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

Molecular Genetics and Metabolism
Barbara K BurtonHOS Investigators

Abstract

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). 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. 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. Providing appropriate factors are considered, it should be feasible for patients w...Continue Reading

References

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May 12, 2009·Molecular Genetics and Metabolism·Barbara K BurtonRachel Katz

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Citations

May 27, 2011·Journal of Inherited Metabolic Disease·Ursula MatteRoberto Giugliani
Nov 1, 2011·European Journal of Pediatrics·Joseph MuenzerPaul Harmatz
Nov 9, 2011·Orphanet Journal of Rare Diseases·Maurizio ScarpaUNKNOWN Hunter Syndrome Europena Expert Council
Sep 10, 2013·Italian Journal of Pediatrics·Ferdinando CeravoloDaniela Concolino
Sep 26, 2013·Medicina clínica·Encarna Guillén-NavarroUNKNOWN grupo de trabajo Hunter España
Jan 22, 2013·Molecular Genetics and Metabolism·B E SmidG E Linthorst
Apr 29, 2011·Revista clínica española·J Pérez-CalvoB Amores Arriaga
Jul 30, 2014·Genetics and Molecular Biology·Roberto GiuglianiHernán Amartino
Jun 24, 2017·Genetics in Medicine : Official Journal of the American College of Medical Genetics·Linda A BradleyGlenn E Palomaki
Feb 20, 2020·International Journal of Molecular Sciences·Francesca D'AvanzoRosella Tomanin
Dec 7, 2017·American Journal of Medical Genetics. Part a·Can FiciciogluRossella Parini
May 4, 2017·Orphanet Journal of Rare Diseases·Joseph MuenzerDavid A H Whiteman
Feb 10, 2021·Fortschritte der Neurologie-Psychiatrie·Andreas HahnBenedikt Schoser

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