1. Ratjen, F, Bell, SC, Rowe, SM, et al. Cystic fibrosis. Nat Rev Dis Primers 2015; 1: 15010.
Google Scholar | Crossref | Medline2. Zolin, A, Orenti, A, Naehrlich, L, et al. ECFS Patient Registry annual data report 2017, https://www.ecfs.eu/projects/ecfs-patient-registry/annual-reports (accessed 5 May 2020).
Google Scholar3. Lukacs, GL, Mohamed, A, Kartner, N, et al. Conformational maturation of CFTR but not its mutant counterpart (delta F508) occurs in the endoplasmic reticulum and requires ATP. EMBO J 1994; 13: 6076–6086.
Google Scholar | Crossref | Medline4. van Goor, F, Straley, KS, Cao, D, et al. Rescue of ΔF508-CFTR trafficking and gating in human cystic fibrosis airway primary cultures by small molecules. Am J Physiol Lung Cell Mol Physiol 2006; 290: L1117–L1130.
Google Scholar | Crossref | Medline5. Deeks, ED . Lumacaftor/ivacaftor: a review in cystic fibrosis. Drugs 2016; 76: 1191–1201.
Google Scholar | Crossref | Medline6. Wainwright, CE, Elborn, JS, Ramsey, BW, et al. Lumacaftor-ivacaftor in patients with cystic fibrosis homozygous for phe508del CFTR. N Engl J Med 2015; 373: 220–231.
Google Scholar | Crossref | Medline | ISI7. Konstan, MW, McKone, EF, Moss, RB, 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. Lancet Respir Med 2017; 5: 107–118.
Google Scholar | Crossref | Medline8. Masson, A, Schneider-Futschik, EK, Baatallah, N, et al. Predictive factors for lumacaftor/ivacaftor clinical response. J Cyst Fibros 2019; 18: 368–374.
Google Scholar | Crossref | Medline9. de Backer, JW, Vos, W, Vinchurkar, S, et al. Validation of computational fluid dynamics in CT-based airway models with SPECT/CT. Radiology 2010; 257: 854–862.
Google Scholar | Crossref | Medline | ISI10. Tahir, BA, Van Holsbeke, C, Ireland, RH, et al. Comparison of CT-based lobar ventilation with 3He MR imaging ventilation measurements. Radiology 2016; 278: 585–592.
Google Scholar | Crossref | Medline | ISI11. De Backer, JW, Vos, WG, Gorlé, CD, et al. Flow analyses in the lower airways: patient-specific model and boundary conditions. Med Eng Phys 2008; 30: 872–879.
Google Scholar | Crossref | Medline12. De Backer, LA, Vos, W, De Backer, J, et al. The acute effect of budesonide/formoterol in COPD: a multi-slice computed tomography and lung function study. Eur Respir J 2012; 40: 298–305.
Google Scholar | Crossref | Medline | ISI13. De Backer, LA, Vos, WG, Salgado, R, et al. Functional imaging using computer methods to compare the effect of salbutamol and ipratropium bromide in patient-specific airway models of COPD. Int J Chron Obstruct Pulmon Dis 2011; 6: 637–646.
Google Scholar | Crossref | Medline14. Vos, W, De Backer, J, Poli, G, et al. Novel functional imaging of changes in small airways of patients treated with extrafine beclomethasone/formoterol. Respiration 2013; 86: 393–401.
Google Scholar | Crossref | Medline | ISI15. Bos, AC, Van Holsbeke, C, De Backer, JW, et al. Patient-specific modeling of regional antibiotic concentration levels in airways of patients with cystic fibrosis: are we dosing high enough? PLoS ONE 2015; 10: e0118454.
Google Scholar | Crossref16. Klemmer, A, Krämer, I, Kamin, W. Physicochemical compatibility and stability of nebulizable drug admixtures containing dornase alfa and tobramycin. Pulm Pharmacol Ther 2014; 28: 53–59.
Google Scholar | Crossref | Medline17. Lins, M, Vandevenne, J, Thillai, M, et al. Assessment of small pulmonary blood vessels in COVID-19 patients using HRCT. Acad Radiol 2020; 27: 1449–1455.
