Effect of ivacaftor on mucociliary clearance and clinical outcomes in cystic fibrosis patients with G551D-CFTR

Scott H. Donaldson, Beth L. Laube, Timothy E. Corcoran, Pradeep Bhambhvani, Kirby Zeman, Agathe Ceppe, Pamela L. Zeitlin, Peter J. Mogayzel, Michael Boyle, Landon W. Locke, Michael M. Myerburg, Joseph M. Pilewski, Brian Flanagan, Steven M. Rowe, William D. Bennett

Research output: Contribution to journalArticlepeer-review

18 Scopus citations


BACKGROUND: The ability to restore cystic fibrosis transmembrane regulator (CFTR) function with effective small molecule modulators in patients with cystic fibrosis provides an opportunity to study relationships between CFTR ion channel function, organ level physiology, and clinical outcomes. METHODS: We performed a multisite, prospective, observational study of ivacaftor, prescribed in patients with the G551D-CFTR mutation. Measurements of lung mucociliary clearance (MCC) were performed before and after treatment initiation (1 and 3 months), in parallel with clinical outcome measures. RESULTS: Marked acceleration in whole lung, central lung, and peripheral lung MCC was observed 1 month after beginning ivacaftor and was sustained at 3 months. Improvements in MCC correlated with improvements in forced expiratory volume in the first second (FEV1) but not sweat chloride or symptom scores. CONCLUSIONS: Restoration of CFTR activity with ivacaftor led to significant improvements in MCC. This physiologic assessment provides a means to characterize future CFTR modulator therapies and may help to predict improvements in lung function. TRIAL REGISTRATION: ClinicialTrials.gov, NCT01521338. FUNDING: CFF Therapeutics (GOAL11K1).

Original languageEnglish (US)
JournalJCI Insight
Issue number24
StatePublished - Dec 20 2018


  • Chloride channels
  • Diagnostic imaging
  • Pulmonology

ASJC Scopus subject areas

  • General Medicine


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