The clinical impact of Lumacaftor-Ivacaftor on structural lung disease and lung function in children aged 6–11 with cystic fibrosis in a real-world setting

[1]  M. Rosenfeld,et al.  WS18.06 Fully automated analysis of airway-artery dimensions on chest-computed tomography in preschool children with cystic fibrosis to evaluate the effect of inhaled hypertonic saline , 2022, Journal of Cystic Fibrosis.

[2]  R. Kronmal,et al.  The effect of inhaled hypertonic saline on lung structure in children aged 3-6 years with cystic fibrosis (SHIP-CT): a multicentre, randomised, double-blind, controlled trial. , 2022, The Lancet. Respiratory medicine.

[3]  Rutger M van den Bor,et al.  Prediction of Real-World Long-Term Outcomes of People with CF Homozygous for the F508del Mutation Treated with CFTR Modulators , 2021, Journal of personalized medicine.

[4]  M. Revel,et al.  Using chest computed tomography and unsupervised machine learning for predicting and evaluating response to lumacaftor–ivacaftor in people with cystic fibrosis , 2021, European Respiratory Journal.

[5]  R. Enaud,et al.  Long-Term Outcomes in Real Life of Lumacaftor–Ivacaftor Treatment in Adolescents With Cystic Fibrosis , 2021, Frontiers in Pediatrics.

[6]  P. Reix,et al.  Real-world assessment of LCI following lumacaftor-ivacaftor initiation in adolescents and adults with cystic fibrosis. , 2021, Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society.

[7]  G. Gorincour,et al.  Computed Tomographic Changes in Patients with Cystic Fibrosis Treated by Combination Therapy with Lumacaftor and Ivacaftor , 2021, Journal of clinical medicine.

[8]  C. Owen,et al.  Long-term safety and efficacy of lumacaftor-ivacaftor therapy in children aged 6-11 years with cystic fibrosis homozygous for the F508del-CFTR mutation: a phase 3, open-label, extension study. , 2021, The Lancet. Respiratory medicine.

[9]  J. Clancy,et al.  Clinical Effectiveness of Lumacaftor/Ivacaftor in Patients with Cystic Fibrosis Homozygous for F508del-CFTR. A Clinical Trial , 2021, Annals of the American Thoracic Society.

[10]  S. Verhulst,et al.  The short-term effects of ORKAMBI (lumacaftor/ivacaftor) on regional and distal lung structures using functional respiratory imaging , 2021, Therapeutic advances in respiratory disease.

[11]  T. Kotsimbos,et al.  Lumacaftor/ivacaftor-associated health stabilisation in adults with severe cystic fibrosis , 2020, ERJ Open Research.

[12]  E. Andrinopoulou,et al.  Chest computed tomography outcomes in a randomized clinical trial in cystic fibrosis: Lessons learned from the first ataluren phase 3 study , 2020, PloS one.

[13]  H. Tiddens,et al.  Risk factors for progression of structural lung disease in school-age children with cystic fibrosis. , 2020, Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society.

[14]  M. Skov,et al.  Prospective longitudinal association between repeated multiple breath washout measurements and computed tomography scores in children with cystic fibrosis. , 2020, Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society.

[15]  M. Skov,et al.  Patients with cystic fibrosis and advanced lung disease benefit from lumacaftor/ivacaftor treatment , 2020, Pediatric pulmonology.

[16]  J. Clancy,et al.  Changes in LCI in F508del/F508del patients treated with lumacaftor/ivacaftor: Results from the prospect study. , 2020, Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society.

[17]  S. Nagle,et al.  Guidance for computed tomography (CT) imaging of the lungs for patients with cystic fibrosis (CF) in research studies. , 2020, Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society.

[18]  J. Carlin,et al.  Early markers of cystic fibrosis structural lung disease: follow-up of the ACFBAL cohort , 2020, European Respiratory Journal.

[19]  D. Caimmi,et al.  Real-Life Safety and Effectiveness of Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis. , 2020, American journal of respiratory and critical care medicine.

[20]  S. Stanojevic,et al.  Integrating the multiple breath washout test into international multicentre trials. , 2019, Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society.

[21]  E. Andrinopoulou,et al.  Airway disease on chest computed tomography of preschool children with cystic fibrosis is associated with school‐age bronchiectasis , 2019, Pediatric pulmonology.

[22]  S. McColley,et al.  Lumacaftor/Ivacaftor reduces pulmonary exacerbations in patients irrespective of initial changes in FEV1. , 2019, Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society.

