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. Author manuscript; available in PMC: 2023 Apr 9.
Published in final edited form as: Int Forum Allergy Rhinol. 2022 Jan 10;12(9):1148–1183. doi: 10.1002/alr.22953

Table VII:

Summary of evidence on CFTR modulators

Study Year Study
Design		 LOE Number
of
Subjects		 Study
Groups		 Intervention Primary Endpoint Conclusion
Hayes47 2014 Case report 5 1 CF CRS ivacaftor

  1. CT sinus

  2. Sinonasal symptoms

 Resolution of sinonasal symptoms with radiographic improvement.
Vreede48 2015 Case report 5 1 CF CRS ivacaftor

  1. CT sinus

  2. Sinonasal symptoms

 Improvement in sinonasal symptoms and radiographic severity.
Sheikh51 2015 Case-series 4 12 CF ivacaftor

  1. CT Sinus rating: normal - severe

 Decrease in radiographic severity of sinus disease in all patients.
Chang49 2015 Case report 5 1 CF CRS ivacaftor

  1. CT sinus

  2. Sinonasal symptoms

  3. Nasal voltage/pH

  4. Airway surface liquid viscosity

 Resolved sinonasal symptoms and resolution of radiographic sinus disease. Increased airway surface pH and decreased viscosity.
McCormick52 2019 Prospective cohort 3 153 CF CRS ivacaftor

  1. SNOT-20

 Statistically significant improvement in SNOT-20 score, but less than the MCID. The greatest impact was found on the rhinologic, psychologic, and sleep domains.
Douglas54 2020 Case Series 4 25 CF elexacaftor/tezacaftor/ivacaftor

  1. SNOT-22

 Significant improvement in SNOT-22 exceeding the MCID.
Gostelie53 2020 Case Series 4 8 CF CRS ivacaftor

  1. Modified Lund-Mackay score

  2. Sinonasal symptoms

  3. Nasal nitric oxide

  4. Nasal Endoscopy

 Significant improvement in radiographic severity of CRS and nitric oxide levels. After 2 months, sinonasal symptoms and endoscopic severity improved.
Pallin50 2020 Case report 5 1 CF CRS ivacaftor

  1. SNOT-22

  2. CFQ-R

 Improvement in SNOT-22 and CFQ-R respiratory domain scores that exceed MCID.
Southern58 2020 Systematic review of RCTs 1 2959 CF CFTR corrector monotherapy, dual therapy, or triple therapy

  1. Quality of life

  2. FEV-1

  3. Pulmonary exacerbations

  4. Adverse events

 CFTR corrector dual and triple therapy was associated with improved quality of life and FEV-1 with fewer pulmonary exacerbations. No difference in sinonasal or other adverse events compared with the placebo group.
DiMango55* 2021 Prospective cohort 3 43 CF elexacaftor/tezacaftor/ivacaftor

  1. SNOT-22

  2. CFQ-R

 Significant improvement in SNOT-22 and respiratory domain of the CFQ-R exceeding the MCID.
DiMango56* 2021 Prospective cohort 3 43 CF elexacaftor/tezacaftor/ivacaftor

  1. SNOT-22

  2. CFQ-R

  3. Correlation between SNOT-22 + CFQ-R

 Correlation between the extra-nasal domain of SNOT-22 with the respiratory domain of CFQ-R.
Beswick57 2021 Prospective cohort 3 25 CF CRS elexacaftor/tezacaftor/ivacaftor

  1. Sinus CT opacification

  2. SNOT-22

  3. Health Utility Value

  4. Presenteesim, Absenteeism, Activity Impairment

 Improvements in sinus CT opacification via machine learning analysis and productivity loss; clinically meaningful improvements in sinonasal QOL and health utility
*

same patient population

CF: cystic fibrosis; CFQ-R: Cystic Fibrosis Questionnaire–Revised; CRS: chronic rhinosinusitis; CT – computed tomography; FEV-1: forced expiratory volume in one second; MCID – minimal clinically important difference; RCT: randomized control trial; SNOT-20: 20 item Sino-Nasal Outcome Test; SNOT-22: 22 item Sino-Nasal Outcome Test