The impact of ivacaftor on sinonasal pathology in S1251N-mediated cystic fibrosis patients.
<h4>Importance</h4>Sinonasal symptoms in patients suffering from cystic fibrosis can negatively influence the quality of life and sinuses can be a niche for pathogens causing infection and inflammation leading to a decrease of lung function. Ivacaftor, a potentiator of the Cystic Fibrosi...
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| Format: | Article |
| Language: | English |
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Public Library of Science (PLoS)
2020-01-01
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| Series: | PLoS ONE |
| Online Access: | https://doi.org/10.1371/journal.pone.0235638 |
| _version_ | 1818590703162228736 |
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| author | Romee Gostelie Inge Stegeman Gitte Berkers Joost Bittermann Ivonne Ligtenberg-van der Drift Peter-Jan van Kipshagen Karin de Winter-de Groot Lucienne Speleman |
| author_facet | Romee Gostelie Inge Stegeman Gitte Berkers Joost Bittermann Ivonne Ligtenberg-van der Drift Peter-Jan van Kipshagen Karin de Winter-de Groot Lucienne Speleman |
| author_sort | Romee Gostelie |
| collection | DOAJ |
| description | <h4>Importance</h4>Sinonasal symptoms in patients suffering from cystic fibrosis can negatively influence the quality of life and sinuses can be a niche for pathogens causing infection and inflammation leading to a decrease of lung function. Ivacaftor, a potentiator of the Cystic Fibrosis Transmembrane Conductance Regulator protein, has shown improvement in pulmonary function in cystic fibrosis patients with different forms of class III gating mutations. However, the effects of ivacaftor on sinonasal pathology have hardly been studied.<h4>Objective</h4>To determine the impact of ivacaftor therapy on sinonasal pathology in patients with cystic fibrosis with an S1251N mutation.<h4>Design</h4>Prospective observational mono-center cohort study, between June 2015 and December 2016.<h4>Setting</h4>A tertiary referral center in Utrecht, The Netherlands.<h4>Participants</h4>Eight patients with cystic fibrosis with an S1251N mutation, treated with the potentiator ivacaftor were investigated.<h4>Exposures</h4>Ivacaftor (Kalydeco, VX-770) therapy. Computed tomography imaging of paranasal sinuses. Nasal nitric oxide concentration measurements and nasal endoscopy.<h4>Main outcomes and measures</h4>Primary outcome is opacification of paranasal sinuses examined with computed tomography scan analysis and scaled by the modified Lund-Mackay score before and one year after treatment. Secondary outcomes are nasal nitric oxide concentration levels, sinonasal symptoms and nasal endoscopic findings before and approximately two months and in some cases one year after treatment.<h4>Results</h4>Computed tomography scan analysis showed a significant decrease in opacification of the majority of paranasal sinuses comparing the opacification score per paranasal sinus before and after one year of treatment with ivacaftor. Median nasal nitric oxide levels significantly improved from 220.00 (IQR:136.00-341.18) to 462.84 (IQR:233.17-636.25) (p = 0.017) parts per billion. Likewise, the majority of sinonasal symptoms and nasal endoscopic pathology decreased or resolved at two months after the use of ivacaftor.<h4>Conclusion and relevance</h4>Ivacaftor appears to improve sinonasal outcome parameters and thereby sinonasal health in patients with cystic fibrosis with an S1251N mutation. |
| first_indexed | 2024-12-16T10:00:45Z |
| format | Article |
| id | doaj.art-2594187a4dbf46f992083e3a0e93febc |
| institution | Directory Open Access Journal |
| issn | 1932-6203 |
| language | English |
| last_indexed | 2024-12-16T10:00:45Z |
| publishDate | 2020-01-01 |
| publisher | Public Library of Science (PLoS) |
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| spelling | doaj.art-2594187a4dbf46f992083e3a0e93febc2022-12-21T22:35:47ZengPublic Library of Science (PLoS)PLoS ONE1932-62032020-01-01157e023563810.1371/journal.pone.0235638The impact of ivacaftor on sinonasal pathology in S1251N-mediated cystic fibrosis patients.Romee GostelieInge StegemanGitte BerkersJoost BittermannIvonne Ligtenberg-van der DriftPeter-Jan van KipshagenKarin de Winter-de GrootLucienne Speleman<h4>Importance</h4>Sinonasal symptoms in patients suffering from cystic fibrosis can negatively influence the quality of life and sinuses can be a niche for pathogens causing infection and inflammation leading to a decrease of lung function. Ivacaftor, a potentiator of the Cystic Fibrosis Transmembrane Conductance Regulator protein, has shown improvement in pulmonary function in cystic fibrosis patients with different forms of class III gating mutations. However, the effects of ivacaftor on sinonasal pathology have hardly been studied.<h4>Objective</h4>To determine the impact of ivacaftor therapy on sinonasal pathology in patients with cystic fibrosis with an S1251N mutation.<h4>Design</h4>Prospective observational mono-center cohort study, between June 2015 and December 2016.<h4>Setting</h4>A tertiary referral center in Utrecht, The Netherlands.<h4>Participants</h4>Eight patients with cystic fibrosis with an S1251N mutation, treated with the potentiator ivacaftor were investigated.<h4>Exposures</h4>Ivacaftor (Kalydeco, VX-770) therapy. Computed tomography imaging of paranasal sinuses. Nasal nitric oxide concentration measurements and nasal endoscopy.<h4>Main outcomes and measures</h4>Primary outcome is opacification of paranasal sinuses examined with computed tomography scan analysis and scaled by the modified Lund-Mackay score before and one year after treatment. Secondary outcomes are nasal nitric oxide concentration levels, sinonasal symptoms and nasal endoscopic findings before and approximately two months and in some cases one year after treatment.<h4>Results</h4>Computed tomography scan analysis showed a significant decrease in opacification of the majority of paranasal sinuses comparing the opacification score per paranasal sinus before and after one year of treatment with ivacaftor. Median nasal nitric oxide levels significantly improved from 220.00 (IQR:136.00-341.18) to 462.84 (IQR:233.17-636.25) (p = 0.017) parts per billion. Likewise, the majority of sinonasal symptoms and nasal endoscopic pathology decreased or resolved at two months after the use of ivacaftor.<h4>Conclusion and relevance</h4>Ivacaftor appears to improve sinonasal outcome parameters and thereby sinonasal health in patients with cystic fibrosis with an S1251N mutation.https://doi.org/10.1371/journal.pone.0235638 |
| spellingShingle | Romee Gostelie Inge Stegeman Gitte Berkers Joost Bittermann Ivonne Ligtenberg-van der Drift Peter-Jan van Kipshagen Karin de Winter-de Groot Lucienne Speleman The impact of ivacaftor on sinonasal pathology in S1251N-mediated cystic fibrosis patients. PLoS ONE |
| title | The impact of ivacaftor on sinonasal pathology in S1251N-mediated cystic fibrosis patients. |
| title_full | The impact of ivacaftor on sinonasal pathology in S1251N-mediated cystic fibrosis patients. |
| title_fullStr | The impact of ivacaftor on sinonasal pathology in S1251N-mediated cystic fibrosis patients. |
| title_full_unstemmed | The impact of ivacaftor on sinonasal pathology in S1251N-mediated cystic fibrosis patients. |
| title_short | The impact of ivacaftor on sinonasal pathology in S1251N-mediated cystic fibrosis patients. |
| title_sort | impact of ivacaftor on sinonasal pathology in s1251n mediated cystic fibrosis patients |
| url | https://doi.org/10.1371/journal.pone.0235638 |
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