Case report of treatment experience with idursulfase beta (Hunterase) in an adolescent patient with MPS II

Abstract

Mucopolysaccharidosis (MPS) II or Hunter syndrome is a chronic, progressive, multi-systemic illness associated with significant morbidity and early mortality. Available evidence in Asian populations shows that Hunter syndrome has a mean age of onset of 2 to 5 years and a life expectancy of 13 years in more severely affected individuals, with respiratory failure reported as the leading cause of death. Enzyme replacement therapy (ERT) with idursulfase (Elaprase, Shire Pharmaceuticals) and idursulfase beta (Hunterase, Green Cross Corp) are the only approved treatment for patients with MPS II. While these agents have the same amino acids, they have different glycosylation patterns because they are produced in different cell lines via different manufacturing processes. In previous studies, the beneficial effects of idursulfase beta have been confirmed in patients up to 35 years of age, without serious treatment-related safety concerns. The major drawbacks associated with ERT include the potential development of serious infusion-related anaphylactic reactions and up to 50% of treated patients develop anti-IDS antibodies. Here we report the case of a 13-year-old Malaysian patient with attenuated MPS II who developed troublesome infusion-associated reactions while receiving idursulfase treatment but tolerated and responded favorably to idursulfase beta.

Impact of intracerebroventricular enzyme replacement therapy in patients with neuronopathic mucopolysaccharidosis type II

“…In addition, all but one patient had a greater increase in DA at week 100 compared with historical controls, with an average difference for all six patients of +5.1 months. Thus, unlike intravenously administered idursulfase and idursulfase beta, which cannot penetrate the blood-brain barrier, 1 , 12 i.c.v. administration of idursulfase beta may be expected to improve CNS manifestations.…”

Epidemiology of Mucopolysaccharidoses Update

“…Another study in 2017 by Ngu and colleagues indicated high MPS II type frequency. Out of 79 MPS patients, a total of 30 patients were identified as MPS II [ 28 ].…”

Experience of Idursulfase Beta Administration in the Child with Mucopolysaccharidosis Type II: Clinical Case

“…Пациентам может потребоваться удлинение времени инфузии и/или приостановка введения препарата из-за развития нежелательных реакций [10][11][12], наиболее частыми из которых являются реакции гиперчувствительности [10][11][12], которые обычно купируются путем замедления скорости инфузии и проведения премедикации антигистаминными препаратами и/или антипиретиками (парацетамол или ибупрофен). Однако в ряде случаев побочные эффекты ФЗТ не поддаются контролю и могут значительно осложнять лечение [18]. В связи с этим целесообразным представляется переключение с одного препарата для ФЗТ на другой [18].…”

“…Однако в ряде случаев побочные эффекты ФЗТ не поддаются контролю и могут значительно осложнять лечение [18]. В связи с этим целесообразным представляется переключение с одного препарата для ФЗТ на другой [18].…”