Abstract
Background. Mucopolysaccharidosis type II (MPS II, Hunter syndrome) is a rare hereditary lysosomal storage disease associated with iduronate-2-sulfatase deficiency. Patients with MPS II require life-long enzyme replacement therapy (ERT) to replace the deficiency of endogenous enzyme. There are two medications — idursulfase and idursulfase beta — that are licensed and recommended for these patients in Russian Federation. However, it is well known that ERT can cause hypersensitivity reactions development.Clinical Case Description. The ERT (idursulfase in the dose of 0.5 mg/kg once per week) onset in the male patient with severe MPS II was at the age of 2.5 years. Hypersensitivity reactions (urticaria, fever) were noted incidentally, thus, the premedication with antihistamines and antipyretics was performed. The ERT side effects has aggravated at the age of 8 years despite the glucocorticosteroids admission and infusion rate reduction up to 8–16 ml/h. That is why we have changed the medication on idursulfase beta with major clinical response: we have achieved control on both disease itself and hypersensitivity reactions.Conclusion. The availability of two ERT medications for patients with MPS II expands treatment opportunities. In case of any allergic reactions due to idursulfase, the change on idursulfase beta reduces the risk of any ERT complications with sufficient control of MPS II course.
Highlights
Mucopolysaccharidosis type II (MPS II, Hunter syndrome) is a rare hereditary lysosomal storage disease associated with iduronate-2-sulfatase deficiency
There are two medications — idursulfase and idursulfase beta — that are licensed and recommended for these patients in Russian Federation
It is well known that enzyme replacement therapy (ERT) can cause hypersensitivity reactions development
Summary
Опыт применения препарата идурсульфаза бета у ребенка с мукополисахаридозом II типа: клинический случай. В Российской Федерации для этих целей разрешены и рекомендованы два препарата — идурсульфаза и идурсульфаза бета. Что ФЗТ сопряжена с риском развития реакций гиперчувствительности. У пациента мужского пола с тяжелой формой МПС II в возрасте 2,5 лет была инициирована ФЗТ, назначен препарат идурсульфаза в дозировке 0,5 мг/кг один раз в неделю. Было проведено переключение пациента на препарат идурсульфаза бета с хорошим клиническим эффектом: достигнут контроль и основного заболевания, и развития аллергических реакций. При возникновении аллергических реакций на препарат идурсульфаза переключение на препарат идурсульфаза бета снижает риск возникновения осложнений ФЗТ при достаточном контроле течения МПС II. Ключевые слова: мукополисахаридоз, ферментозаместительная терапия, идурсульфаза бета, дети, клинический случай. Experience of Idursulfase Beta Administration in the Child with Mucopolysaccharidosis Type II: Clinical Case
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Disclaimer: All third-party content on this website/platform is and will remain the property of their respective owners and is provided on "as is" basis without any warranties, express or implied. Use of third-party content does not indicate any affiliation, sponsorship with or endorsement by them. Any references to third-party content is to identify the corresponding services and shall be considered fair use under The CopyrightLaw.