Figure 5
Treatment efficacy on primary end point in study population. (a) Mean change in sweat chloride concentrations (mmol/liter) from baseline during the whole study period in treated (blue line) (weeks 8, 12, 20, 24 of treatment schedule) and untreated (red line) groups (week 12, 24 of observation). In treated cohorts, weeks 16, 24 and 28 (italic) indicate the corresponding time points in the subgroup of patients who received combination treatment for 8 weeks. Data from all cohorts of treated patients at the end of each treatment period are pooled for the analysis. Bars are 95% CI. #P<0.0001. (b–c) Mean values of CFTR function (b) and CFTR band C protein (c) expressed as percent of non-CF control subjects in treated (left) and untreated (right) patients during the whole study period. Bars are 95% CI. #P <0.001 versus baseline. (d) Scatter plot of nasal and sweat measure of CFTR function. The red line represents the estimated linear regression model, with a regression coefficient of −1.1 and its associated P-value of 0.0046. Wk=week. (e) Individual sweat chloride values (mmol/liter) in all study cohorts. Black lines indicate mean values in different cohorts of treated patients (weeks 0, 8, 12, 20, 24 of treatment schedule). Red lines indicate mean values of untreated patients (cohort 4) (week 0, 12, 24 of observation). Red dashed lines indicate the pathological sweat test cutoff value
