Beck, M;
Hughes, D;
Kampmann, C;
Pintos-Morell, G;
Ramaswami, U;
West, ML;
Giugliani, R;
(2018)
Long-term outcomes with agalsidase alfa enzyme replacement therapy: Analysis using deconstructed composite events.
Molecular Genetics and Metabolism Reports
, 14
pp. 31-35.
10.1016/j.ymgmr.2017.10.008.
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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.
| Type: | Article |
|---|---|
| Title: | Long-term outcomes with agalsidase alfa enzyme replacement therapy: Analysis using deconstructed composite events |
| Location: | United States |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1016/j.ymgmr.2017.10.008 |
| Publisher version: | http://doi.org/10.1016/j.ymgmr.2017.10.008 |
| Language: | English |
| Additional information: | © 2017 The Authors. Published by Elsevier Inc. This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/BY-NC-ND/4.0/). |
| 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 |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute > Research Department of Haematology |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10039771 |
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