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.
CITATION STYLE
Quartel, A., Harmatz, P. R., Lampe, C., Guffon, N., Ketteridge, D., Leão-Teles, E., … Giugliani, R. (2018). Long-term galsulfase treatment associated with improved survival of patients with mucopolysaccharidosis VI (Maroteaux-Lamy Syndrome): 15-year follow-up from the survey study. Journal of Inborn Errors of Metabolism and Screening, 6. https://doi.org/10.1177/2326409818755800
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