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).


METHODS
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.
Burton
Barbara K
BK
Northwestern University Feinberg School of Medicine and Division of Genetics, Birth Defects and Metabolism, Children's Memorial Hospital, 2300 Children's Plaza, Chicago, IL 60614, USA. BBurton@childrensmemorial.org
Guffon
Nathalie
N
Roberts
Jane
J
van der Ploeg
Ans T
AT
Jones
Simon A
SA
D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D056947:Enzyme Replacement Therapy; D006699:Home Care Services; D006701:Home Nursing; D006801:Humans; D007066:Iduronate Sulfatase; D015918:Infusion Pumps, Implantable; D007262:Infusions, Intravenous / Q000009:adverse effects; D016532:Mucopolysaccharidosis II / Q000188:drug therapy; D016896:Treatment Outcome
D007066:Iduronate Sulfatase; C517982:idursulfase