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

Michael Beck; Derralynn Hughes; Christoph Kampmann; Guillem Pintos-Morell; Uma Ramaswami; Michael L West; Roberto Giugliani

doi:10.1016/j.ymgmr.2017.10.008

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

Mol Genet Metab Rep. 2017 Nov 9:14:31-35. doi: 10.1016/j.ymgmr.2017.10.008. eCollection 2018 Mar.

Authors

Michael Beck¹, Derralynn Hughes², Christoph Kampmann¹, Guillem Pintos-Morell³, Uma Ramaswami², Michael L West⁴, Roberto Giugliani⁵

Affiliations

¹ University Medical Center, Mainz, Germany.
² Royal Free London NHS Foundation Trust, University College London, London, UK.
³ University Hospital and Research Institute "Germans Trias i Pujol", Badalona, Universitat Autònoma de Barcelona, Barcelona, Spain.
⁴ Department of Medicine, Dalhousie University, Halifax, NS, Canada.
⁵ Medical Genetics Service HCPA/Department of Genetics UFRGS and INAGEMP, Porto Alegre, Brazil.

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: Agalsidase alfa; ERT, enzyme replacement therapy; Enzyme replacement therapy; FOS, Fabry Outcome Survey; Fabry disease; LVH, left ventricular hypertrophy; Long-term effectiveness; NE, not estimable; eGFR, estimated glomerular filtration rate.