Long-Term Galsulfase Treatment Associated With Improved Survival of Patients With Mucopolysaccharidosis VI (Maroteaux-Lamy Syndrome): 15-Year Follow-Up From the Survey Study
J. inborn errors metab. screen
;
6: e170025, 2018. tab, graf
Article
in English
|
LILACS-Express
| LILACS
| ID: biblio-1090972
ABSTRACT
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 » 104) continue to have a survival advantage over treatment-naive patients (n » 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.
Full text:
Available
Index:
LILACS (Americas)
Type of study:
Observational study
/
Prognostic study
/
Risk factors
Language:
English
Journal:
J. inborn errors metab. screen
Journal subject:
Medicina Cl¡nica
/
Patologia
Year:
2018
Type:
Article
/
Project document
Affiliation country:
Australia
/
Brazil
/
France
/
Germany
/
Portugal
/
United States
/
United kingdom
Institution/Affiliation country:
BioMarin Pharmaceutical Inc./US
/
Centro Hospitalar S. João/PT
/
Helios Dr Horst Schmidt Kliniken/DE
/
Hospital de Clinicas de Porto Alegre/BR
/
Hôpital Femme Mere Enfant/FR
/
Manchester Centre for Genomic Medicine/GB
/
UCSF/US
/
Women's and Children's Hospital/AU
Similar
MEDLINE
...
LILACS
LIS