Validation of bronchiectasis patient reported outcome measures in adults with primary ciliary dyskinesia: Data from the EMBARC-BRIDGE study and BEAT-PCD

Patient reported outcomes are important measures in clinical trials. We assessed two bronchiectasis disease specific quality of life questionnaires in patients with bronchiectasis due to PCD. The Bronchiectasis Health Questionnaire is a ten question disease-specific tool with a total score ranging from 0-100 with 100 being best quality of life. The QoL-B has 37 items in 8 domains. Questionnaires were administered in 86 people with PCD from 11 centres in 9 European countries as part of the EMBARC-BRIDGE study Average patient age was 44yrs (+/-16yrs), 31 male (36%), FEV1%pred (66% +/- 24). Average BHQ score was 60.9, range from 42.91-83.83. Average QoLB-Respiratory score was 63 (11.1-88.9). BHQ Total score and Qol-B Resp showed good correlation with disease severity (BSI score r= -0.41; 95%CI -0.64 to -0.26, r=-0.34 95%CI -0.60-0.02) and breathlessness (MRC dyspnoea score r= -0.61; 95%CI -0.74 to -0.44, r=-0.55 95%CI -0.73 to -0.31), but not sputum volume or lung function p>0.05. There was good correlation between BHQ and all QoL-B domains (Physical r=0.61, Role 0.60, Vitality 0.80, Emotion, 0.54, Social 0.44, Treatment Burden 0.50, Health 0.70 and Respiration 0.73, all p<0.001). Patients with worse BHQ and QoLB-Resp scores had more annual exacerbations [no exacerbations (BHQ 65.8 CI 61.7-70.2, QolB 68.5 CI 59.4-77.8) vs 1-2 (61.35 56.4-66.4; 54.2 CI 45.4-63.0) vs 3+ (56.1, CI 53.1-59.9 ; 54.5. CI 42.2 – 66.7)]. MCID was calculated using 0.5stdev of the mean as 5.1 points for BHQ and 9.7 for QoLB-Resp. QOL-B and BHQ scores correlate with disease severity and clinical outcomes in adults with PCD.