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Airway Findings in Patients with Hunter Syndrome Treated with Intravenous Idursulfase

People with Hunter syndrome are known to be affected by a variety of airway pathologies. Treatment of Hunter syndrome with the enzyme replacement therapy (ERT) idursulfase is now the standard of care. However, it is not known how ERT changes the progression of airway involvement. To evaluate this, w...

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Autores principales: De Vuyst, Richard, Jalazo, Elizabeth, Tsujimoto, Tamy Moraes, Lin, Feng-Chang, Muenzer, Joseph, Muhlebach, Marianne S.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9863383/
https://www.ncbi.nlm.nih.gov/pubmed/36675409
http://dx.doi.org/10.3390/jcm12020480
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author De Vuyst, Richard
Jalazo, Elizabeth
Tsujimoto, Tamy Moraes
Lin, Feng-Chang
Muenzer, Joseph
Muhlebach, Marianne S.
author_facet De Vuyst, Richard
Jalazo, Elizabeth
Tsujimoto, Tamy Moraes
Lin, Feng-Chang
Muenzer, Joseph
Muhlebach, Marianne S.
author_sort De Vuyst, Richard
collection PubMed
description People with Hunter syndrome are known to be affected by a variety of airway pathologies. Treatment of Hunter syndrome with the enzyme replacement therapy (ERT) idursulfase is now the standard of care. However, it is not known how ERT changes the progression of airway involvement. To evaluate this, we performed a retrospective analysis of bronchoscopies performed on children with Hunter syndrome who were part of intrathecal ERT trials. Findings for airway pathology were extracted from bronchoscopy reports and analyses were performed for cross-sectional and longitudinal changes in airway disease. One-hundred and thirty bronchoscopies from 23 subjects were analyzed. Upper airway disease (adenoid hypertrophy and/or pharyngomalacia) was reported in 93% and 87% of bronchoscopies, respectively. Laryngeal abnormalities were recognized in 46% of cases. There were lower airway (tracheal and or bronchial) findings in 64% of all bronchoscopies and prevalence increased with age. Evaluations over time adjusted for repeat evaluations showed that increasing airway involvement was associated with older age (p = 0.0007) despite ongoing ERT. No association was discovered between age of intravenous ERT initiation and progression of airway disease. Individuals with Hunter syndrome who are receiving intravenous enzyme replacement therapy showed the progression of airways disease supporting the need for regular airway monitoring and intervention.
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spelling pubmed-98633832023-01-22 Airway Findings in Patients with Hunter Syndrome Treated with Intravenous Idursulfase De Vuyst, Richard Jalazo, Elizabeth Tsujimoto, Tamy Moraes Lin, Feng-Chang Muenzer, Joseph Muhlebach, Marianne S. J Clin Med Article People with Hunter syndrome are known to be affected by a variety of airway pathologies. Treatment of Hunter syndrome with the enzyme replacement therapy (ERT) idursulfase is now the standard of care. However, it is not known how ERT changes the progression of airway involvement. To evaluate this, we performed a retrospective analysis of bronchoscopies performed on children with Hunter syndrome who were part of intrathecal ERT trials. Findings for airway pathology were extracted from bronchoscopy reports and analyses were performed for cross-sectional and longitudinal changes in airway disease. One-hundred and thirty bronchoscopies from 23 subjects were analyzed. Upper airway disease (adenoid hypertrophy and/or pharyngomalacia) was reported in 93% and 87% of bronchoscopies, respectively. Laryngeal abnormalities were recognized in 46% of cases. There were lower airway (tracheal and or bronchial) findings in 64% of all bronchoscopies and prevalence increased with age. Evaluations over time adjusted for repeat evaluations showed that increasing airway involvement was associated with older age (p = 0.0007) despite ongoing ERT. No association was discovered between age of intravenous ERT initiation and progression of airway disease. Individuals with Hunter syndrome who are receiving intravenous enzyme replacement therapy showed the progression of airways disease supporting the need for regular airway monitoring and intervention. MDPI 2023-01-06 /pmc/articles/PMC9863383/ /pubmed/36675409 http://dx.doi.org/10.3390/jcm12020480 Text en © 2023 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).
spellingShingle Article
De Vuyst, Richard
Jalazo, Elizabeth
Tsujimoto, Tamy Moraes
Lin, Feng-Chang
Muenzer, Joseph
Muhlebach, Marianne S.
Airway Findings in Patients with Hunter Syndrome Treated with Intravenous Idursulfase
title Airway Findings in Patients with Hunter Syndrome Treated with Intravenous Idursulfase
title_full Airway Findings in Patients with Hunter Syndrome Treated with Intravenous Idursulfase
title_fullStr Airway Findings in Patients with Hunter Syndrome Treated with Intravenous Idursulfase
title_full_unstemmed Airway Findings in Patients with Hunter Syndrome Treated with Intravenous Idursulfase
title_short Airway Findings in Patients with Hunter Syndrome Treated with Intravenous Idursulfase
title_sort airway findings in patients with hunter syndrome treated with intravenous idursulfase
topic Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9863383/
https://www.ncbi.nlm.nih.gov/pubmed/36675409
http://dx.doi.org/10.3390/jcm12020480
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