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

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

Medicine

Research output: Contribution to journal › Article › peer-review

12 Citations (Scopus)

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.

Original language	English
Pages (from-to)	31-35
Number of pages	5
Journal	Molecular Genetics and Metabolism Reports
Volume	14
DOIs	https://doi.org/10.1016/j.ymgmr.2017.10.008
Publication status	Published - Mar 2018

Bibliographical note

Funding Information:
This work was supported by Shire International GmbH .

Funding Information:
Michael Beck has received unrestricted grants, travel support, and honoraria from Actelion, BioMarin, Genzyme, Shire, and Synageva. Derralynn Hughes has received travel/research grants and honoraria for speaking/consulting from Amicus, Protalix, Sanofi-Genzyme, and Shire. Christoph Kampmann has received research support from BioMarin and Shire; consulting/speaker fees from Amicus, BioMarin, Gore, and Shire; participated in clinical studies sponsored by Actelion, BioMarin, Genzyme, Gore, and Shire; and served on advisory committees for BioMarin and Shire. Guillem Pintos-Morell has received travel grants/honoraria for consulting/lectures from Alexion, BioMarin, and Shire. Uma Ramaswami has received travel/research grants and honoraria for lectures/consulting from Amicus, Alexion, Genzyme, Protalix, and Shire. Michael L. West has received research grants, consulting/speaker fees, and/or travel support from Actelion, Alexion, Amicus, AvroBio, Excelsior, GlaxoSmithKline, Sanofi-Genzyme, Shire, and Sumitomo. Roberto Giugliani has received consulting fees from Amicus, BioMarin, Genzyme, Shire, and Synageva/Alexion; participated in clinical trials sponsored by Amicus, BioMarin, Genzyme, GlaxoSmithKline, Shire, and Synageva/Alexion; and assisted in the design of and/or participated in clinical studies using products manufactured by Actelion, BioMarin, Genzyme, and Shire.

Publisher Copyright:
© 2017 The Authors

ASJC Scopus Subject Areas

Molecular Biology
Genetics
Endocrinology

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10.1016/j.ymgmr.2017.10.008

Cite this

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title = "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.",

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note = "Funding Information: This work was supported by Shire International GmbH . Funding Information: Michael Beck has received unrestricted grants, travel support, and honoraria from Actelion, BioMarin, Genzyme, Shire, and Synageva. Derralynn Hughes has received travel/research grants and honoraria for speaking/consulting from Amicus, Protalix, Sanofi-Genzyme, and Shire. Christoph Kampmann has received research support from BioMarin and Shire; consulting/speaker fees from Amicus, BioMarin, Gore, and Shire; participated in clinical studies sponsored by Actelion, BioMarin, Genzyme, Gore, and Shire; and served on advisory committees for BioMarin and Shire. Guillem Pintos-Morell has received travel grants/honoraria for consulting/lectures from Alexion, BioMarin, and Shire. Uma Ramaswami has received travel/research grants and honoraria for lectures/consulting from Amicus, Alexion, Genzyme, Protalix, and Shire. Michael L. West has received research grants, consulting/speaker fees, and/or travel support from Actelion, Alexion, Amicus, AvroBio, Excelsior, GlaxoSmithKline, Sanofi-Genzyme, Shire, and Sumitomo. Roberto Giugliani has received consulting fees from Amicus, BioMarin, Genzyme, Shire, and Synageva/Alexion; participated in clinical trials sponsored by Amicus, BioMarin, Genzyme, GlaxoSmithKline, Shire, and Synageva/Alexion; and assisted in the design of and/or participated in clinical studies using products manufactured by Actelion, BioMarin, Genzyme, and Shire. Publisher Copyright: {\textcopyright} 2017 The Authors",

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

Abstract

Bibliographical note

ASJC Scopus Subject Areas

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