Natural variability of lung function in primary ciliary dyskinesia: longitudinal analysis from the PROVALF-PCD cohort
The result's identifiers
Result code in IS VaVaI
<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00064203%3A_____%2F25%3A10499328" target="_blank" >RIV/00064203:_____/25:10499328 - isvavai.cz</a>
Alternative codes found
RIV/00216208:11130/25:10499328
Result on the web
<a href="https://verso.is.cuni.cz/pub/verso.fpl?fname=obd_publikace_handle&handle=KYB71Fzq0Y" target="_blank" >https://verso.is.cuni.cz/pub/verso.fpl?fname=obd_publikace_handle&handle=KYB71Fzq0Y</a>
DOI - Digital Object Identifier
<a href="http://dx.doi.org/10.1183/23120541.01115-2024" target="_blank" >10.1183/23120541.01115-2024</a>
Alternative languages
Result language
angličtina
Original language name
Natural variability of lung function in primary ciliary dyskinesia: longitudinal analysis from the PROVALF-PCD cohort
Original language 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 (FEV(1)) 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 FEV(1) 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 (<4 months apart). RESULTS: We included 252 participants from 12 countries with confirmed or highly likely PCD. We included 1028 FEV(1) measurements from patients in stable state. The ULN for relative change between two measurements of FEV(1) was 25%. Test variability remained high in all sensitivity analyses. The CR was 1.88 FEV(1) z-score. CONCLUSIONS: Changes in intra-individual FEV(1) >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 FEV(1) is not a sensitive test for monitoring lung health in PCD.
Czech name
—
Czech description
—
Classification
Type
J<sub>imp</sub> - Article in a specialist periodical, which is included in the Web of Science database
CEP classification
—
OECD FORD branch
30209 - Paediatrics
Result continuities
Project
—
Continuities
I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace
Others
Publication year
2025
Confidentiality
S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů
Data specific for result type
Name of the periodical
ERJ Open Research
ISSN
2312-0541
e-ISSN
2312-0541
Volume of the periodical
11
Issue of the periodical within the volume
3
Country of publishing house
GB - UNITED KINGDOM
Number of pages
12
Pages from-to
01115-2024
UT code for WoS article
001523589100001
EID of the result in the Scopus database
2-s2.0-105009097829