Clinical Case of Drug Allergy to Enzyme Replacement Therapy in a Patient with Mucopolysaccharidosis Type II

Background. Enzyme replacement therapy (ERT) with iduronate-2-sulfatase recombinant forms (idursulfase and idursulfase beta) is effective for the management of mucopolysaccharidosis type II (MPS II). Patients with Hunter syndrome require lifelong ERT that can negate endogenous enzyme deficiency. How...

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Autori principali: Julia G. Levina, Nato D. Vashakmadze, Leyla S. Namazova-Baranova, Elena A. Vishneva, Mariya S. Karaseva, Natalia V. Zhurkova, Kamilla E. Efendieva, Anna A. Alekseeva, Vera G. Kalugina, Artur V. Zaz’yan
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Lingua:russo
Pubblicazione: Union of pediatricians of Russia 2022-07-01
Serie:Педиатрическая фармакология
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Accesso online:https://www.pedpharma.ru/jour/article/view/2178
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author Julia G. Levina
Nato D. Vashakmadze
Leyla S. Namazova-Baranova
Elena A. Vishneva
Mariya S. Karaseva
Natalia V. Zhurkova
Kamilla E. Efendieva
Anna A. Alekseeva
Vera G. Kalugina
Artur V. Zaz’yan
author_facet Julia G. Levina
Nato D. Vashakmadze
Leyla S. Namazova-Baranova
Elena A. Vishneva
Mariya S. Karaseva
Natalia V. Zhurkova
Kamilla E. Efendieva
Anna A. Alekseeva
Vera G. Kalugina
Artur V. Zaz’yan
author_sort Julia G. Levina
collection DOAJ
description Background. Enzyme replacement therapy (ERT) with iduronate-2-sulfatase recombinant forms (idursulfase and idursulfase beta) is effective for the management of mucopolysaccharidosis type II (MPS II). Patients with Hunter syndrome require lifelong ERT that can negate endogenous enzyme deficiency. However, hypersensitivity reactions may occur during ERT, and they significantly complicate the implementation of vital therapy.Clinical case description. This article describes clinical case of a child with hypersensitivity reaction to ERT. The patient with confirmed diagnosis of MPS II was administrated with idursulfase. Then, the drug was replaced with idursulfase beta due to the allergic reaction. Thus, even after the drug change, side effects maintained without sustained improvement with underlying glucocorticosteroids (GCS), antihistamines and with decreased infusion rate. Concerning the vital need to continue ERT, this patient with drug allergy to this pharmacotherapeutic group was further administered with combined therapy of cyclosporine and omalizumab. Personalised protocol for the administration of idursulfase beta with desensitization was developed. Such experience was firstly described In Russian patient.Conclusion. The presented personalised combination therapy made it possible to prevent hypersensitivity reactions during ERT in the patient with MPS II.
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spelling doaj-art-666e065089ca44c8a429bcce968c79182025-08-03T19:21:30ZrusUnion of pediatricians of RussiaПедиатрическая фармакология1727-57762500-30892022-07-0119325025710.15690/pf.v19i3.24381912Clinical Case of Drug Allergy to Enzyme Replacement Therapy in a Patient with Mucopolysaccharidosis Type IIJulia G. Levina0Nato D. Vashakmadze1Leyla S. Namazova-Baranova2Elena A. Vishneva3Mariya S. Karaseva4Natalia V. Zhurkova5Kamilla E. Efendieva6Anna A. Alekseeva7Vera G. Kalugina8Artur V. Zaz’yan9Pediatrics and Child Health Research Institute in Petrovsky National Research Centre of Surgery; Pirogov Russian National Research Medical UniversityPediatrics and Child Health Research Institute in Petrovsky National Research Centre of Surgery; Pirogov Russian National Research Medical UniversityPediatrics and Child Health Research Institute in Petrovsky National Research Centre of Surgery; Pirogov Russian National Research Medical University; Belgorod National Research UniversityPediatrics and Child Health Research Institute in Petrovsky National Research Centre of Surgery; Pirogov Russian National Research Medical UniversityPediatrics and Child Health Research Institute in Petrovsky National Research Centre of SurgeryPediatrics and Child Health Research Institute in Petrovsky National Research Centre of Surgery; Medical Genetic Research Center named after N.P. BochkovPediatrics and Child Health Research Institute in Petrovsky National Research Centre of Surgery; Pirogov Russian National Research Medical UniversityPediatrics and Child Health Research Institute in Petrovsky National Research Centre of SurgeryPediatrics and Child Health Research Institute in Petrovsky National Research Centre of SurgeryChildren’s City Hospital № 1Background. Enzyme replacement therapy (ERT) with iduronate-2-sulfatase recombinant forms (idursulfase and idursulfase beta) is effective for the management of mucopolysaccharidosis type II (MPS II). Patients with Hunter syndrome require lifelong ERT that can negate endogenous enzyme deficiency. However, hypersensitivity reactions may occur during ERT, and they significantly complicate the implementation of vital therapy.Clinical case description. This article describes clinical case of a child with hypersensitivity reaction to ERT. The patient with confirmed diagnosis of MPS II was administrated with idursulfase. Then, the drug was replaced with idursulfase beta due to the allergic reaction. Thus, even after the drug change, side effects maintained without sustained improvement with underlying glucocorticosteroids (GCS), antihistamines and with decreased infusion rate. Concerning the vital need to continue ERT, this patient with drug allergy to this pharmacotherapeutic group was further administered with combined therapy of cyclosporine and omalizumab. Personalised protocol for the administration of idursulfase beta with desensitization was developed. Such experience was firstly described In Russian patient.Conclusion. The presented personalised combination therapy made it possible to prevent hypersensitivity reactions during ERT in the patient with MPS II.https://www.pedpharma.ru/jour/article/view/2178mucopolysaccharidosis type iihunter syndromeenzyme replacement therapyidursulfaseidursulfase betadrug allergyurticariachildrenclinical case
spellingShingle Julia G. Levina
Nato D. Vashakmadze
Leyla S. Namazova-Baranova
Elena A. Vishneva
Mariya S. Karaseva
Natalia V. Zhurkova
Kamilla E. Efendieva
Anna A. Alekseeva
Vera G. Kalugina
Artur V. Zaz’yan
Clinical Case of Drug Allergy to Enzyme Replacement Therapy in a Patient with Mucopolysaccharidosis Type II
Педиатрическая фармакология
mucopolysaccharidosis type ii
hunter syndrome
enzyme replacement therapy
idursulfase
idursulfase beta
drug allergy
urticaria
children
clinical case
title Clinical Case of Drug Allergy to Enzyme Replacement Therapy in a Patient with Mucopolysaccharidosis Type II
title_full Clinical Case of Drug Allergy to Enzyme Replacement Therapy in a Patient with Mucopolysaccharidosis Type II
title_fullStr Clinical Case of Drug Allergy to Enzyme Replacement Therapy in a Patient with Mucopolysaccharidosis Type II
title_full_unstemmed Clinical Case of Drug Allergy to Enzyme Replacement Therapy in a Patient with Mucopolysaccharidosis Type II
title_short Clinical Case of Drug Allergy to Enzyme Replacement Therapy in a Patient with Mucopolysaccharidosis Type II
title_sort clinical case of drug allergy to enzyme replacement therapy in a patient with mucopolysaccharidosis type ii
topic mucopolysaccharidosis type ii
hunter syndrome
enzyme replacement therapy
idursulfase
idursulfase beta
drug allergy
urticaria
children
clinical case
url https://www.pedpharma.ru/jour/article/view/2178
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