Abstract / Summary
Background Patient-reported outcomes (PROs) have become essential endpoints in clinical trials for bronchiectasis. However, no validated PRO instrument currently exists for the Chinese population. This study aimed to develop and validate a disease-specific Patient-Reported Outcome scale for Bronchiectasis (BE-PRO) tailored to Chinese patients.Methods A mixed-methods approach was used to develop the BE-PRO. The conceptual framework and initial item pool were developed primarily from semi-structured patient interviews and supplemented by literature and existing-instrument reviews and medical-record analysis. Items were refined through qualitative review by experts and patients. Two field surveys were then conducted, with items selected using classical test theory (CTT), item response theory (IRT), and structural equation modeling (SEM). Finally, a formal validation survey assessed the psychometric properties of the BE-PRO.Results The final BE-PRO consists of 16 items organized into five domains: physiology, psychology, environment, society, and treatment satisfaction. The scale demonstrated excellent internal consistency (Cronbach’s α = 0.912), split-half reliability (0.856) and satisfactory structural validity (CFI = 0.961; RMSEA = 0.068). Strong correlations with the SF-36 and QOL-B supported criterion validity, while moderate correlations with the BSI and E-FACED supported its clinical relevance. The scale showed high feasibility, with 100% response and completion rates and a mean completion time of 9.07 min.Conclusion The 16-item BE-PRO is a reliable, psychometrically validated and feasible instrument for assessing patient-reported outcomes in Chinese patients with bronchiectasis. Its strong psychometric properties support its use in both clinical trials and routine practice.