Peter van Mourik

155 Personalized treatment of CF using rectal organoids 18. Zainal Abidin, N., Haq, I. J., Gardner, A. I. & Brodlie, M. Ataluren in cystic fibrosis: development, clinical studies and where are we now? Expert Opin. Pharmacother. 1–9 (2017) doi:10.1080/14656566.2017.1359255. 19. de Winter-de Groot, K. M. et al. Stratifying infants with cystic fibrosis for disease severity using intestinal organoid swelling as a biomarker of CFTR function. Eur. Respir. J. 52 , 1702529 (2018). 20. Sato, T. et al. Long-term expansion of epithelial organoids from human colon, adenoma, adenocarcinoma, and Barrett’s epithelium. Gastroenterology 141 , 1762–72 (2011). 21. Boj, S. F. et al. Forskolin-induced Swelling in Intestinal Organoids: An <em>In Vitro</em> Assay for Assessing Drug Response in Cystic Fibrosis Patients. J. Vis. Exp. (2017) doi:10.3791/55159. 22. Nederlandse CF Stichting. Nederlandse Cystic Fibrosis Registratie 2015. (2016). 23. Standardization of Spirometry, 1994 Update. American Thoracic Society. Am. J. Respir. Crit. Care Med. 152 , 1107–1136 (1995). 24. Beydon, N. et al. American Thoracic Society Documents An Official American Thoracic Society / European Respiratory Society Statement : Pulmonary Function Testing in Preschool Children. 175 , 1304–1345 (2007). 25. YOUDEN, W. J. Index for rating diagnostic tests. Cancer 3 , 32–5 (1950). 26. Leeflang, M. M. G., Moons, K. G. M., Reitsma, J. B. & Zwinderman, A. H. Bias in sensitivity and specificity caused by data-driven selection of optimal cutoff values: mechanisms, magnitude, and solutions. Clin. Chem. 54 , 729–37 (2008). 27. Lorenz, D. J., Datta, S. & Harkema, S. J. Marginal association measures for clustered data. Stat. Med. 30 , 3181–3191 (2011). 28. Obuchowski, N. A. Nonparametric analysis of clustered ROC curve data. Biometrics 53 , 567–78 (1997). 29. Ramsey, B. W. et al. A CFTR Potentiator in Patients with Cystic Fibrosis and the G551D Mutation. N. Engl. J. Med. 365 , 1663–1672 (2011). 30. Wainwright, C. E. et al. Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR . N. Engl. J. Med. 150517100015004 (2015) doi:10.1056/NEJMoa1409547. 31. Wood, M. E. et al. Ivacaftor in severe cystic fibrosis lung disease and a G551D mutation. Respirol. case reports 1 , 52–4 (2013). 32. Taylor-Cousar, J. L. et al. Lumacaftor/ivacaftor in patients with cystic fibrosis and advanced lung disease homozygous for F508del-CFTR. J. Cyst. Fibros. (2017) doi:10.1016/j.jcf.2017.09.012. 33. Seliger, V. I., Rodman, D., Van Goor, F., Schmelz, A. & Mueller, P. The predictive potential of the sweat chloride test in cystic fibrosis patients with the G551D mutation. J. Cyst. Fibros. 12 , 706–713 (2013). 34. Metz, C. E. Basic principles of ROC analysis. Semin. Nucl. Med. 8 , 283–98 (1978). 7

RkJQdWJsaXNoZXIy ODAyMDc0