Study design (if review, criteria of inclusion for studies)
Phase 3 randomized double blind controlled, trial
238 people with cystic fibrosis (CF) and at least one nonsense mutation (nmCF)
Ataluren. 48 weeks
Independent scoring systems to characterize structural lung disease. Perth-Rotterdam Annotated Grid Morphometric Analysis for CF (PRAGMA-CF) scoring systems. CF-CT and PRAGMA-CF subscores were expressed as %maximal score and %total lung volume, respectively.
PRAGMA-CF subscores %Disease (p = 0.008) and %Mucus Plugging (p = 0.029) progressed over 48 weeks. CF-CT subscores did not show progression. There was no difference in progression of structural lung disease between treatment arm and placebo independent of tobramycin use.
PRAGMA-CF Chest CT scores can be used as an outcome measure to study the effect of potential disease modifying drugs in CF on lung structure.