Development and initial validation of the bronchiectasis exacerbation and symptom tool (BEST)

Area of Science:

• Respiratory Medicine
• Clinical Trials
• Patient-Reported Outcomes

Background:

• Recurrent bronchiectasis exacerbations lead to lung function decline, disease progression, reduced quality of life, and increased mortality.
• Patient-completed diaries have improved exacerbation detection in COPD, suggesting potential for similar tools in bronchiectasis.
• Early and accurate identification of exacerbations is crucial for managing bronchiectasis.

Purpose of the Study:

• To develop and validate a novel symptom diary, the Bronchiectasis Exacerbation Symptom Tool (BEST) diary.
• To assess the BEST diary's ability to capture symptom burden and detect exacerbations in patients with bronchiectasis.
• To evaluate the BEST diary's correlation with existing health status measures.

Main Methods:

• Prospective observational study involving symptomatic bronchiectasis patients with a history of exacerbations.
• Daily symptom recording using the BEST diary, which includes cough, sputum, dyspnea, fatigue, and systemic symptoms (scored 0-26).
• Concurrent validity assessed by comparing BEST diary scores with COPD Assessment Test, Leicester Cough Questionnaire, St Georges Respiratory Questionnaire, and 6-minute walk test.

Main Results:

• Twenty-one patients were followed for 6 months, identifying 29 treated and 23 untreated exacerbations.
• The BEST diary demonstrated good correlation with established health status measures (e.g., COPD Assessment Test, r=0.61; Leicester Cough Questionnaire, r=-0.52).
• The mean BEST score change during exacerbations was 9.1 points, with a proposed minimum clinically important difference of 4 points. Mean exacerbation duration was 15.3 days.

Conclusions:

• The BEST symptom diary exhibits concurrent validity and responsiveness to exacerbation onset and recovery in bronchiectasis.
• The BEST diary is a promising tool for detecting and characterizing exacerbations in clinical trials.
• This diary can enhance the monitoring of symptom burden and disease activity in bronchiectasis management.