Long-term galsufase treatment associated with improved survival of patients with mucopolysaccharidosis VI (Maroteaux-Lamy Syndrome) : 15-year follow-up from the survey study
dc.contributor.author | Quartel, Adrian | pt_BR |
dc.contributor.author | Harmatz, Paul | pt_BR |
dc.contributor.author | Lampe, Christina | pt_BR |
dc.contributor.author | Guffon, Nathalie | pt_BR |
dc.contributor.author | Ketteridge, David | pt_BR |
dc.contributor.author | Teles, Elisa Leão | pt_BR |
dc.contributor.author | Jones, Simon A. | pt_BR |
dc.contributor.author | Giugliani, Roberto | pt_BR |
dc.date.accessioned | 2019-05-29T02:43:17Z | pt_BR |
dc.date.issued | 2018 | pt_BR |
dc.identifier.issn | 2326-4594 | pt_BR |
dc.identifier.uri | http://hdl.handle.net/10183/194695 | pt_BR |
dc.description.abstract | Mucopolysaccharidosis VI (MPS VI) is a progressive lysosomal storage disorder with multiorgan and multisystemic pathology. Currently, galsulfase enzyme replacement therapy (ERT) is the only approved treatment for MPS VI. A crosssectional survey study of 121 patients with MPS VI conducted in 2001 to 2002 and a 10-year follow-up study of the same patients (resurvey study; ClinicalTrials.gov NCT01387854) found that those receiving galsulfase at any time showed physical improvements and a lower mortality rate (16.5%) versus treatment-naive patients (50%). After *15 years, galsulfasetreated patients (n ¼ 104) continue to have a survival advantage over treatment-naive patients (n ¼ 14), as demonstrated by a 24% versus 57% mortality rate. This survival advantage is further supported by data from the commercial use of galsulfase (2005-2016), which show a 5-year mortality rate for galsulfase-treated patients of 12.5%. Together, these findings suggest that galsulfase ERT can increase life expectancies for patients with MPS VI over a period of at least 15 years. | en |
dc.format.mimetype | application/pdf | pt_BR |
dc.language.iso | eng | pt_BR |
dc.relation.ispartof | Journal of inborn errors of metabolism & screeing. Thousand Oaks. vol. 5 (2017), 11 f. | pt_BR |
dc.rights | Open Access | en |
dc.subject | Mucopolysaccharidosis VI | en |
dc.subject | Terapia de reposição de enzimas | pt_BR |
dc.subject | Mucopolissacaridose IV | pt_BR |
dc.subject | Maroteaux-Lamy syndrome | en |
dc.subject | Enzyme replacement therapy | en |
dc.subject | Arylsulfatase B | en |
dc.subject | Galsulfase | en |
dc.subject | Survey study | en |
dc.title | Long-term galsufase treatment associated with improved survival of patients with mucopolysaccharidosis VI (Maroteaux-Lamy Syndrome) : 15-year follow-up from the survey study | pt_BR |
dc.type | Artigo de periódico | pt_BR |
dc.identifier.nrb | 001090042 | pt_BR |
dc.type.origin | Estrangeiro | pt_BR |
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