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

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 511 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. (C) 2010 Elsevier Inc. All rights reserved.