Long-term experience with idursulfase beta (Hunterase) in two adolescent patients with MPS II: A case series.
Asian
Enzyme replacement therapy
Idursulfase beta
Mucopolysaccharidosis
Journal
Molecular genetics and metabolism reports
ISSN: 2214-4269
Titre abrégé: Mol Genet Metab Rep
Pays: United States
ID NLM: 101624422
Informations de publication
Date de publication:
Sep 2023
Sep 2023
Historique:
received:
01
02
2023
revised:
07
07
2023
accepted:
07
07
2023
medline:
6
9
2023
pubmed:
6
9
2023
entrez:
6
9
2023
Statut:
epublish
Résumé
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.
Identifiants
pubmed: 37670899
doi: 10.1016/j.ymgmr.2023.100991
pii: S2214-4269(23)00037-X
pmc: PMC10475840
doi:
Types de publication
Case Reports
Langues
eng
Pagination
100991Informations de copyright
© 2023 The Authors.
Déclaration de conflit d'intérêts
The authors declare that they have no conflict of interest.
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