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

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 9.0months of idursulfase. Most were aged 5-11years at transfer (45.7%; median age, 8.0years), but many (19.6%) were <5years 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 12months. 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.

• Authors: Barbara K Burton, Nathalie Guffon, Jane Roberts, Ans T van der Ploeg, Simon A Jones, HOS investigators
• Journal: Molecular genetics and metabolism (Mol Genet Metab) 2010 Oct-Nov Vol. 101 Issue 2-3 Pg. 123-9 ISSN: 1096-7206 [Electronic] United States
• PMID: 20638311 (Publication Type: Journal Article)
• Copyright: Copyright © 2010 Elsevier Inc. All rights reserved.

Chemical References

• Iduronate Sulfatase
• idursulfase

Topics

• Adolescent
• Child
• Child, Preschool
• Enzyme Replacement Therapy
• Home Care Services
• Home Nursing
• Humans
• Iduronate Sulfatase
• Infusion Pumps, Implantable
• Infusions, Intravenous (adverse effects)
• Mucopolysaccharidosis II (drug therapy)
• Treatment Outcome