Effect of highly effective modulator treatment on sinonasal symptoms in cystic fibrosis

Emily DiMango; Jonathan Overdevest; Claire Keating; Sarah Fracasso Francis; David Dansky; David Gudis

doi:10.1016/j.jcf.2020.07.002

Effect of highly effective modulator treatment on sinonasal symptoms in cystic fibrosis

J Cyst Fibros. 2021 May;20(3):460-463. doi: 10.1016/j.jcf.2020.07.002. Epub 2020 Jul 18.

Authors

Emily DiMango¹, Jonathan Overdevest², Claire Keating³, Sarah Fracasso Francis⁴, David Dansky⁵, David Gudis⁶

Affiliations

¹ Columbia University Irving Medical Center Department of Medicine, Pulmonary, Allergy and Critical Care, 622 West 168th Street, New York, New York 10032, United States. Electronic address: ead3@cumc.columbia.edu.
² Columbia University Irving Medical Center, Department of Otolaryngology, United States. Electronic address: jo2566@cumc.columbia.edu.
³ Columbia University Irving Medical Center Department of Medicine, Pulmonary, Allergy and Critical Care, 622 West 168th Street, New York, New York 10032, United States. Electronic address: ck2132@cumc.columbia.edu.
⁴ Columbia University Irving Medical Center Department of Medicine, Pulmonary, Allergy and Critical Care, 622 West 168th Street, New York, New York 10032, United States. Electronic address: smf2218@cumc.columbia.edu.
⁵ Columbia University Irving Medical Center Department of Medicine, Pulmonary, Allergy and Critical Care, 622 West 168th Street, New York, New York 10032, United States. Electronic address: dansky.david1@gmail.com.
⁶ Columbia University Irving Medical Center, Department of Otolaryngology, United States. Electronic address: dag62@cumc.columbia.edu.

PMID: 32694034
DOI: 10.1016/j.jcf.2020.07.002

Abstract

Background: Elexacaftor-tezacaftor-ivacaftor is a highly effective modulator for cystic fibrosis (CF) patients homozygous or heterozygous for F508del. Effects of the drug on sinonasal symptoms have not been studied.

Methods: Adult participants were prospectively evaluated at baseline and after three months of treatment using validated questionnaires assessing sinonasal symptoms (SNOT-22) and CF-related quality of life (CFQ-R).

Results: Forty-three participants completed the study; 23 were taking other CF transmembrane conductance (CFTR) modulators at the time of study participation. There was a significant improvement in mean SNOT-22 from 34.8 (29.4-40, 95% confidence interval) to 24.4 (19.9-29.0) (p = 0.000003) and in the Respiratory domain of the CFQR from 60.6 (57.1-64.1) to 83.3 (79.4-87.2) (p = 0.0000002), both achieving a minimal clinically important difference. Patients previously taking CFTR modulators experienced a greater benefit in sinonasal and respiratory symptoms.

Conclusions: Elexacaftor-tezacaftor-ivacaftor is associated with significant improvement in sinonasal symptoms; previous use of CFTR modulators is associated with greater benefit.

Keywords: Cystic fibrosis; Highly effective modulator; Quality of life; SNOT-22; Sinonasal.

MeSH terms

Adult
Aminophenols / therapeutic use
Benzodioxoles / therapeutic use
Chloride Channel Agonists / therapeutic use*
Cystic Fibrosis / complications*
Cystic Fibrosis / drug therapy*
Drug Combinations
Female
Humans
Indoles / therapeutic use
Male
Paranasal Sinus Diseases / drug therapy*
Paranasal Sinus Diseases / etiology*
Prospective Studies
Pyrazoles / therapeutic use
Pyridines / therapeutic use
Pyrrolidines / therapeutic use
Quality of Life
Quinolones / therapeutic use

Substances

Aminophenols
Benzodioxoles
Chloride Channel Agonists
Drug Combinations
Indoles
Pyrazoles
Pyridines
Pyrrolidines
Quinolones
tezacaftor
ivacaftor
elexacaftor