRUITINGMassachusetts General Hospital
Boston, Massachusetts, United States
RECRUITINGUniversity at Buffalo
Buffalo, New York, United States
RECRUITINGUniversity of North Carolina School of Medicine
Chapel Hill, North Carolina, United States
RECRUITINGCincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
RECRUITINGBrown University Health
Providence, Rhode Island, United States
ACTIVE_NOT_RECRUITING...and 3 more locations
Assess parent mental health using the Patient Health Questionnaire-8 (PHQ-8) and Generalized Anxiety Disorder-7 (GAD-7) at each assessment.
Time frame: 07/01/2024 - 06/30/2028
Neuropsychiatric symptoms associated with CFTR modulator therapy
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.
Time frame: 07/01/2024 - 06/30/2028
Qualitative experiences related to CFTR modulator-associated neuropsychiatric symptoms
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.
Time frame: 07/01/2024 - 06/30/2028