Long-term outcomes with agalsidase alfa enzyme replacement therapy: Analysis using deconstructed composite events

Abstract

This is a retrospective analysis of Fabry Outcome Survey data from children/adults (n = 677) receiving agalsidase alfa enzyme replacement therapy for a median of 3 years, examining cerebrovascular, cardiac, and renal morbidity endpoints separately. Cardiac events occurred at younger ages than cerebrovascular or renal events, cerebrovascular events were more frequent in females than males, and males were more likely to experience cardiac and renal events at a younger age than females.

Keywords

• Fabry disease
• Enzyme replacement therapy
• Agalsidase alfa
• Long-term effectiveness