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Home treatment with Elaprase® and Naglazyme® is safe in patients with mucopolysaccharidoses types II and VI, respectively
Author(s) -
Bagewadi S.,
Roberts J.,
Mercer J.,
Jones S.,
Stephenson J.,
Wraith J. E.
Publication year - 2008
Publication title -
journal of inherited metabolic disease
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.462
H-Index - 102
eISSN - 1573-2665
pISSN - 0141-8955
DOI - 10.1007/s10545-008-0980-0
Subject(s) - enzyme replacement therapy , mucopolysaccharidosis , medicine , mucopolysaccharidosis type ii , regimen , intensive care medicine , quality of life (healthcare) , hurler syndrome , lysosomal storage disorders , pediatrics , disease , nursing
Summary Enzyme replacement therapy for lysosomal storage disorders has made an important contribution to improving the quality of life of affected patients. The treatment, however, is invasive and onerous, involving weekly or biweekly intravenous infusions of product over a 3–4 h period. Such therapy can be extremely disruptive of normal family life and the provision of a safe, home treatment regimen is greatly appreciated by affected families. In this report we demonstrate the safety of home treatment with Elaprase for mucopolysaccharidosis type II (17 patients) and Naglazyme for mucopolysaccharidosis type VI (6 patients). Careful patient selection, an experienced home care company and a detailed management plan for potential anaphylaxis and infusion‐associated reactions are important components in a successful home treatment programme.