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Pregled bibliografske jedinice broj: 538188

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

Burton, B.K.; Guffon, N.; Roberts, J.; van der Ploeg, A.T.; Jones, S.A.; on behalf of the HOS investigators: Guelbert, N. ...; Barić, Ivo; Barišić, Ingeborg; ...; Ibrahim, J.
Home treatment with intravenous enzyme replacement therapy with idursulfase for mucopolysaccharidosis type II - data from the Hunter Outcome Survey // Molecular genetics and metabolism, 101 (2010), 2/3; 123-129 doi:10.1016/j.ymgme.2010.06.011 (međunarodna recenzija, članak, znanstveni)

CROSBI ID: 538188 Za ispravke kontaktirajte CROSBI podršku putem web obrasca

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

Autori
Burton, B.K. ; Guffon, N. ; Roberts, J. ; van der Ploeg, A.T. ; Jones, S.A. ; on behalf of the HOS investigators: Guelbert, N. ... ; Barić, Ivo ; Barišić, Ingeborg ; ... ; Ibrahim, J.

Izvornik
Molecular genetics and metabolism (1096-7192) 101 (2010), 2/3; 123-129

Vrsta, podvrsta i kategorija rada
Radovi u časopisima, članak, znanstveni

Ključne riječi
Hunter syndrome; mucopolysaccharidoses; Infusion; home therapy

Sažetak
Objestive waa 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 with MPS II, including those who are severely affected, to receive infusions at home.

Izvorni jezik
Engleski

Znanstvena područja
Temeljne medicinske znanosti, Kliničke medicinske znanosti



POVEZANOST RADA

Projekti:
072-1083107-0365 - Istraživanje epidemiologijskih i genetičkih osnova prirođenih mana (Barišić, Ingeborg, MZOS ) ( CroRIS)
108-1081870-1885 - Nasljedne metaboličke i ostale monogenske bolesti djece (Barić, Ivo, MZOS ) ( CroRIS)

Ustanove:
Klinika za dječje bolesti Medicinskog fakulteta,
Medicinski fakultet, Zagreb

Profili:

Avatar Url Ivo Barić (autor)

Avatar Url Ingeborg Barišić (autor)

Poveznice na cjeloviti tekst rada:

doi ac.els-cdn.com www.sciencedirect.com dx.doi.org

Citiraj ovu publikaciju:

Burton, B.K.; Guffon, N.; Roberts, J.; van der Ploeg, A.T.; Jones, S.A.; on behalf of the HOS investigators: Guelbert, N. ...; Barić, Ivo; Barišić, Ingeborg; ...; Ibrahim, J.
Home treatment with intravenous enzyme replacement therapy with idursulfase for mucopolysaccharidosis type II - data from the Hunter Outcome Survey // Molecular genetics and metabolism, 101 (2010), 2/3; 123-129 doi:10.1016/j.ymgme.2010.06.011 (međunarodna recenzija, članak, znanstveni)
Burton, B., Guffon, N., Roberts, J., van der Ploeg, A., Jones, S., on behalf of the HOS investigators: Guelbert, N., Barić, I., Barišić, I., ... & Ibrahim, J. (2010) Home treatment with intravenous enzyme replacement therapy with idursulfase for mucopolysaccharidosis type II - data from the Hunter Outcome Survey. Molecular genetics and metabolism, 101 (2/3), 123-129 doi:10.1016/j.ymgme.2010.06.011.
@article{article, author = {Burton, B.K. and Guffon, N. and Roberts, J. and van der Ploeg, A.T. and Jones, S.A. and on behalf of the HOS investigators: Guelbert, N. ... and Bari\'{c}, Ivo and Bari\v{s}i\'{c}, Ingeborg and Ibrahim, J.}, year = {2010}, pages = {123-129}, DOI = {10.1016/j.ymgme.2010.06.011}, keywords = {Hunter syndrome, mucopolysaccharidoses, Infusion, home therapy}, journal = {Molecular genetics and metabolism}, doi = {10.1016/j.ymgme.2010.06.011}, volume = {101}, number = {2/3}, issn = {1096-7192}, title = {Home treatment with intravenous enzyme replacement therapy with idursulfase for mucopolysaccharidosis type II - data from the Hunter Outcome Survey}, keyword = {Hunter syndrome, mucopolysaccharidoses, Infusion, home therapy} }
@article{article, author = {Burton, B.K. and Guffon, N. and Roberts, J. and van der Ploeg, A.T. and Jones, S.A. and on behalf of the HOS investigators: Guelbert, N. ... and Bari\'{c}, Ivo and Bari\v{s}i\'{c}, Ingeborg and Ibrahim, J.}, year = {2010}, pages = {123-129}, DOI = {10.1016/j.ymgme.2010.06.011}, keywords = {Hunter syndrome, mucopolysaccharidoses, Infusion, home therapy}, journal = {Molecular genetics and metabolism}, doi = {10.1016/j.ymgme.2010.06.011}, volume = {101}, number = {2/3}, issn = {1096-7192}, title = {Home treatment with intravenous enzyme replacement therapy with idursulfase for mucopolysaccharidosis type II - data from the Hunter Outcome Survey}, keyword = {Hunter syndrome, mucopolysaccharidoses, Infusion, home therapy} }

Časopis indeksira:


  • Current Contents Connect (CCC)
  • Web of Science Core Collection (WoSCC)
    • Science Citation Index Expanded (SCI-EXP)
    • SCI-EXP, SSCI i/ili A&HCI
  • Scopus
  • MEDLINE

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