Patient-reported outcome measures developed for non–cystic fibrosis bronchiectasis may be applied to cystic fibrosis bronchiectasis

BACKGROUND: Bronchiectasis (BE) is a chronic lung disease that impacts health-related quality of life (HRQoL). BE has historically been separated into cystic fibrosis (CF)-related BE (CFBE) and non-CF-related BE (NCFBE [where NCFBE is etiologically heterogeneous or idiopathic]), despite both sharing similar pathophysiology. Patient-reported outcome measures (PROMs) assessing BE symptoms and impacts on HRQoL have largely been developed and validated in NCFBE. This study assessed whether PROMs developed for NCFBE reflect HRQoL experiences of people with CFBE. METHODS: A preliminary conceptual model of symptoms and HRQoL impacts most relevant to people with CFBE was developed based on expert opinion, the content of existing PROMs, and previous qualitative research. Items from 11 existing PROMs were mapped to this preliminary conceptual model. A focus group discussion guide was created from the preliminary conceptual model. US participants aged ≥ 19 years with a self-reported diagnosis of CF and BE were eligible for participation in one of two focus groups (December 2023 or January 2024). Focus groups were recorded and transcribed verbatim, and thematic analysis (ATLAS.ti Web) was used to identify recurring themes from the discussions. Final themes were mapped back to the preliminary conceptual model to identify potential gaps between existing PROMs and relevant HRQoL impacts. RESULTS: A total of 19 people with CFBE participated in the virtual focus groups. Most participants were female (63%) and non-Hispanic White (95%), ranging from 22 to 67 years of age. Focus group discussions revealed five primary themes: Emotional Symptoms, Physical Function, Physical Symptoms, Social Health, and Treatment Burden. A total of 242 unique mentions of BE-related symptoms and impacts on HRQoL were mapped to the model. Generally, concerns about symptoms and HRQoL impacts expressed by participants were consistent with the model. Potential discrepancies included feelings of loss of control, sinusitis, and hemoptysis. Although not included in the model, loss of control likely overlapped with anxiety or treatment burden. Hemoptysis, although raised during focus group discussions, was not captured in the model, but is captured in several PROMs. CONCLUSIONS: Measures developed to assess patient‑reported outcomes in NCFBE may be extended to CFBE, pending cognitive and psychometric validation.