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

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Home treatment with intravenous enzyme replacement therapy with idursulfase for mucopolysaccharidosis type II - data from the Hunter Outcome Survey. / Burton, Barbara K; Guffon, Nathalie; Roberts, Jane; van der Ploeg, Ans T; Jones, Simon A; HOS Investigators ; Muschol, Nicole Maria.

in: MOL GENET METAB, Jahrgang 101, Nr. 2-3, 20.07.2010, S. 123-9.

Publikationen: SCORING: Beitrag in Fachzeitschrift/Zeitung › SCORING: Zeitschriftenaufsatz › Forschung › Begutachtung

Harvard

Burton, BK, Guffon, N, Roberts, J, van der Ploeg, AT, Jones, SA, HOS Investigators & Muschol, NM 2010, 'Home treatment with intravenous enzyme replacement therapy with idursulfase for mucopolysaccharidosis type II - data from the Hunter Outcome Survey', MOL GENET METAB, Jg. 101, Nr. 2-3, S. 123-9. https://doi.org/10.1016/j.ymgme.2010.06.011

APA

Burton, B. K., Guffon, N., Roberts, J., van der Ploeg, A. T., Jones, S. A., HOS Investigators, & Muschol, N. M. (2010). Home treatment with intravenous enzyme replacement therapy with idursulfase for mucopolysaccharidosis type II - data from the Hunter Outcome Survey. MOL GENET METAB, 101(2-3), 123-9. https://doi.org/10.1016/j.ymgme.2010.06.011

Vancouver

Burton BK, Guffon N, Roberts J, van der Ploeg AT, Jones SA, HOS Investigators et al. Home treatment with intravenous enzyme replacement therapy with idursulfase for mucopolysaccharidosis type II - data from the Hunter Outcome Survey. MOL GENET METAB. 2010 Jul 20;101(2-3):123-9. https://doi.org/10.1016/j.ymgme.2010.06.011

Bibtex

@article{37c1843c67674a78a6a55e73f8d9337e,
title = "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.",
keywords = "Adolescent, Child, Child, Preschool, Enzyme Replacement Therapy, Home Care Services, Home Nursing, Humans, Iduronate Sulfatase, Infusion Pumps, Implantable, Infusions, Intravenous, Mucopolysaccharidosis II, Treatment Outcome",
author = "Burton, {Barbara K} and Nathalie Guffon and Jane Roberts and {van der Ploeg}, {Ans T} and Jones, {Simon A} and {HOS Investigators} and Muschol, {Nicole Maria}",
note = "Copyright {\textcopyright} 2010 Elsevier Inc. All rights reserved.",
year = "2010",
month = jul,
day = "20",
doi = "10.1016/j.ymgme.2010.06.011",
language = "English",
volume = "101",
pages = "123--9",
journal = "MOL GENET METAB",
issn = "1096-7192",
publisher = "Academic Press Inc.",
number = "2-3",

}

RIS

TY - JOUR

T1 - Home treatment with intravenous enzyme replacement therapy with idursulfase for mucopolysaccharidosis type II - data from the Hunter Outcome Survey

AU - Burton, Barbara K

AU - Guffon, Nathalie

AU - Roberts, Jane

AU - van der Ploeg, Ans T

AU - Jones, Simon A

AU - HOS Investigators

AU - Muschol, Nicole Maria

N1 - Copyright © 2010 Elsevier Inc. All rights reserved.

PY - 2010/7/20

Y1 - 2010/7/20

N2 - 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.

AB - 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.

KW - Adolescent

KW - Child

KW - Child, Preschool

KW - Enzyme Replacement Therapy

KW - Home Care Services

KW - Home Nursing

KW - Humans

KW - Iduronate Sulfatase

KW - Infusion Pumps, Implantable

KW - Infusions, Intravenous

KW - Mucopolysaccharidosis II

KW - Treatment Outcome

U2 - 10.1016/j.ymgme.2010.06.011

DO - 10.1016/j.ymgme.2010.06.011

M3 - SCORING: Journal article

C2 - 20638311

VL - 101

SP - 123

EP - 129

JO - MOL GENET METAB

JF - MOL GENET METAB

SN - 1096-7192

IS - 2-3

ER -