Peter van Mourik

134 Chapter 6 Table 1: Clinical characteristics at baseline and after initiation of lumacaftor–ivacaftor Clinical characteristic Baseline mean ± SD (range) or n (%) Lumacaftor-ivacaftor therapy mean ± SD (range) Mean change ± SD (range) p-value Number of patients 21 Age (years) 20.4 ± 7.9 (12.0 - 46.0) 20.7 ± 7.9 (12.3 - 46.2) 0.3 ± 0.1 (0.2 - 0.4) Sex (female) 8 (38%) Genotype F508del/F508del 21 (100%) Pancreatic insufficiency 21 (100%) Sweat chloride (mmol/l) 88.4 ± 9.2 (69.5 - 109.5) 72.4 ± 13.7 (41.0 - 93.5) -16.0 ± 11.6 (-37.0 - 8.0) *<0.001 NPD total chloride response (mV) 0.0 ± 3.5 (-6.8 - 11.6) -0.9 ± 2.2 (-6.3 - 2.6) -0.9 ± 4.3 (-13.4 - 5.7) 0.987 ICM cAMP response (µA/cm²) -5.9 ± 6.6 (-27.5 - 2.4) 26.6 ± 15.7 (3.1 - 68.2) 32.5 ± 16.7 (13.5 - 73.0) *<0.001 BMI (kg/m²) 19.4 ± 3.2 (15.4 - 26.4) 19.7 ± 3.2 (15.6 - 28.1) 0.3 ± 0.7 (-1.1 - 1.7) 0.287 FEV 1 absolut (L) 2.21 ± 0.68 (0.92 - 3.55) 2.26 ± 0.67 (0.86 - 3.51) 0.05 ± 0.18 (-0.34 - 0.38) 0.987 ppFEV 65.2 ± 19.4 (26.9 - 98.4) 65.4 ± 18.4 (25.2 - 93.8) 0.2 ± 6.0 (-15.3 - 10.0) 0.987 ppVC max 81.3 ± 15.1 (44.0 - 100.6) 81.9 ± 14.5 (39.5 - 101.2) 0.5 ± 5.5 (-6.6 - 13.8) 0.987 ppFEV 1 /Vcmax 83.1 ± 12.4 (51.1 - 103.3) 82.6 ± 12.1 (48.4 - 105.9) -0.5 ± 4.4 (-10.5 - 5.8) 0.987 ppMEF 25 35.2 ± 23.2 (9.0 - 86.0) 35.2 ± 20.7 (9.7 - 82.0) 0.0 ± 11.0 (-29.8 - 18.7) 0.987 Definition of abbreviations: NPD: nasal potential difference; ICM = intestinal current measurement; BMI = body mass index; FEV1 = forced expiratory flow in one second; pp = percent predicted; VC max  = maximum vital capacity; MEF 25  = mean expiratory flow at 25% of capacity. Statistical analyses were performed with paired Student’s t-test for sweat chloride, NPD total chloride response, BMI, FEV1 absolut, ppFEV1, ppVCmax, ppFEV1 /Vcmax, ppMEF25 and Wilcoxon signed rank test for ICM cAMP response. P -values were adjusted for multiple comparisons with the Benjamini–Hochberg procedure.