51. Long-term outcomes with agalsidase alfa enzyme replacement therapy : analysis using deconstructed composite events
- Author
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Beck, Michael, Hughes, Derralynn, Kampmann, Christoph, Pintos-Morell, Guillem, Ramaswami, Uma, West, Michael L., Giugliani, Roberto, and Universitat Autònoma de Barcelona
- Subjects
medicine.medical_specialty ,Pediatrics ,Younger age ,Short Communication ,Endocrinology, Diabetes and Metabolism ,030232 urology & nephrology ,Terapia de reposição de enzimas ,Renal function ,Left ventricular hypertrophy ,Biochemistry ,Doença de Fabry ,LVH, left ventricular hypertrophy ,Long-term effectiveness ,03 medical and health sciences ,0302 clinical medicine ,Endocrinology ,Internal medicine ,medicine ,Retrospective analysis ,Long term outcomes ,Genetics ,lcsh:QH301-705.5 ,Molecular Biology ,Agalsidase alfa ,lcsh:R5-920 ,Fabry disease ,Egfr, estimated glomerular filtration rate ,business.industry ,eGFR, estimated glomerular filtration rate ,Enzyme replacement therapy ,NE, not estimable ,medicine.disease ,ERT, enzyme replacement therapy ,lcsh:Biology (General) ,lcsh:Medicine (General) ,business ,FOS, Fabry Outcome Survey ,030217 neurology & neurosurgery - Abstract
Altres ajuts: This work was supported by Shire International GmbH. 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.
- Published
- 2017