Short-term effects of Lumacaftor/Ivacaftor (Orkambi™) on exertional symptoms, exercise performance, and ventilatory responses in adults with cystic fibrosis

Respir Res. 2020 Jun 1;21(1):135. doi: 10.1186/s12931-020-01406-z.

Abstract

Rationale: Lumacaftor/ivacaftor (LUM/IVA) modestly improves lung function following 1 month of treatment but it is unknown if this translates into improvements in exercise endurance and exertional symptoms.

Methods: Adult CF participants completed a symptom-limited constant load cycling test with simultaneous assessments of dyspnea and leg discomfort ratings pre- and 1 month post-initiation of LUM/IVA.

Results: Endurance time, exertional dyspnea and leg discomfort ratings at submaximal exercise did not change significantly. There was a significant inverse correlation between changes in leg discomfort and endurance time (r = - 0.88; p = 0.009) following 1-month of LUM/IVA.

Conclusions: Overall, 1-month of LUM/IVA did not increase endurance time or modify exertional dyspnea or leg discomfort ratings. However, individuals who experienced a reduction in leg discomfort following LUM/IVA had an improvement in endurance time. Future studies with a larger sample size are needed to verify these findings and to assess the long-term effects of LUM/IVA on exercise outcomes.

Trial registration: ClinicalTrials.gov Identifier: NCT02821130. Registered July 1, 2016.

Publication types

  • Clinical Trial
  • Letter
  • Observational Study

MeSH terms

  • Adult
  • Aminophenols / administration & dosage*
  • Aminopyridines / administration & dosage*
  • Benzodioxoles / administration & dosage*
  • Cystic Fibrosis / diagnosis
  • Cystic Fibrosis / drug therapy*
  • Cystic Fibrosis / physiopathology
  • Drug Combinations
  • Exercise Test / drug effects*
  • Exercise Test / methods
  • Female
  • Forced Expiratory Volume / drug effects*
  • Forced Expiratory Volume / physiology
  • Humans
  • Male
  • Physical Exertion / drug effects*
  • Physical Exertion / physiology
  • Pulmonary Ventilation / drug effects*
  • Pulmonary Ventilation / physiology
  • Quinolones / administration & dosage*
  • Treatment Outcome
  • Young Adult

Substances

  • Aminophenols
  • Aminopyridines
  • Benzodioxoles
  • Drug Combinations
  • Quinolones
  • lumacaftor, ivacaftor drug combination
  • ivacaftor
  • lumacaftor

Associated data

  • ClinicalTrials.gov/NCT02821130