The Impact of Enzyme-Replacement Therapy on Upper Airway Obstruction in Children with Mucopolysaccharidoses: Retrospective Cohort Study

Background. Data on the efficacy of enzyme replacement therapy (ERT) in relation to the pathology of ENT organs and respiratory performance in sleep in children with mucopolysaccharidosis (MPS) is poorly presented in the literature. Our aim was to assess the effect of ERT on the upper respiratory tr...

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Main Authors: Liliya A. Osipova, Ludmila M. Kuzenkova, Leyla S. Namazova-Baranova, Anait K. Gevorkyan, Tatiana V. Podkletnova, Viktor V. Altunin, Olga V. Kozhevnikova, Liya V. Toropchina, Tatiana A. Polunina, E. V. Zelenkova, Nato D. Vashakmadze
Format: Article
Language:Russian
Published: Union of pediatricians of Russia 2018-05-01
Series:Педиатрическая фармакология
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Online Access:https://www.pedpharma.ru/jour/article/view/1615
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author Liliya A. Osipova
Ludmila M. Kuzenkova
Leyla S. Namazova-Baranova
Anait K. Gevorkyan
Tatiana V. Podkletnova
Viktor V. Altunin
Olga V. Kozhevnikova
Liya V. Toropchina
Tatiana A. Polunina
E. V. Zelenkova
Nato D. Vashakmadze
author_facet Liliya A. Osipova
Ludmila M. Kuzenkova
Leyla S. Namazova-Baranova
Anait K. Gevorkyan
Tatiana V. Podkletnova
Viktor V. Altunin
Olga V. Kozhevnikova
Liya V. Toropchina
Tatiana A. Polunina
E. V. Zelenkova
Nato D. Vashakmadze
author_sort Liliya A. Osipova
collection DOAJ
description Background. Data on the efficacy of enzyme replacement therapy (ERT) in relation to the pathology of ENT organs and respiratory performance in sleep in children with mucopolysaccharidosis (MPS) is poorly presented in the literature. Our aim was to assess the effect of ERT on the upper respiratory tract in children with MPS. Methods. According to the case histories, we studied treatment results of children with MPS type I and II who received ERT in the Research Center of Children’s Health from January 2007 to November 2016. The severity of upper airway obstruction and its change during ERT was assessed according to indices of apnea-hypopnea and desaturation (SpO2), average/minimal SpO2, duration of SpO2 episodes < 90%, and hypertrophy degree of palatine tonsils and adenoids. Results. The severity of the obstructive sleep apnea syndrome did not progress in children (n = 15) with MPS against the background of ERT with a median duration of 38 (23; 48) months: initially, the apnea-hypopnea index was 3 (1.3; 7.7), while the repeated study — 2.6 (0.9; 13.5) (p = 0.507). There was also no statistically significant change in cardiorespiratory monitoring values. Conclusion. Long-term ERT in children with MPS type I and II interferes with the progression of airway obstruction.
