The Bronchiectasis Exacerbation Diary: a novel patient-reported outcome for non-cystic fibrosis bronchiectasis

Abstract

Bronchiectasis is a chronic, progressive lung disease believed to result from a vicious cycle of infection and inflammation, with symptoms of chronic cough with sputum production, chronic fatigue, rhinosinusitis, chest pain, breathlessness and haemoptysis. There are currently no established instruments to monitor daily symptoms and exacerbations for use in clinical trials. Following a literature review and three expert clinician interviews, we conducted concept elicitation interviews with 20 patients with bronchiectasis to understand their personal disease experience. Findings from literature and clinician feedback were used to develop a draft version of the Bronchiectasis Exacerbation Diary (BED), which was designed to monitor key symptoms on a daily basis and during exacerbations. Patients were eligible to be interviewed if they were US residents aged ≥18 years, had a computed tomography scan-confirmed diagnosis of bronchiectasis with ≥two exacerbations in the previous 2 years and had no other uncontrolled respiratory conditions. Four waves of five patient interviews each were conducted. Patients (n=20) had a mean±_SD age of 53.9±12.8 years, and most were female (85%) and white (85%). A total of 33 symptoms and 23 impacts arose from the patient concept elicitation interviews. The BED was revised and finalised based upon patient feedback. The final BED is a novel, eight-item patient-reported outcome (PRO) instrument for monitoring key exacerbation symptoms on a daily basis with content validity established through comprehensive qualitative research and direct patient insight. The BED PRO development framework will be completed following psychometric evaluations of the data from a phase 3 bronchiectasis clinical trial.

FIGURE 1

Development of the draft Bronchiectasis Exacerbation Diary. PRO: patient-reported outcome.

FIGURE 2

Workflow for patient interviews. BED: Bronchiectasis Exacerbation Diary.

FIGURE 3

Mean disturbance rating (left) and number of patient mentions (right) for signs and symptoms of bronchiectasis. ^#: concept was identified during wave 1 of patient interviews; ^¶: concept was identified during wave 3 of patient interviews; ⁺: concept was identified during wave 4 of patient interviews.

FIGURE 4

Final bronchiectasis conceptual model: salient concepts. ^#: sputum production refers to the act of coughing up sputum, whereas sputum volume refers to the amount of sputum.

FIGURE 5

Final daily eight-item Bronchiectasis Exacerbation Diary. ^#: includes “have not noticed” as a response option.