Google Scholar | Crossref | Medline18. Brody, AS, Kosorok, MR, Li, Z, et al. Reproducibility of a scoring system for computed tomography scanning in cystic fibrosis. J Thorac Imaging 2006; 21: 14–21.
Google Scholar | Crossref | Medline19. Wainwright, CE, Vidmar, S, Armstrong, DS, et al. Effect of bronchoalveolar lavage-directed therapy on Pseudomonas aeruginosa infection and structural lung injury in children with cystic fibrosis: a randomized trial. JAMA 2011; 306: 163–171.
Google Scholar | Medline | ISI20. Koo, TK, Li, MY. A guideline of selecting and reporting intraclass correlation coefficients for reliability research. J Chiropr Med 2016; 15: 155–163.
Google Scholar | Crossref | Medline | ISI21. Rosenow, T, Oudraad, MCJ, Murray, CP, et al. PRAGMA-CF . A quantitative structural lung disease computed tomography outcome in young children with cystic fibrosis. Am J Respir Crit Care Med 2015; 191: 1158–1165.
Google Scholar | Crossref | Medline22. Arnaud, F, Stremler-Le Bel, N, Reynaud-Gaubert, M, et al. Computed tomographic changes in patients with cystic fibrosis treated by combination therapy with lumacaftor and ivacaftor. J Clin Med 2021; 10: 1999.
Google Scholar | Crossref23. Sheikh, SI, Long, FR, McCoy, KS, et al. Computed tomography correlates with improvement with ivacaftor in cystic fibrosis patients with G551D mutation. J Cyst Fibros 2015; 14: 84–89.
Google Scholar | Crossref | Medline24. Gramegna, A, Contarini, M, Aliberti, S, et al. From ivacaftor to triple combination: a systematic review of efficacy and safety of CFTR modulators in people with cystic fibrosis. Int J Mol Sci 2020; 21: 5882.
Google Scholar | Crossref25. Petousi, N, Talbot, NP, Pavord, I, et al. Measuring lung function in airways diseases: current and emerging techniques. Thorax 2019; 74: 797–805.
Google Scholar | Crossref | Medline26. Aaron, SD, Stephenson, AL, Cameron, DW, et al. A statistical model to predict one-year risk of death in patients with cystic fibrosis. J Clin Epidemiol 2015; 68: 1336–1345.
Google Scholar | Crossref | Medline27. Liou, TG, Adler, FR, Fitzsimmons, SC, et al. Predictive 5-year survivorship model of cystic fibrosis. Am J Epidemiol 2001; 153: 345–352.
Google Scholar | Crossref | Medline | ISI28. Tiddens, HAWM, Puderbach, M, Venegas, JG, et al. Novel outcome measures for clinical trials in cystic fibrosis. Pediatr Pulmonol 2015; 50: 302–315.
Google Scholar | Crossref | Medline29. Goralski, JL, Stewart, NJ, Woods, JC. Novel imaging techniques for cystic fibrosis lung disease. Pediatr Pulmonol 2021; 56(Suppl. 1): S40–S54.
Google Scholar | Crossref | Medline30. Kent, L, Reix, P, Innes, JA, et al. Lung clearance index: evidence for use in clinical trials in cystic fibrosis. J Cyst Fibros 2014; 13: 123–138.
Google Scholar | Crossref | Medline31. Stahl, M, Wielpütz, MO, Graeber, SY, et al. Comparison of lung clearance index and magnetic resonance imaging for assessment of lung disease in children with cystic fibrosis. Am J Respir Crit Care Med 2017; 195: 349–359.
Google Scholar | Medline32. Graeber, SY, Boutin, S, Wielpütz, MO, et al. Effects of lumacaftor-ivacaftor on lung clearance index, magnetic resonance imaging and airway microbiome in Phe508del homozygous patients with cystic fibrosis. Ann Am Thorac Soc 2021; 18: 971–980.
