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Long-term experience with idursulfase beta (Hunterase) in two adolescent patients with MPS II: A case series
Mucopolysaccharidosis (MPS) type II (Hunter syndrome) is a rare X-linked, recessive, lysosomal storage disorder caused by the deficit of the enzyme iduronate 2-sulfatase (IDS), resulting in accumulation of glycosaminoglycans (GAGs) impairing cellular function in multiple organ systems. Idursulfase (...
Autores principales: | , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Elsevier
2023
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10475840/ https://www.ncbi.nlm.nih.gov/pubmed/37670899 http://dx.doi.org/10.1016/j.ymgmr.2023.100991 |
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author | Chan, Mei-Yan Nelson, Andrew Jack Ngu, Lock-Hock |
author_facet | Chan, Mei-Yan Nelson, Andrew Jack Ngu, Lock-Hock |
author_sort | Chan, Mei-Yan |
collection | PubMed |
description | Mucopolysaccharidosis (MPS) type II (Hunter syndrome) is a rare X-linked, recessive, lysosomal storage disorder caused by the deficit of the enzyme iduronate 2-sulfatase (IDS), resulting in accumulation of glycosaminoglycans (GAGs) impairing cellular function in multiple organ systems. Idursulfase (Elaprase, Takeda Pharmaceuticals) and idursulfase beta (Hunterase, GC Biopharma Corp.) are the two currently available enzyme replacement therapies (ERT) for MPS II in Malaysia. ERT in patients with MPS II is associated with improvements in somatic symptoms, pulmonary function, endurance, joint mobility, and quality of life. Though mostly well tolerated, infusion-associated reactions (IARs), such as allergic (IgE-mediated) or nonallergic (non- immunologic) reactions can develop during ERT. In certain cases, when patients develop recurrent IARs despite reduced infusion rate and premedication, either interruption or cessation of ERT might be necessary. However, interruption of ERT is associated with worsening of clinical symptoms such as recurrent respiratory infections, difficulty in standing and walking, and increased joint stiffness, emphasizing the need for continuation of ERT. Here we report successful long-term experience with the use of idursulfase beta in two adolescent Malaysian patients with MPS II, who experienced recurrent infusion-associated reactions warranting discontinuation of ERT with idursulfase. |
format | Online Article Text |
id | pubmed-10475840 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2023 |
publisher | Elsevier |
record_format | MEDLINE/PubMed |
spelling | pubmed-104758402023-09-05 Long-term experience with idursulfase beta (Hunterase) in two adolescent patients with MPS II: A case series Chan, Mei-Yan Nelson, Andrew Jack Ngu, Lock-Hock Mol Genet Metab Rep Case Report Mucopolysaccharidosis (MPS) type II (Hunter syndrome) is a rare X-linked, recessive, lysosomal storage disorder caused by the deficit of the enzyme iduronate 2-sulfatase (IDS), resulting in accumulation of glycosaminoglycans (GAGs) impairing cellular function in multiple organ systems. Idursulfase (Elaprase, Takeda Pharmaceuticals) and idursulfase beta (Hunterase, GC Biopharma Corp.) are the two currently available enzyme replacement therapies (ERT) for MPS II in Malaysia. ERT in patients with MPS II is associated with improvements in somatic symptoms, pulmonary function, endurance, joint mobility, and quality of life. Though mostly well tolerated, infusion-associated reactions (IARs), such as allergic (IgE-mediated) or nonallergic (non- immunologic) reactions can develop during ERT. In certain cases, when patients develop recurrent IARs despite reduced infusion rate and premedication, either interruption or cessation of ERT might be necessary. However, interruption of ERT is associated with worsening of clinical symptoms such as recurrent respiratory infections, difficulty in standing and walking, and increased joint stiffness, emphasizing the need for continuation of ERT. Here we report successful long-term experience with the use of idursulfase beta in two adolescent Malaysian patients with MPS II, who experienced recurrent infusion-associated reactions warranting discontinuation of ERT with idursulfase. Elsevier 2023-07-12 /pmc/articles/PMC10475840/ /pubmed/37670899 http://dx.doi.org/10.1016/j.ymgmr.2023.100991 Text en © 2023 The Authors https://creativecommons.org/licenses/by/4.0/This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/). |
spellingShingle | Case Report Chan, Mei-Yan Nelson, Andrew Jack Ngu, Lock-Hock Long-term experience with idursulfase beta (Hunterase) in two adolescent patients with MPS II: A case series |
title | Long-term experience with idursulfase beta (Hunterase) in two adolescent patients with MPS II: A case series |
title_full | Long-term experience with idursulfase beta (Hunterase) in two adolescent patients with MPS II: A case series |
title_fullStr | Long-term experience with idursulfase beta (Hunterase) in two adolescent patients with MPS II: A case series |
title_full_unstemmed | Long-term experience with idursulfase beta (Hunterase) in two adolescent patients with MPS II: A case series |
title_short | Long-term experience with idursulfase beta (Hunterase) in two adolescent patients with MPS II: A case series |
title_sort | long-term experience with idursulfase beta (hunterase) in two adolescent patients with mps ii: a case series |
topic | Case Report |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10475840/ https://www.ncbi.nlm.nih.gov/pubmed/37670899 http://dx.doi.org/10.1016/j.ymgmr.2023.100991 |
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