TY - JOUR
T1 - Lung function from school age to adulthood in primary ciliary dyskinesia
AU - Halbeisen, Florian S.
AU - Pedersen, Eva S. L.
AU - Goutaki, Myrofora
AU - Spycher, Ben D.
AU - Amirav, Israel
AU - Boon, Mieke
AU - Cohen-Cymberknoh, Malena
AU - Crowley, Suzanne
AU - Emiralioglu, Nagehan
AU - Haarman, Eric G.
AU - Karadag, Bulent
AU - Koerner-Rettberg, Cordula
AU - Latzin, Philipp
AU - Loebinger, Michael R.
AU - Lucas, Jane S.
AU - Mazurek, Henryk
AU - Morgan, Lucy
AU - Marthin, June
AU - Pohunek, Petr
AU - Santamaria, Francesca
AU - Schwerk, Nicolaus
AU - Thouvenin, Guillaume
AU - Yiallouros, Panayiotis
AU - Nielsen, Kim G.
AU - Kuehni, Claudia E.
N1 - Funding Information: Support statement: This study is supported by Swiss National Science Foundation (320030B_192804). The development of the iPCD Cohort has been funded from the European Union’s Seventh Framework Programme under EG-GA number 35404 BESTCILIA: Better Experimental Screening and Treatment for Primary Ciliary Dyskinesia. PCD research at ISPM Bern also receives national funding from the Lung Leagues of Bern, St Gallen, Vaud, Ticino, and Valais, and the Milena-Carvajal Pro Kartagener Foundation. M. Goutaki is supported by a Swiss National Science Foundation fellowship (PZ00P3_185923). Most participating researchers and data contributors participate in the BEAT-PCD clinical research collaboration, supported by the European Respiratory Society and the ERN-LUNG (PCD core). The contribution by the Prague centre was supported by Czech health research council AZV ČR (NV 19-07-00210). Funding information for this article has been deposited with the Crossref Funder Registry. Publisher Copyright: © 2022 European Respiratory Society. All rights reserved.
PY - 2022/10/1
Y1 - 2022/10/1
N2 - Primary ciliary dyskinesia (PCD) presents with symptoms early in life and the disease course may be progressive, but longitudinal data on lung function are scarce. This multinational cohort study describes lung function trajectories in children, adolescents and young adults with PCD. We analysed data from 486 patients with repeated lung function measurements obtained between the age of 6 and 24 years from the International PCD Cohort and calculated z-scores for forced expiratory volume in 1 s (FEV1), forced vital capacity (FVC) and FEV1/FVC ratio using the Global Lung Function Initiative 2012 references. We described baseline lung function and change of lung function over time and described their associations with possible determinants in mixed-effects linear regression models. Overall, FEV1, FVC and FEV1/FVC z-scores declined over time (average crude annual FEV1 decline was −0.07 z-scores), but not at the same rate for all patients. FEV1 z-scores improved over time in 21% of patients, remained stable in 40% and declined in 39%. Low body mass index was associated with poor baseline lung function and with further decline. Results differed by country and ultrastructural defect, but we found no evidence of differences by sex, calendar year of diagnosis, age at diagnosis, diagnostic certainty or laterality defect. Our study shows that on average lung function in PCD declines throughout the entire period of lung growth, from childhood to young adult age, even among patients treated in specialised centres. It is essential to develop strategies to reverse this tendency and improve prognosis.
AB - Primary ciliary dyskinesia (PCD) presents with symptoms early in life and the disease course may be progressive, but longitudinal data on lung function are scarce. This multinational cohort study describes lung function trajectories in children, adolescents and young adults with PCD. We analysed data from 486 patients with repeated lung function measurements obtained between the age of 6 and 24 years from the International PCD Cohort and calculated z-scores for forced expiratory volume in 1 s (FEV1), forced vital capacity (FVC) and FEV1/FVC ratio using the Global Lung Function Initiative 2012 references. We described baseline lung function and change of lung function over time and described their associations with possible determinants in mixed-effects linear regression models. Overall, FEV1, FVC and FEV1/FVC z-scores declined over time (average crude annual FEV1 decline was −0.07 z-scores), but not at the same rate for all patients. FEV1 z-scores improved over time in 21% of patients, remained stable in 40% and declined in 39%. Low body mass index was associated with poor baseline lung function and with further decline. Results differed by country and ultrastructural defect, but we found no evidence of differences by sex, calendar year of diagnosis, age at diagnosis, diagnostic certainty or laterality defect. Our study shows that on average lung function in PCD declines throughout the entire period of lung growth, from childhood to young adult age, even among patients treated in specialised centres. It is essential to develop strategies to reverse this tendency and improve prognosis.
UR - http://www.scopus.com/inward/record.url?scp=85140416482&partnerID=8YFLogxK
U2 - https://doi.org/10.1183/13993003.01918-2021
DO - https://doi.org/10.1183/13993003.01918-2021
M3 - Article
C2 - 35301251
SN - 0903-1936
VL - 60
JO - European respiratory journal
JF - European respiratory journal
IS - 4
M1 - 2101918
ER -