Experience of Idursulfase Beta Administration in the Child with Mucopolysaccharidosis Type II: Clinical Case
https://doi.org/10.15690/vsp.v19i5.2212
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.
Keywords
Supporting agencies: Not specified.
About the Authors
Tatiana K. Kruchina
Saint Petersburg State Pediatric Medical University;
Children’s City Multidisciplinary Clinical Specialized Center of High Medical Technologies
Russian Federation
Disclosure of interest:
Russian Federation
Saint Petersburg
Disclosure of interest:
Tatiana K. Kruchina — lecturing for Sanofi, Nanolek companies
Konstantin V. Bruchikov
Children’s City Multidisciplinary Clinical Specialized Center of High Medical Technologies
Russian Federation
Disclosure of interest:
Russian Federation
Saint Petersburg
Disclosure of interest:
The other contributors confirmed the absence of a reportable conflict of interests
Gennady A. Novik
Saint Petersburg State Pediatric Medical University
Russian Federation
Disclosure of interest:
Russian Federation
Saint Petersburg
Disclosure of interest:
The other contributors confirmed the absence of a reportable conflict of interests
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Review
For citations:
Kruchina T.K., Bruchikov K.V., Novik G.A. Experience of Idursulfase Beta Administration in the Child with Mucopolysaccharidosis Type II: Clinical Case. Current Pediatrics. 2020;19(5):364-370. (In Russ.) https://doi.org/10.15690/vsp.v19i5.2212
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