Abstract Rationale Respiratory tract exacerbations (RTEs) are common and a major source of morbidity among people with primary ciliary dyskinesia (PCD). They are a logical target for PCD clinical trials but are poorly characterized. Objective to prospectively describe self-reported RTEs in terms of rate, treatment, and symptom recovery. Methods Multicenter, prospective, longitudinal, observational study in children and adults with PCD utilizing remote monitoring. Participants (age ≥6 with confirmed PCD at 6 North American sites) enrolled for ∼12 months, during which they completed electronic patient-reported outcomes (PROs), home spirometry and home respiratory sample collection. A weekly survey asked if they were experiencing new or increased cough or difficulty breathing. An affirmative response defined the onset of an RTE, during which participants completed “sick state” surveys including the PCD-Respiratory Symptom Diary (PCD-RSD) daily for 14 days. We describe patient-reported RTEs, including feasibility of remote weekly surveys; and rate, treatment, and symptom trajectory. The difference between the mean PCD-RSD score at Days 1 and 14 was estimated with a linear mixed effects model with a subject specific intercept and repeated RTEs nested within subject. Results 105 participants enrolled between 2022 and 2024, of whom 99 completed ≥1 study procedure and comprise the study cohort. Mean age was 24.3 (SD 16.6) years, 49.5% female, 89.6% white / 4% Black / 2% Asian, 9% Hispanic, mean FEV1 82.4 % predicted (SD 22.9) and 22.2% were colonized with Pseudomonas aeruginosa. Participants completed a median of 50 weekly surveys (range 7-58) over a median of 53 weeks (range 11-71), representing 85% adherence to expected completion. 84 participants reported 242 RTEs (median 3/yr (range 1-9)). 47 participants reported starting oral antibiotics for an RTE (median 2 courses/yr (range 1-7)). Across the 242 RTEs, 2353 PCD Respiratory Symptom Diaries were completed; ≥1 diary was completed for 231 RTEs (95%). Figure 1 shows the daily symptom score trajectory across all RTEs. The difference in the mean PCD-RSD score between Day 0 and 14 was -12.5 (SE = 0.93) (p 0.01). Conclusions Adherence to weekly self-report of RTEs over a year in a cohort of children and adults with PCD was high and revealed RTEs to be common. Mean symptom scores improved significantly over the course of the RTEs. Next steps include evaluating associations of RTEs with longitudinal trajectories of additional PROs, lung function by home spirometry, sputum microbiology, and respiratory viral detection and airway inflammation in a subset. This abstract is funded by: NIH 5U54HL096458, 5R21TR004057
Kinghorn et al. (Fri,) studied this question.