Justin McCormick1, Do-Yeon Cho1, Brooks Lampkin2, Joshua Richman1, Heather Hathorne3, Steven M Rowe4,5, Bradford A Woodworth1. 1. Department of Otolaryngology-Head and Neck Surgery, University of Alabama at Birmingham, Birmingham, AL. 2. College of Medicine, University of South Alabama, Mobile, AL. 3. Department of Pediatrics, University of Alabama at Birmingham, Birmingham, AL. 4. Department of Medicine, University of Alabama at Birmingham, Birmingham, AL. 5. Gregory Fleming James Cystic Fibrosis Research Center, Birmingham, AL.
Abstract
BACKGROUND: Ivacaftor is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator that improves pulmonary function in cystic fibrosis (CF) patients with at least 1 copy of the G551D CFTR mutation. The purpose of this study is to evaluate the impact of ivacaftor on chronic rhinosinusitis (CRS) symptoms in this population. METHODS: The G551D Observational (GOAL) study was a multicenter prospective cohort study enrolling CF patients ≥6 years with at least 1 G551D mutation. Subjects were provided 20-item Sino-Nasal Outcome Test (SNOT-20) questionnaires prior to ivacaftor therapy and at 1, 3, and 6 months afterward. The impact on rhinologic (R), psychological (P), sleep (S), and ear/facial (E) quality of life (QOL) domains was evaluated separately. RESULTS: Of 153 subjects, 129 (84%) completed all questionnaires. Typical baseline symptom burden was low (75% with scores <1) and degree of improvement (ie, reduced scores) was greater with higher baseline scores. SNOT-20 decreased, reflecting improvement, at all follow-up intervals (1 month: [mean change ± standard deviation] -0.25 ± 0.53, p < 0.01; 3 months; -0.29 ± 0.58, p < 0.01; 6 months: -0.21 ± 0.58, p = 0.02), but less than the prespecified minimal clinically important difference (0.8). Significant improvement was observed at 1, 3, and 6 months in the R domain (1 month: -0.24, p < 0.01; 3 months: -0.34, p < 0.01; 6 months: -0.25, p < 0.01) and P domain (1 month: -0.25, p < 0.01; 3 months: -0.32, p < 0.01; 6 months: -0.26, p < 0.01), and 1 and 3 months in the S domain (1 months: -0.35, p < 0.01; 3 months: -0.32, p < 0.01; 6 months: -0.18, p = 0.07). There was no improvement in the E domain at any time point. CONCLUSION: Ivacaftor improves QOL in the R, P, and S domains in G551D CF patients, although QOL instruments validated for CRS may not translate well to CF CRS patients because symptom burden was surprisingly low.
BACKGROUND:Ivacaftor is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator that improves pulmonary function in cystic fibrosis (CF) patients with at least 1 copy of the G551DCFTR mutation. The purpose of this study is to evaluate the impact of ivacaftor on chronic rhinosinusitis (CRS) symptoms in this population. METHODS: The G551D Observational (GOAL) study was a multicenter prospective cohort study enrolling CFpatients ≥6 years with at least 1 G551D mutation. Subjects were provided 20-item Sino-Nasal Outcome Test (SNOT-20) questionnaires prior to ivacaftor therapy and at 1, 3, and 6 months afterward. The impact on rhinologic (R), psychological (P), sleep (S), and ear/facial (E) quality of life (QOL) domains was evaluated separately. RESULTS: Of 153 subjects, 129 (84%) completed all questionnaires. Typical baseline symptom burden was low (75% with scores <1) and degree of improvement (ie, reduced scores) was greater with higher baseline scores. SNOT-20 decreased, reflecting improvement, at all follow-up intervals (1 month: [mean change ± standard deviation] -0.25 ± 0.53, p < 0.01; 3 months; -0.29 ± 0.58, p < 0.01; 6 months: -0.21 ± 0.58, p = 0.02), but less than the prespecified minimal clinically important difference (0.8). Significant improvement was observed at 1, 3, and 6 months in the R domain (1 month: -0.24, p < 0.01; 3 months: -0.34, p < 0.01; 6 months: -0.25, p < 0.01) and P domain (1 month: -0.25, p < 0.01; 3 months: -0.32, p < 0.01; 6 months: -0.26, p < 0.01), and 1 and 3 months in the S domain (1 months: -0.35, p < 0.01; 3 months: -0.32, p < 0.01; 6 months: -0.18, p = 0.07). There was no improvement in the E domain at any time point. CONCLUSION:Ivacaftor improves QOL in the R, P, and S domains in G551DCFpatients, although QOL instruments validated for CRS may not translate well to CF CRSpatients because symptom burden was surprisingly low.
Authors: Do-Yeon Cho; Shaoyan Zhang; Daniel F Skinner; Dong Jin Lim; Catherine Banks; Jessica W Grayson; Guillermo J Tearney; Steven M Rowe; Bradford A Woodworth Journal: Int Forum Allergy Rhinol Date: 2021-10-26 Impact factor: 5.426
Authors: Joel Reiter; Alex Gileles-Hillel; Malena Cohen-Cymberknoh; Dennis Rosen; Eitan Kerem; David Gozal; Erick Forno Journal: Sleep Med Rev Date: 2020-02-19 Impact factor: 11.609
Authors: Zainab Farzal; Kelly M Dean; Satyan B Sreenath; Sarah E Hodge; Brian D Thorp; Charles S Ebert; Adam M Zanation; Brent A Senior; Adam J Kimple Journal: Int Forum Allergy Rhinol Date: 2020-01-17 Impact factor: 3.858
Authors: Saangyoung E Lee; Zainab Farzal; M Leigh Anne Daniels; Brian D Thorp; Adam M Zanation; Brent A Senior; Charles S Ebert; Adam J Kimple Journal: Am J Rhinol Allergy Date: 2020-03-13 Impact factor: 2.467
Authors: Daniel M Beswick; Stephen M Humphries; Connor D Balkissoon; Matthew Strand; Eszter K Vladar; David A Lynch; Jennifer L Taylor-Cousar Journal: Ann Am Thorac Soc Date: 2022-01
Authors: Valentine Sergeev; Frank Y Chou; Grace Y Lam; Christopher Michael Hamilton; Pearce G Wilcox; Bradley S Quon Journal: Ann Am Thorac Soc Date: 2020-02