Peter van Mourik

133 Comparing in vitro and in vivo effects of lumacaftor-ivacaftor Figure 1: Correlation of effects of lumacaftor–ivacaftor treatment on organoid swelling with effects on in vivo biomarkers of CFTR function and ppFEV1 in F508del-homozygous patients with cystic fibrosis. (A–D) Relationship between forskolin-induced swelling (FIS) of intestinal organoids and absolute change in sweat chloride concentration (A), nasal potential difference total chloride response (zero chloride plus isoproterenol solutions) (B), intestinal current measurement cAMP response (C), and absolute change in ppFEV1 (D). AUC = area under the curve; ICM = intestinal current measurement; iso = isoproterenol; NPD = nasal potential difference. NPD studies were performed in 19 of 21 patients with CF. For panel A, B and D Pearson and for panel C Spearman correlation coefficient r and respective P-values adjusted for multiple comparisons with the Benjamini–Hochberg procedure are provided. 6