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The effect of Ivacaftor in adolescents with cystic fibrosis (G551D mutation): an exercise physiology perspective

Research output: Contribution to journalArticlepeer-review

  • Dr Zoe Saynor
  • Alan Robert Barker
  • Patrick John Oades
  • Craig Anthony Williams
The purpose of this report was to evaluate the influence of 12 weeks of ivacaftor treatment on the aerobic function of 2 teenage patients with cystic fibrosis (CF; ΔF508/G551D) using a maximal cardiopulmonary exercise test.

Summary of Key Points:
One patient, with relatively mild disease, demonstrated no clinically meaningful changes in maximal oxygen uptake (o2max). However, in the second case, with more established lung disease on imaging, o2max improved by approximately 30%, an improvement out of proportion with early lung function changes. This improvement resulted from increased muscle oxygen delivery and extraction.

Statement of Conclusions:
Cardiopulmonary exercise testing can monitor the extent and cause(s) of change following interventions such as ivacaftor, with the potential to identify functional changes independent from spirometry indices.

Recommendations for Clinical Practice:
Cardiopulmonary exercise testing represents an important and comprehensive clinical assessment tool, and its use as an outcome measure in the functional assessment of patients with CF is encouraged.
Original languageEnglish
Pages (from-to)454-461
JournalPediatric Physical Therapy
Issue number4
Publication statusPublished - Dec 2014
Externally publishedYes


  • The effect of Ivacaftor

    Rights statement: This is a non-final version of an article published in final form in: Saynor, Zoe Louise MSc; Barker, Alan Robert PhD; Oades, Patrick John FRCPCH; Williams, Craig Anthony PhD, The Effect of Ivacaftor in Adolescents With Cystic Fibrosis (G551D Mutation): An Exercise Physiology Perspective, Pediatric Physical Therapy. 26(4):454-461, Winter 2014, DOI: 10.1097/PEP.0000000000000086

    Accepted author manuscript (Post-print), 474 KB, PDF document

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