10.3390/polym8030084

Vollständiger Abstract

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<b>Background/Objectives:</b> Highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy has changed cystic fibrosis (CF) care, but Romanian patient-reported health-related quality of life (HRQoL) data remain limited. This national cross-sectional mixed-methods survey aimed to describe patient- and parent-reported HRQoL in Romanian people with CF (pwCF) aged ≥6 years using age-appropriate Cystic Fibrosis Questionnaire-Revised (CFQ-R) versions, with the respiratory domain as the primary outcome. <b>Methods:</b> The online survey was conducted between February and April 2026. Respondents provided demographic and clinical data, including genotype/F508del status, CFTR modulator status, percent predicted forced expiratory volume in 1 s (ppFEV 1 ) category, weight, height, and IV antibiotic-treated pulmonary exacerbations in the previous year. CFQ-R scores were summarized descriptively, and exploratory unadjusted associations were assessed using Mann-Whitney U tests and Spearman correlations. Open-ended responses were analyzed descriptively. <b>Results:</b> Of 67 responses, 61 were included: 43 participants aged 6-13 years and 18 aged ≥14 years. Most respondents were currently receiving CFTR modulators (52/61, 85.2%), predominantly elexacaftor/tezacaftor/ivacaftor. Mean CFQ-R Respiratory scores were 74.5 ± 23.1 in parent-proxy questionnaires for children aged 6-13 years, 83.3 ± 11.8 in self-reported questionnaires for children aged 12-13 years, and 69.8 ± 27.4 among respondents aged ≥14 years. Respiratory scores were higher among currently treated respondents and those with at least one F508del variant, and lower among those reporting IV antibiotic-treated exacerbations. Treatment burden remained among the lower-scoring domains. Qualitative responses described perceived respiratory, nutritional, and functional improvements, alongside residual treatment burden, psychosocial challenges, and access-related expectations. <b>Conclusions:</b> This national Romanian mixed-methods survey provides descriptive, exploratory CFQ-R-based HRQoL data in the CFTR modulator era. The findings suggest more favorable respiratory HRQoL among currently treated respondents, while supporting continued multidimensional patient-reported outcome monitoring in Romanian CF care.

Abstract: PubMed · Datensatz