Natural variability of lung function in primary ciliary dyskinesia: longitudinal analysis from the PROVALF-PCD cohort

Kód výsledku v 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>

Nalezeny alternativní kódy

RIV/00216208:11130/25:10499328

Výsledek na webu

<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>

Jazyk výsledku

angličtina

Název v původním jazyce

Natural variability of lung function in primary ciliary dyskinesia: longitudinal analysis from the PROVALF-PCD cohort

Popis výsledku v původním jazyce

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 &gt;=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 (&lt;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) &gt;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.

Název v anglickém jazyce

Natural variability of lung function in primary ciliary dyskinesia: longitudinal analysis from the PROVALF-PCD cohort

Popis výsledku anglicky

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 &gt;=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 (&lt;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) &gt;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.

Druh

J_imp - Článek v periodiku v databázi Web of Science

CEP obor

—

OECD FORD obor

30209 - Paediatrics

Projekt

—

Návaznosti

I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace

Rok uplatnění

2025

Kód důvěrnosti údajů

S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů

Název periodika

ERJ Open Research

ISSN

2312-0541

e-ISSN

2312-0541

Svazek periodika

11

Číslo periodika v rámci svazku

3

Stát vydavatele periodika

GB - Spojené království Velké Británie a Severního Irska

Počet stran výsledku

12

Strana od-do

01115-2024

Kód UT WoS článku

001523589100001

EID výsledku v databázi Scopus

2-s2.0-105009097829