Long-term outcomes with agalsidase alfa enzyme replacement therapy

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 cerebr...

Full description

Bibliographic Details
Authors: Beck, Michael, Hughes, Derralynn A|||0000-0003-4531-9173, Kampmann, Christoph, Pintos-Morell, Guillem|||0000-0002-9347-2386, Ramaswami, Uma|||0000-0002-4703-7447, West, Michael L., Giugliani, Roberto
Format: article
Publication Date:2017
Country:España
Institution:Universitat Autònoma de Barcelona
Repository:Dipòsit Digital de Documents de la UAB
Language:English
OAI Identifier:oai:ddd.uab.cat:186384
Online Access:https://ddd.uab.cat/record/186384
https://dx.doi.org/urn:doi:10.1016/j.ymgmr.2017.10.008
Access Level:Open access
Keyword:Egfr, estimated glomerular filtration rate
ERT, enzyme replacement therapy
FOS, Fabry Outcome Survey
LVH, left ventricular hypertrophy
NE, not estimable
Fabry disease
Enzyme replacement therapy
Agalsidase alfa
Long-term effectiveness
Description
Summary: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.