| Title: |
Natural variability of lung function in primary ciliary dyskinesia: longitudinal analysis from the PROVALF-PCD cohort |
| Authors: |
Zhang, K; Kant, A; Boon, M; Borrelli, M; Constant, C; Corullon, SC; Cutrera, R; Dillenhöfer, S; Polat, SE; Eralp, E; Feyaerts, N; Harris, A; Hogg, C; Koerner-Rettberg, C; Kouis, P; Lombardi, E; Lorent, N; Marthin, JK; Martinu, V; Moreno-Galdo, A; Morgan, L; Nielsen, K; Omran, H; Ozcelik, U; Pohunek, P; Robinson, P; Rovira-Amigo, S; Santamaria, F; Schlegtendal, A; Tamalet, A; Thouvenin, G; Tural, DA; Ullmann, N; Walker, WT; Yiallouros, P; Pearse, C; Frauchiger, B; Kuehni, CE; Beydon, N; Latzin, P; Lucas, JS; Rubbo, B |
| Publisher Information: |
European Respiratory Society (ERS) |
| Publication Year: |
2025 |
| Collection: |
The University of Melbourne: Digital Repository |
| Description: |
BACKGROUND: The extent to which changes in lung function are due to natural variability in patients with primary ciliary dyskinesia (PCD) is unknown. We aimed to assess intra-individual variability in forced expiratory volume in 1 s (FEV1) derived from spirometry to define the extent to which the observed changes were due to test variability in clinically stable PCD patients. METHODS: PROVALF-PCD (Prospective Observational Multicentre Study on Variability of Lung Function in Stable PCD Patients) was a large international prospective cohort conducted in 2017-2019. We included patients aged ≥5 years who were clinically stable at two or more consecutive visits and provided spirometry-derived lung function measurements. To calculate the upper limit of normal (ULN), we fitted an unadjusted multilevel mixed-effect model, and to determine the absolute change in FEV1 z-scores, we calculated the coefficient of repeatability (CR). We performed sensitivity analyses by stratifying relative change by age (adults versus children), number of measurements (at least four), and time between measurements (25% between visits in stable PCD patients lie beyond the expected test variability and therefore could be considered physiologically relevant. These findings inform the selection of end-points for pulmonary intervention trials in PCD, as they suggest that FEV1 is not a sensitive test for monitoring lung health in PCD. |
| Document Type: |
article in journal/newspaper |
| Language: |
English |
| ISSN: |
2312-0541 |
| Relation: |
https://hdl.handle.net/11343/364447 |
| Availability: |
https://hdl.handle.net/11343/364447 |
| Rights: |
https://creativecommons.org/licenses/by/4.0/ ; CC-BY |
| Accession Number: |
edsbas.581F4E60 |
| Database: |
BASE |