Ten years of the Hunter Outcome Survey (HOS) : insights, achievements, and lessons learned from a global patient registry

Detalhes bibliográficos
Autor(a) principal: Muenzer, Joseph
Data de Publicação: 2017
Outros Autores: Jones, Simon A., Tylki-Szymanska, Anna, Harmatz, Paul, Mendelsohn, Nancy J., Guffon, Nathalie, Giugliani, Roberto, Burton, Barbara K., Scarpa, Maurizio, Beck, Michael, Jangelind, Yvonne, Hernberg-Stahl, E., Larsen, Maria Paabøl, Pulles, Tom, Whiteman, David A.H.
Tipo de documento: Artigo
Idioma: eng
Título da fonte: Repositório Institucional da UFRGS
Texto Completo: http://hdl.handle.net/10183/194230
Resumo: Mucopolysaccharidosis type II (MPS II; Hunter syndrome; OMIM 309900) is a rare lysosomal storage disease with progressive multisystem manifestations caused by deficient activity of the enzyme iduronate-2-sulfatase. Diseasespecific treatment is available in the form of enzyme replacement therapy with intravenous idursulfase (Elaprase®, Shire). Since 2005, the Hunter Outcome Survey (HOS) has collected real-world, long-term data on the safety and effectiveness of this therapy, as well as the natural history of MPS II. Individuals with a confirmed diagnosis of MPS II who are untreated or who are receiving/have received treatment with idursulfase or bone marrow transplant can be enrolled in HOS. A broad range of disease- and treatment-related information is captured in the registry and, over the past decade, data from more than 1000 patients from 124 clinics in 29 countries have been collected. Evidence generated from HOS has helped to improve our understanding of disease progression in both treated and untreated patients and has extended findings from the formal clinical trials of idursulfase. As a long-term, global, observational registry, various challenges relating to data collection, entry, and analysis have been encountered. These have resulted in changes to the HOS database platform, and novel approaches to maximize the value of the information collected will also be needed in the future. The continued evolution of the registry should help to ensure that HOS provides further insights into the burden of the disease and patient care and management in the coming years.
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spelling Muenzer, JosephJones, Simon A.Tylki-Szymanska, AnnaHarmatz, PaulMendelsohn, Nancy J.Guffon, NathalieGiugliani, RobertoBurton, Barbara K.Scarpa, MaurizioBeck, MichaelJangelind, YvonneHernberg-Stahl, E.Larsen, Maria PaabølPulles, TomWhiteman, David A.H.2019-05-15T02:37:47Z20171750-1172http://hdl.handle.net/10183/194230001089715Mucopolysaccharidosis type II (MPS II; Hunter syndrome; OMIM 309900) is a rare lysosomal storage disease with progressive multisystem manifestations caused by deficient activity of the enzyme iduronate-2-sulfatase. Diseasespecific treatment is available in the form of enzyme replacement therapy with intravenous idursulfase (Elaprase®, Shire). Since 2005, the Hunter Outcome Survey (HOS) has collected real-world, long-term data on the safety and effectiveness of this therapy, as well as the natural history of MPS II. Individuals with a confirmed diagnosis of MPS II who are untreated or who are receiving/have received treatment with idursulfase or bone marrow transplant can be enrolled in HOS. A broad range of disease- and treatment-related information is captured in the registry and, over the past decade, data from more than 1000 patients from 124 clinics in 29 countries have been collected. Evidence generated from HOS has helped to improve our understanding of disease progression in both treated and untreated patients and has extended findings from the formal clinical trials of idursulfase. As a long-term, global, observational registry, various challenges relating to data collection, entry, and analysis have been encountered. These have resulted in changes to the HOS database platform, and novel approaches to maximize the value of the information collected will also be needed in the future. The continued evolution of the registry should help to ensure that HOS provides further insights into the burden of the disease and patient care and management in the coming years.application/pdfengOrphanet journal of rare diseases. London. vol. 12 (2017), 82, 9 f.Registros médicosMucopolissacaridose IITerapia de reposição de enzimasPatient registryMucopolysaccharidosis type IIHunter syndromeEnzyme replacement therapyTen years of the Hunter Outcome Survey (HOS) : insights, achievements, and lessons learned from a global patient registryEstrangeiroinfo:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersioninfo:eu-repo/semantics/openAccessreponame:Repositório Institucional da UFRGSinstname:Universidade Federal do Rio Grande do Sul (UFRGS)instacron:UFRGSTEXT001089715.pdf.txt001089715.pdf.txtExtracted Texttext/plain42799http://www.lume.ufrgs.br/bitstream/10183/194230/2/001089715.pdf.txtfb8e65a07cabb67699c8de49135c6934MD52ORIGINAL001089715.pdfTexto completo (inglês)application/pdf1646798http://www.lume.ufrgs.br/bitstream/10183/194230/1/001089715.pdf04cf7f7fcf7f0bbdb9726cc2bed865caMD5110183/1942302019-05-16 02:37:05.665269oai:www.lume.ufrgs.br:10183/194230Repositório de PublicaçõesPUBhttps://lume.ufrgs.br/oai/requestopendoar:2019-05-16T05:37:05Repositório Institucional da UFRGS - Universidade Federal do Rio Grande do Sul (UFRGS)false
dc.title.pt_BR.fl_str_mv Ten years of the Hunter Outcome Survey (HOS) : insights, achievements, and lessons learned from a global patient registry
title Ten years of the Hunter Outcome Survey (HOS) : insights, achievements, and lessons learned from a global patient registry
spellingShingle Ten years of the Hunter Outcome Survey (HOS) : insights, achievements, and lessons learned from a global patient registry
Muenzer, Joseph
Registros médicos
Mucopolissacaridose II
Terapia de reposição de enzimas
Patient registry
Mucopolysaccharidosis type II
Hunter syndrome
Enzyme replacement therapy
title_short Ten years of the Hunter Outcome Survey (HOS) : insights, achievements, and lessons learned from a global patient registry
title_full Ten years of the Hunter Outcome Survey (HOS) : insights, achievements, and lessons learned from a global patient registry
title_fullStr Ten years of the Hunter Outcome Survey (HOS) : insights, achievements, and lessons learned from a global patient registry
title_full_unstemmed Ten years of the Hunter Outcome Survey (HOS) : insights, achievements, and lessons learned from a global patient registry
title_sort Ten years of the Hunter Outcome Survey (HOS) : insights, achievements, and lessons learned from a global patient registry
author Muenzer, Joseph
author_facet Muenzer, Joseph
Jones, Simon A.
