Fig 1.
Table 1.
Patient characteristics at the start of study of patients with a Start of Study (SOS) and End of Study (EOS) CT which were included in the CT image analysis.
Fig 2.
Stacked bar plots showing the wide spectrum of disease scores found by the CF-CT (A) and PRAGMA-CF scoring system (B). A. This stacked bar plot shows the distribution of the CF-CT %total score scores at start of study (SOS) for 195 patients. Patients are sorted based on the CF-CT %total score. %total score for each patient is subdivided by the subscores %Bronchiectasis, %Mucus Plugging, %Airway Wall Thickening and %Atelectasis. Note the wide distribution of %total score ranging from 0 to 37%. B. This stacked bar plot shows the distribution of the % volume of the lung at start of study (SOS) for 195 patients occupied by structural lung abnormalities as determined by PRAGMA-CF score. Patients are sorted based on the PRAGMA-CF %Disease subscore which is the sum of PRAGMA-CF %Bronchiectasis, %Mucus Plugging, and %Airway Wall Thickening. %Atelectasis (is depicted on top of %disease). Note the wide distribution of %Disease severities ranging from 0 to 35.5%.
Table 2.
This table shows the PRAGMA-CF and CF-CT subscores of CTs at the Start of Study (SOS) (n = 195) and End of Study (EOS) (n = 196).
Fig 3.
SOS (A) and EOS (B) CT scan image analysis results of %Disease subscore: CF-CT score vs. PRAGMA-CF method. This figure shows CF-CT %Disease scored by observer 1 plotted against PRAGMA-CF %Disease scored by observer 2 at the start of study (SOS, A) and at the end of study (EOS, B). The black line represents the regression line. The correlation between PRAGMA-CF and CF-CT for %Disease at SOS and EOS is 0.74 (p = <0.001).
Fig 4.
Change of PRAGMA-CF %BE (A) and %Disease (B) from EOS to SOS per patient. These plots show the change in PRAGMA-CF %BE (A) and PRAGMA-CF %Disease (B) from start of study (SOS) to end of study (EOS). Grey open circles represent the measurements of individual patients and the grey lines are plotted between the measurements of a single patient at SOS and EOS time-point. Black solid line represents the change obtained by the linear mixed model for patients with ataluren and tobramycin treatment. Note that for other subgroups the lines would not be statistically different as there was no significant impact for any of the other investigated confounders. The dashed black lines represent the confidence interval, and the dashed red lines the prediction interval. Note that for PRAGMA-CF %Disease values are presented in the square root scale—which is the scale that was used to analyse the data as the errors for this subscore were not normal distributed. Furthermore, note that due to the large number of patients included in the figure the grey open circles might appear filled.
Table 3.
Results of the linear mixed effects models for the outcome PRAGMA-CF %Mucus plugging and %Disease.