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spelling doaj-art-a7bb8f3e83444eb7a078a8fa7e48b8532025-08-20T03:39:29ZrusUnion of pediatricians of RussiaПедиатрическая фармакология1727-57762500-30892018-05-0115213514010.15690/pf.v15i2.18701571The Impact of Enzyme-Replacement Therapy on Upper Airway Obstruction in Children with Mucopolysaccharidoses: Retrospective Cohort StudyLiliya A. Osipova0Ludmila M. Kuzenkova1Leyla S. Namazova-Baranova2Anait K. Gevorkyan3Tatiana V. Podkletnova4Viktor V. Altunin5Olga V. Kozhevnikova6Liya V. Toropchina7Tatiana A. Polunina8E. V. Zelenkova9Nato D. Vashakmadze10National Medical Research Center of Children’s Health of the Ministry of Health of the Russian Federation, MoscowNational Medical Research Center of Children’s Health of the Ministry of Health of the Russian Federation, Moscow; Sechenov First Moscow State Medical University, MoscowNational Medical Research Center of Children’s Health of the Ministry of Health of the Russian Federation, Moscow; Sechenov First Moscow State Medical University, Moscow; N.I. Pirogov Russian National Medical Research University, MoscowNational Medical Research Center of Children’s Health of the Ministry of Health of the Russian Federation, Moscow; Sechenov First Moscow State Medical University, MoscowNational Medical Research Center of Children’s Health of the Ministry of Health of the Russian Federation, MoscowNational Medical Research Center of Children’s Health of the Ministry of Health of the Russian Federation, MoscowNational Medical Research Center of Children’s Health of the Ministry of Health of the Russian Federation, MoscowRussian Medical Academy of Continuous Professional Education, Moscow; Moscow Regional Consultative and Diagnostic Center for Children, MoscowNational Medical Research Center of Children’s Health of the Ministry of Health of the Russian Federation, MoscowNational Medical Research Center of Children’s Health of the Ministry of Health of the Russian Federation, MoscowNational Medical Research Center of Children’s Health of the Ministry of Health of the Russian Federation, Moscow; N.I. Pirogov Russian National Medical Research University, MoscowBackground. Data on the efficacy of enzyme replacement therapy (ERT) in relation to the pathology of ENT organs and respiratory performance in sleep in children with mucopolysaccharidosis (MPS) is poorly presented in the literature. Our aim was to assess the effect of ERT on the upper respiratory tract in children with MPS. Methods. According to the case histories, we studied treatment results of children with MPS type I and II who received ERT in the Research Center of Children’s Health from January 2007 to November 2016. The severity of upper airway obstruction and its change during ERT was assessed according to indices of apnea-hypopnea and desaturation (SpO2), average/minimal SpO2, duration of SpO2 episodes < 90%, and hypertrophy degree of palatine tonsils and adenoids. Results. The severity of the obstructive sleep apnea syndrome did not progress in children (n = 15) with MPS against the background of ERT with a median duration of 38 (23; 48) months: initially, the apnea-hypopnea index was 3 (1.3; 7.7), while the repeated study — 2.6 (0.9; 13.5) (p = 0.507). There was also no statistically significant change in cardiorespiratory monitoring values. Conclusion. Long-term ERT in children with MPS type I and II interferes with the progression of airway obstruction.https://www.pedpharma.ru/jour/article/view/1615childrenmucopolysaccharidosisenzyme replacement therapyairway obstructionapnea-hypopnea indexhypertrophy of palatine tonsilshypertrophy of adenoids
spellingShingle Liliya A. Osipova
Ludmila M. Kuzenkova
Leyla S. Namazova-Baranova
Anait K. Gevorkyan
Tatiana V. Podkletnova
Viktor V. Altunin
Olga V. Kozhevnikova
Liya V. Toropchina
Tatiana A. Polunina
E. V. Zelenkova
Nato D. Vashakmadze
The Impact of Enzyme-Replacement Therapy on Upper Airway Obstruction in Children with Mucopolysaccharidoses: Retrospective Cohort Study
Педиатрическая фармакология
children
mucopolysaccharidosis
enzyme replacement therapy
airway obstruction
apnea-hypopnea index
hypertrophy of palatine tonsils
hypertrophy of adenoids
title The Impact of Enzyme-Replacement Therapy on Upper Airway Obstruction in Children with Mucopolysaccharidoses: Retrospective Cohort Study
title_full The Impact of Enzyme-Replacement Therapy on Upper Airway Obstruction in Children with Mucopolysaccharidoses: Retrospective Cohort Study
title_fullStr The Impact of Enzyme-Replacement Therapy on Upper Airway Obstruction in Children with Mucopolysaccharidoses: Retrospective Cohort Study
title_full_unstemmed The Impact of Enzyme-Replacement Therapy on Upper Airway Obstruction in Children with Mucopolysaccharidoses: Retrospective Cohort Study
title_short The Impact of Enzyme-Replacement Therapy on Upper Airway Obstruction in Children with Mucopolysaccharidoses: Retrospective Cohort Study
title_sort impact of enzyme replacement therapy on upper airway obstruction in children with mucopolysaccharidoses retrospective cohort study
topic children
mucopolysaccharidosis
enzyme replacement therapy
airway obstruction
apnea-hypopnea index
hypertrophy of palatine tonsils
hypertrophy of adenoids
url https://www.pedpharma.ru/jour/article/view/1615
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