[23]  T. Corcoran,et al.  Effect of ivacaftor on mucociliary clearance and clinical outcomes in cystic fibrosis patients with G551D-CFTR. , 2018, JCI insight.

[24]  F. van Goor,et al.  VX‐445–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles , 2018, The New England journal of medicine.

[25]  E. Andrinopoulou,et al.  Quantitative assessment of airway dimensions in young children with cystic fibrosis lung disease using chest computed tomography , 2017, Pediatric pulmonology.

[26]  M. Maher,et al.  CORK Study in Cystic Fibrosis: Sustained Improvements in Ultra‐Low‐Dose Chest CT Scores After CFTR Modulation With Ivacaftor , 2017, Chest.

[27]  S. Stanojevic,et al.  Efficacy and safety of lumacaftor and ivacaftor in patients aged 6-11 years with cystic fibrosis homozygous for F508del-CFTR: a randomised, placebo-controlled phase 3 trial. , 2017, The Lancet. Respiratory medicine.

[28]  Marleen de Bruijne,et al.  Diagnosis of bronchiectasis and airway wall thickening in children with cystic fibrosis: Objective airway-artery quantification , 2017, European Radiology.

[29]  M. Rosenfeld,et al.  Lumacaftor/Ivacaftor in Patients Aged 6‐11 Years with Cystic Fibrosis and Homozygous for F508del‐CFTR , 2017, American journal of respiratory and critical care medicine.

[30]  C. Goss,et al.  Assessment of safety and efficacy of long-term treatment with combination lumacaftor and ivacaftor therapy in patients with cystic fibrosis homozygous for the F508del-CFTR mutation (PROGRESS): a phase 3, extension study. , 2017, The Lancet. Respiratory medicine.

[31]  H. Tiddens,et al.  Spirometer guided chest imaging in children: It is worth the effort! , 2017, Pediatric pulmonology.

[32]  M. Skov,et al.  Association between spirometry controlled chest CT scores using computer-animated biofeedback and clinical markers of lung disease in children with cystic fibrosis , 2017, European clinical respiratory journal.

[33]  M. Revel,et al.  Long-term computed tomographic changes in cystic fibrosis patients treated with ivacaftor , 2016, European Respiratory Journal.

[34]  Xiaohong Huang,et al.  Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR. , 2015, The New England journal of medicine.

[35]  Margaret Rosenfeld,et al.  Multiple-Breath Washout as a Lung Function Test in Cystic Fibrosis. A Cystic Fibrosis Foundation Workshop Report. , 2015, Annals of the American Thoracic Society.

[36]  Marleen de Bruijne,et al.  PRAGMA-CF. A Quantitative Structural Lung Disease Computed Tomography Outcome in Young Children with Cystic Fibrosis. , 2015, American journal of respiratory and critical care medicine.

[37]  Garry R. Cutting,et al.  Cystic fibrosis genetics: from molecular understanding to clinical application , 2014, Nature Reviews Genetics.

[38]  Janet Stocks,et al.  Consensus statement for inert gas washout measurement using multiple- and single- breath tests , 2013, European Respiratory Journal.

[39]  P. Sly,et al.  Progression of early structural lung disease in young children with cystic fibrosis assessed using CT , 2011, Thorax.

[40]  Matthias Griese,et al.  A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. , 2011, The New England journal of medicine.

[41]  Marleen de Bruijne,et al.  Cystic fibrosis: are volumetric ultra-low-dose expiratory CT scans sufficient for monitoring related lung disease? , 2009, Radiology.

[42]  J. Gustafson,et al.  Cystic Fibrosis , 2009, Journal of the Iowa Medical Society.

[43]  H. Tiddens,et al.  Multiple-breath inert gas washout and spirometry versus structural lung disease in cystic fibrosis , 2007, Thorax.

[44]  T. Robinson.  Computed tomography scanning techniques for the evaluation of cystic fibrosis lung disease. , 2007, Proceedings of the American Thoracic Society.

[45]  B. Yankaskas,et al.  Computed tomography reflects lower airway inflammation and tracks changes in early cystic fibrosis. , 2007, American journal of respiratory and critical care medicine.

[46]  J. Mayo,et al.  Progressive damage on high resolution computed tomography despite stable lung function in cystic fibrosis , 2004, European Respiratory Journal.

[47]  Marleen de Bruijne,et al.  Objective airway artery dimensions compared to CT scoring methods assessing structural cystic fibrosis lung disease. , 2017, Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society.

[48]  K. McCoy,et al.  Computed tomography correlates with improvement with ivacaftor in cystic fibrosis patients with G551D mutation. , 2015, Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society.