Effects of highly effective modulator therapy on the dynamics of the respiratory mucosal environment and inflammatory response in cystic fibrosis

Pediatr Pulmonol. 2024 May;59(5):1266-1273. doi: 10.1002/ppul.26898. Epub 2024 Feb 14.

Abstract

Background: While the widespread initiation of elexacaftor/tezacaftor/ivacaftor (ETI) has led to dramatic clinical improvements among persons with cystic fibrosis (pwCF), little is known about how ETI affects the respiratory mucosal inflammatory and physiochemical environment, or how these changes relate to lung function.

Methods: We performed a prospective, longitudinal study of adults with CF and chronic rhinosinusitis (CF-CRS) followed at our CF center (n = 18). Endoscopic upper respiratory tract (paranasal sinus) aspirates from multiple visit dates, both pre- and post-ETI initiation, were collected and tested for cytokines, metals, pH, and lactate levels. Generalized estimating equations were used to identify relationships between ETI and upper respiratory tract (URT) biomarker levels, and between URT biomarkers and lung function or clinical sinus parameters.

Results: ETI was associated with decreased upper respiratory mucosal cytokines B-cell activating factor (BAFF), IL-12p40, IL-32, IL-8, IL-22 and soluble tumor necrosis factor-1 (sTNFR1), and an increase in a proliferation-inducing ligand (APRIL) and IL-19. ETI was also associated with decreased URT levels of copper, manganese, and zinc. In turn, lower URT levels of BAFF, IL-8, lactate, and potassium were each associated with ~1.5% to 4.3% improved forced expiratory volume in 1 s (FEV1), while higher levels of IFNγ, iron, and selenium were associated with ~2% to 10% higher FEV1.

Conclusions: Our observations suggest a dampening of inflammatory signals and restriction in microbial nutrients in the upper respiratory tract with ETI. These findings improve our understanding of how ETI impacts the mucosal environment in the respiratory tract, and may give insight into the improved infectious and inflammatory status and the resulting clinical improvements seen in pwCF.

Keywords: ETI; chronic rhinosinusitis; cystic fibrosis; highly effective modulator therapy.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Aminophenols* / therapeutic use
  • Benzodioxoles* / therapeutic use
  • Biomarkers / analysis
  • Chronic Disease
  • Cystic Fibrosis* / complications
  • Cystic Fibrosis* / drug therapy
  • Cystic Fibrosis* / physiopathology
  • Cytokines
  • Drug Combinations
  • Female
  • Humans
  • Indoles / therapeutic use
  • Inflammation / drug therapy
  • Longitudinal Studies
  • Male
  • Prospective Studies
  • Pyridines / therapeutic use
  • Quinolones* / therapeutic use
  • Respiratory Mucosa* / drug effects
  • Rhinitis / drug therapy
  • Sinusitis / drug therapy
  • Young Adult

Substances

  • Aminophenols
  • Quinolones
  • Benzodioxoles
  • Cytokines
  • Indoles
  • Drug Combinations
  • Pyridines
  • Biomarkers
  • ivacaftor
  • tezacaftor