Google Scholar | Crossref | Medline33. Shaw, M, Khan, U, Clancy, JP, et al. Changes in LCI in F508del/F508del patients treated with lumacaftor/ivacaftor: results from the prospect study. J Cyst Fibros 2020; 19: 931–933.
Google Scholar | Crossref | Medline34. Reix, P, Tatopoulos, A, Ioan, I, et al. Real-world assessment of LCI following lumacaftor-ivacaftor initiation in adolescents and adults with cystic fibrosis. J Cyst Fibros. Epub ahead of print 25 June 2021. DOI: 10.1016/j.jcf.2021.06.002.
Google Scholar | Crossref35. Horsley, A, Wild, JM. Ventilation heterogeneity and the benefits and challenges of multiple breath washout testing in patients with cystic fibrosis. Paediatr Respir Rev 2015; 16(Suppl. 1): 15–18.
Google Scholar | Medline36. Calder, AD, Bush, A, Brody, AS, et al. Scoring of chest CT in children with cystic fibrosis: state of the art. Pediatr Radiol 2014; 44: 1496–1506.
Google Scholar | Crossref | Medline37. Kuo, W, de Bruijne, M, Petersen, J, et al. Diagnosis of bronchiectasis and airway wall thickening in children with cystic fibrosis: objective airway-artery quantification. Eur Radiol 2017; 27: 4680–4689.
Google Scholar | Crossref | Medline38. Eichinger, M, Optazaite, DE, Kopp-Schneider, A, et al. Morphologic and functional scoring of cystic fibrosis lung disease using MRI. Eur J Radiol 2012; 81: 1321–1329.
Google Scholar | Crossref | Medline | ISI39. De Backer, J, Vos, W, Vinchurkar, S, et al. The effects of extrafine beclometasone/formoterol (BDP/F) on lung function, dyspnea, hyperinflation, and airway geometry in COPD patients: novel insight using functional respiratory imaging. J Aerosol Med Pulm Drug Deliv 2015; 28: 88–99.
Google Scholar | Crossref | Medline | ISI40. Montgomery, AB, Abuan, T, Yeager, MA. Regulatory aspects of phase 3 endpoints for new inhaled antibiotics for cystic fibrosis patients with chronic pseudomonas aeruginosa infections. J Aerosol Med Pulm Drug Deliv 2012; 25: 198–203.
Google Scholar | Crossref | Medline41. Owens, CM, Aurora, P, Stanojevic, S, et al. Lung clearance index and HRCT are complementary markers of lung abnormalities in young children with CF. Thorax 2011; 66: 481–488.
Google Scholar | Crossref | Medline42. Gustafsson, PM, De Jong, PA, Tiddens, HA, et al. Multiple-breath inert gas washout and spirometry versus structural lung disease in cystic fibrosis. Thorax 2008; 63: 129–134.
Google Scholar | Crossref | Medline | ISI43. Wark, PAB, Cookson, K, Thiruchelvam, T, et al. Lumacaftor/ ivacaftor improves exercise tolerance in patients with Cystic Fibrosis and severe airflow obstruction. BMC Pulm Med 2019; 19: 106.
Google Scholar | Crossref | Medline44. Ratjen, F, Hug, C, Marigowda, G, 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. Lancet Respir Med 2017; 5: 557–567.
Google Scholar | Crossref | Medline45. Heijerman, HGM, McKone, EF, Downey, DG, et al. Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial. Lancet 2019; 394: 1940–1948.
Google Scholar46. Middleton, PG, Mall, MA, Dřevínek, P, et al. Elexacaftor–tezacaftor–ivacaftor for cystic fibrosis with a single Phe508del allele. N Engl J Med 2019; 381: 1809–1819.
Google Scholar | Crossref | Medline47. Griese, M, Costa, S, Linnemann, RW, et al. Safety and efficacy of elexacaftor/tezacaftor/ivacaftor for 24 weeks or longer in people with cystic fibrosis and one or more F508del alleles: interim results of an open-label phase 3 clinical trial. Am J Respir Crit Care Med 2021; 203: 381–385.
Google Scholar | Crossref | Medline