ABSTRACT
BACKGROUND: Psychosocial risk factors are known to impact quality of life, treatment adherence, and health outcomes. No standardized comprehensive psychosocial risk screener is routinely utilized in cystic fibrosis (CF) care. The objectives of the study were to describe the range and severity of psychosocial risk within this CF population, investigate the reliability of a comprehensive psychosocial screener in pediatric CF clinical care, and explore relationships between psychosocial risk and key factors affecting health outcomes. It was hypothesized that the PAT-CF total and subscale α coefficients would be similar to those found in other pediatric medical populations. METHOD: Parents of 154 children with CF completed a CF-specific version of the Psychosocial Assessment Tool_All-lit (PAT-CF), an empirically-based psychosocial risk assessment, during routine CF clinical care. RESULTS: The internal consistency of the PAT-CF Total score was 0.71. Total score and subscale reliabilities reflect findings in other pediatric populations. Total risk scores fell in the following categories: 7% (Clinical-highest risk), 41% (Targeted), and 52% (Universal-lowest risk), respectively. Increased psychosocial risk was associated with Medicaid status and lower parent education, whereas having private insurance was associated with decreased psychosocial risk. CONCLUSIONS: The PAT-CF can feasibly be used as an empirically-based comprehensive psychosocial risk tool in routine CF care and is acceptable by parents. In addition to providing universal anticipatory guidance regarding child and family wellness, early identification of risk factors allows care teams to proactively provide targeted support and intervention for specific psychosocial risk factors to promote improved quality of life and ability to sustain daily care.
Subject(s)
Cystic Fibrosis/psychology , Surveys and Questionnaires , Adolescent , Caregivers , Child , Child, Preschool , Family Health , Female , Health Status , Humans , Male , Mass Screening , Mental Disorders/etiology , Parents , Psychometrics , Quality of Life , Reproducibility of Results , Risk Assessment/methods , Risk FactorsABSTRACT
Traditional pulmonary therapies for cystic fibrosis (CF) target the downstream effects of CF transmembrane conductance regulator (CFTR) dysfunction (the cause of CF). Use of one such therapy, ß-adrenergic bronchodilators (such as albuterol), is nearly universal for airway clearance. Conversely, novel modulator therapies restore function to select mutant CFTR proteins, offering a disease-modifying treatment. Recent trials of modulators targeting F508del-CFTR, the most common CFTR mutation, suggest that chronic ß-agonist use may undermine clinical modulator benefits. We therefore sought to understand the impact of chronic or excess ß-agonist exposure on CFTR activation in human airway epithelium. The present studies demonstrate a greater than 60% reduction in both wild-type and modulator-corrected F508del-CFTR activation following chronic exposure to short- and long-acting ß-agonists. This reduction was due to reduced cellular generation of cAMP downstream of the ß-2 adrenergic receptor-G protein complex. Our results point towards a posttranscriptional reduction in adenylyl cyclase function as the mechanism of impaired CFTR activation produced by prolonged ß-agonist exposure. ß-Agonist-induced CFTR dysfunction was sufficient to abrogate VX809/VX770 modulation of F508del-CFTR in vitro. Understanding the clinical relevance of our observations is critical for CF patients using these drugs, and for investigators to inform future CFTR modulator drug trials.
