Home treatment with intravenous enzyme replacement therapy with idursulfase for mucopolysaccharidosis type II - data from the Hunter Outcome Survey

Barbara K Burton*, Nathalie Guffon, Jane Roberts, Ans T. van der Ploeg, Simon A. Jones

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

22 Scopus citations

Abstract

Objective: To use our experience with patients in the observational database HOS - the Hunter Outcome Survey - to evaluate the feasibility of home infusions of idursulfase for patients with mucopolysaccharidosis type II (MPS II). Study design: Data were collected on or before 16 October 2009. Of 421 patients receiving idursulfase, 92 (21.9%) had received infusions at home or in an alternative, approved non-hospital environment. Information in HOS relating to the transition to home therapy was analyzed. Results: Patients started home therapy after a median of 9.0. months of idursulfase. Most were aged 5-11. years at transfer (45.7%; median age, 8.0. years), but many (19.6%) were < 5. years of age. Patients had disease manifestations typical of the wider population; over one-third had cognitive impairment, in some cases severe. Illness was the most frequent cause of missed home infusions. Six patients stopped home therapy; four subsequently resumed home infusions. Five infusion-related reactions occurred in 2 of the 59 patients who had received home therapy for at least 12. months. Reactions were classified as mild-to-moderate. All reactions that occurred at home were readily managed at home. Conclusions: Providing appropriate factors are considered, it should be feasible for patients with MPS II, including those who are severely affected, to receive infusions at home.

Original languageEnglish (US)
Pages (from-to)123-129
Number of pages7
JournalMolecular Genetics and Metabolism
Volume101
Issue number2-3
DOIs
StatePublished - Jan 1 2010

Keywords

  • Home therapy
  • Hunter syndrome
  • Infusion
  • Mucopolysaccharidoses

ASJC Scopus subject areas

  • Endocrinology, Diabetes and Metabolism
  • Biochemistry
  • Molecular Biology
  • Genetics
  • Endocrinology

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