UCL Discovery
UCL home » Library Services » Electronic resources » UCL Discovery

The utility of the FIPI score in predicting long-term clinical outcomes in patients with Fabry disease receiving enzyme replacement therapy with agalsidase alfa

Mac Lochlainn, DJ; McKechnie, DGJ; Mehta, AB; Hughes, DA; (2018) The utility of the FIPI score in predicting long-term clinical outcomes in patients with Fabry disease receiving enzyme replacement therapy with agalsidase alfa. Molecular Genetics and Metabolism , 123 (2) pp. 154-158. 10.1016/j.ymgme.2017.10.001. Green open access

[thumbnail of McKechnie_MGM_2017_361_Revision 1_V0 (3).pdf]
Preview
Text
McKechnie_MGM_2017_361_Revision 1_V0 (3).pdf - Accepted Version

Download (202kB) | Preview

Abstract

Fabry disease is a rare X-linked lysosomal storage disorder in which there is deficiency of alpha galactosidase A. Enzyme replacement therapy (ERT) is commercially available and has been demonstrated to improve cardiac and renal outcomes. Predictive scores, such as the Fabry International Prognostic Index (FIPI), have been developed to stratify disease severity; however, these have not been validated to predict outcomes in patients receiving ERT. We show that the FIPI score at baseline can predict outcomes in a group of patients on long-term ERT.

Type: Article
Title: The utility of the FIPI score in predicting long-term clinical outcomes in patients with Fabry disease receiving enzyme replacement therapy with agalsidase alfa
Location: United States
Open access status: An open access version is available from UCL Discovery
DOI: 10.1016/j.ymgme.2017.10.001
Publisher version: http://doi.org/10.1016/j.ymgme.2017.10.001
Language: English
Additional information: © 2017 Published by Elsevier Inc. This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions.
Keywords: Agalsidase alfa, Alpha galactosidase A, Enzyme replacement therapy, Fabry disease, Long-term effectiveness
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
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > Institute of Epidemiology and Health
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > Institute of Epidemiology and Health > Primary Care and Population Health
URI: https://discovery.ucl.ac.uk/id/eprint/10044906
Downloads since deposit
190Downloads
Download activity - last month
Download activity - last 12 months
Downloads by country - last 12 months

Archive Staff Only

View Item View Item