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Home treatment with Elaprase® and Naglazyme® is safe in patients with mucopolysaccharidoses types II and VI, respectively

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Journal of Inherited Metabolic Disease

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.

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References

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Author information

Authors and Affiliations

  1. Willink Biochemical Genetics Unit, Royal Manchester Children’s Hospital, Manchester, M27 4HA, UK

    S. Bagewadi, J. Roberts, J. Mercer, S. Jones & J. E. Wraith

  2. Healthcare at Home, Burton-upon-Trent, Staffordshire, UK

    J. Stephenson

Authors
  1. S. Bagewadi
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  2. J. Roberts
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  3. J. Mercer
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  4. S. Jones
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  5. J. Stephenson
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  6. J. E. Wraith
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Corresponding author

Correspondence to J. E. Wraith.

Additional information

Communicating editor: Douglas Brooks

Competing interests: J. E. Wraith has been Principal Investigator in clinical trials of both Elaprase® and Naglazyme® and has provided paid and unpaid consultancy duties for both Shire HGT (Elaprase®) and Biomarin (Naglazyme®).

References to electronic databases: MPS II: OMIM 309900. MPS VI: OMIM 253200. MPS I: Hurler OMIM 607014; Hurler-Scheie OMIM 607015; Scheie OMIM 607016.

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Bagewadi, S., Roberts, J., Mercer, J. et al. Home treatment with Elaprase® and Naglazyme® is safe in patients with mucopolysaccharidoses types II and VI, respectively. J Inherit Metab Dis 31, 733–737 (2008). https://doi.org/10.1007/s10545-008-0980-0

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  • DOI: https://doi.org/10.1007/s10545-008-0980-0

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