Risk factors for severe clinical events in male and female patients with Fabry disease treated with agalsidase beta enzyme replacement therapy: Data from the Fabry Registry

Risk factors for severe clinical events in male and female patients with Fabry disease treated with agalsidase beta enzyme replacement therapy: Data from the Fabry Registry

Author Hopkin, Robert J. Google Scholar
Cabrera, Gustavo Google Scholar
Charrow, Joel Google Scholar
Lemay, Roberta Google Scholar
Martins, Ana Maria Autor UNIFESP Google Scholar
Mauer, Michael Google Scholar
Ortiz, Alberto Google Scholar
Patel, Manesh R. Google Scholar
Sims, Katherine Google Scholar
Waldek, Stephen Google Scholar
Warnock, David G. Google Scholar
Wilcox, William R. Google Scholar
Abstract Background: Fabry disease, an X-linked lysosomal storage disorder, causes intracellular accumulation of glycosphingolipids leading to progressive renal, cardiovascular, and cerebrovascular disease, and premature death. Methods: This longitudinal Fabry Registry study analyzed data from patients with Fabry disease to determine the incidence and type of severe clinical events following initiation of enzyme replacement therapy (ERT) with agalsidase beta, as well as risk factors associated with occurrence of these events. Severe events assessed included chronic dialysis, renal transplantation, cardiac events, stroke, and death. Results: The analyses included 969 male and 442 female Fabry patients. The mean age at first agalsidase beta infusion was 35 and 44, and median treatment follow-up 4.3 years and 3.2 years, respectively. Among males, cardiac events were the most common on-ERT events, followed by renal, stroke, and non-cardiac death. Among females, cardiac events were also most common followed by stroke and renal events. Patients with on-ERT events had significantly more advanced cardiac and renal disease at baseline as compared with patients without on-ERT events. Severe events were also associated with older age at ERT initiation (males and females), a history of pre-ERT events (females

approaching statistical significance in males), and a higher urinary protein/creatinine ratio (females). Approximately 65% of patients with pre-ERT events did not experience subsequent on-ERT events. Of patients without pre-ERT events, most (84% of males, 92% of females) remained event-free. Conclusions: Patients with on-ERT severe events had more advanced Fabry organ involvement at baseline than those without such events and patients who initiated ERT at a younger age had less residual risk of on-ERT events. The observed patterns of residual risk may aid clinicians in multidisciplinary monitoring of male and female patients with Fabry disease receiving ERT, and in determining the need for administration of adjunctive therapies. (C) 2016 The Authors. Published by Elsevier Inc.
Keywords Fabry disease
Severe clinical events
Risk factors
Agalsidase beta
Language English
Sponsor Sanofi Genzyme
Fabry Registry
Date 2016
Published in Molecular Genetics And Metabolism. San Diego, v. 119, n. 43497, p. 151-159, 2016.
ISSN 1096-7192 (Sherpa/Romeo, impact factor)
Publisher Academic Press Inc Elsevier Science
Extent 151-159
Origin http://dx.doi.org/10.1016/j.ymgme.2016.06.007
Access rights Closed access
Type Article
Web of Science ID WOS:000384631000020
URI http://repositorio.unifesp.br/handle/11600/51074

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