{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,23]],"date-time":"2026-08-23T15:44:31Z","timestamp":1787499871360,"version":"build-2736575974"},"reference-count":33,"publisher":"European Respiratory Society (ERS)","issue":"5","license":[{"start":{"date-parts":[[2023,8,17]],"date-time":"2023-08-17T00:00:00Z","timestamp":1692230400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"name":"Rigshospitalets research foundation"},{"name":"B\u00f8rnelungefonden"},{"name":"T\u00f8mrermester J\u00f8rgen Holm og Hustru Elisa f. Hansens Mindelegat","award":["21049"],"award-info":[{"award-number":["21049"]}]}],"content-domain":{"domain":["publications.ersnet.org"],"crossmark-restriction":true},"short-container-title":["ERJ Open Res"],"accepted":{"date-parts":[[2023,8,1]]},"published-print":{"date-parts":[[2023,9]]},"abstract":"<jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>Cystic fibrosis (CF) lung disease starts in infancy and can be assessed for structural lung abnormalities using computed tomography or magnetic resonance scans, or for lung function impairment using multiple breath washout (MBW). However, in infancy these two methods are not well correlated. Trajectories of CF lung disease assessed by MBW in infants and toddlers remain poorly described, which is why we aimed to 1) describe the trajectory of lung function, 2) explore risk factors for progression and 3) explore the real-life effect of lumacaftor\/ivacaftor.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>This was a nationwide observational cohort study (2018\u20132021) using data collected as part of the routine clinical surveillance programme (including MBW and monthly endo-laryngeal suction sampling for bacterial pathogens) in children born after implementation of newborn screening for CF (May 2016). Lumacaftor\/ivacaftor commenced from age 2\u2005years in children homozygous for F508del. Ventilation distribution efficiency (VDE), recently described to have advantages over lung clearance index (LCI), was reported as the primary MBW outcome after z-score calculations based on published reference data. Mixed effect linear regression models were the main statistical analyses performed in this study.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      59 children, aged 2\u201345\u2005months, contributed with 211 MBW occasions (median (interquartile range (IQR)) 3 (2\u20135) MBW occasions per child) with a median (IQR) follow-up time of 10.8 (5.2\u201322.3)\u2005months. An overall mean annual deterioration rate of \u22120.50 (95% CI \u22120.78\u2013\u2009\u22120.22) z-VDE was observed, starting from an estimated mean z-VDE of \u22121.68 (95% CI \u22122.15\u2013\u2009\u22121.22) at age 0.0\u2005years (intercept).\n                      <jats:italic>Pseudomonas aeruginosa<\/jats:italic>\n                      \u201cever\u201d (n=14, MBWs 50) had a significantly worse z-VDE trajectory\n                      <jats:italic>versus P. aeruginosa<\/jats:italic>\n                      \u201cnever\u201d (mean difference 0.53 (95% CI 0.16\u20130.89)\u2005per year; p=0.0047) and lumacaftor\/ivacaftor treatment (n=22, MBWs 46) significantly improved the trajectory of z-VDE (mean difference 1.72 (95% CI 0.79\u20132.66)\u2005per year; p=0.0004), leading to a stable mean z-VDE trajectory after start of treatment.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>\n                      Infants and toddlers with CF demonstrated progressive deterioration in z-VDE over the first years of life.\n                      <jats:italic>P. aeruginosa<\/jats:italic>\n                      isolation \u201cever\u201d was associated with an accelerated deterioration in lung function, while lumacaftor\/ivacaftor therapy significantly improved and stabilised the trajectory.\n                    <\/jats:p>\n                  <\/jats:sec>","DOI":"10.1183\/23120541.00317-2023","type":"journal-article","created":{"date-parts":[[2023,8,17]],"date-time":"2023-08-17T12:11:20Z","timestamp":1692274280000},"page":"00317-2023","update-policy":"https:\/\/doi.org\/10.1183\/ers-crossmark-policy","source":"Crossref","is-referenced-by-count":7,"title":["Nationwide lung function monitoring from infancy in newborn-screened children with cystic fibrosis"],"prefix":"10.1183","volume":"9","author":[{"given":"Rikke 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