Tylki-Szymanska, Anna
Harmatz, Paul
Mendelsohn, Nancy J.
Guffon, Nathalie
Giugliani, Roberto
Burton, Barbara K.
Scarpa, Maurizio
Beck, Michael
Jangelind, Yvonne
Hernberg-Stahl, E.
Larsen, Maria Paabøl
Pulles, Tom
Whiteman, David A.H.
author_role author
author2 Jones, Simon A.
Tylki-Szymanska, Anna
Harmatz, Paul
Mendelsohn, Nancy J.
Guffon, Nathalie
Giugliani, Roberto
Burton, Barbara K.
Scarpa, Maurizio
Beck, Michael
Jangelind, Yvonne
Hernberg-Stahl, E.
Larsen, Maria Paabøl
Pulles, Tom
Whiteman, David A.H.
author2_role author
author
author
author
author
author
author
author
author
author
author
author
author
author
dc.contributor.author.fl_str_mv Muenzer, Joseph
Jones, Simon A.
Tylki-Szymanska, Anna
Harmatz, Paul
Mendelsohn, Nancy J.
Guffon, Nathalie
Giugliani, Roberto
Burton, Barbara K.
Scarpa, Maurizio
Beck, Michael
Jangelind, Yvonne
Hernberg-Stahl, E.
Larsen, Maria Paabøl
Pulles, Tom
Whiteman, David A.H.
dc.subject.por.fl_str_mv Registros médicos
Mucopolissacaridose II
Terapia de reposição de enzimas
topic Registros médicos
Mucopolissacaridose II
Terapia de reposição de enzimas
Patient registry
Mucopolysaccharidosis type II
Hunter syndrome
Enzyme replacement therapy
dc.subject.eng.fl_str_mv Patient registry
Mucopolysaccharidosis type II
Hunter syndrome
Enzyme replacement therapy
description Mucopolysaccharidosis type II (MPS II; Hunter syndrome; OMIM 309900) is a rare lysosomal storage disease with progressive multisystem manifestations caused by deficient activity of the enzyme iduronate-2-sulfatase. Diseasespecific treatment is available in the form of enzyme replacement therapy with intravenous idursulfase (Elaprase®, Shire). Since 2005, the Hunter Outcome Survey (HOS) has collected real-world, long-term data on the safety and effectiveness of this therapy, as well as the natural history of MPS II. Individuals with a confirmed diagnosis of MPS II who are untreated or who are receiving/have received treatment with idursulfase or bone marrow transplant can be enrolled in HOS. A broad range of disease- and treatment-related information is captured in the registry and, over the past decade, data from more than 1000 patients from 124 clinics in 29 countries have been collected. Evidence generated from HOS has helped to improve our understanding of disease progression in both treated and untreated patients and has extended findings from the formal clinical trials of idursulfase. As a long-term, global, observational registry, various challenges relating to data collection, entry, and analysis have been encountered. These have resulted in changes to the HOS database platform, and novel approaches to maximize the value of the information collected will also be needed in the future. The continued evolution of the registry should help to ensure that HOS provides further insights into the burden of the disease and patient care and management in the coming years.
publishDate 2017
dc.date.issued.fl_str_mv 2017
dc.date.accessioned.fl_str_mv 2019-05-15T02:37:47Z
dc.type.driver.fl_str_mv Estrangeiro
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dc.identifier.uri.fl_str_mv http://hdl.handle.net/10183/194230
dc.identifier.issn.pt_BR.fl_str_mv 1750-1172
dc.identifier.nrb.pt_BR.fl_str_mv 001089715
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dc.language.iso.fl_str_mv eng
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dc.relation.ispartof.pt_BR.fl_str_mv Orphanet journal of rare diseases. London. vol. 12 (2017), 82, 9 f.
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