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Effectiveness of agalsidase alfa enzyme replacement in Fabry disease: cardiac outcomes after 10 years’ treatment

BACKGROUND: To explore long-term effects of agalsidase alfa on Fabry disease cardiomyopathy in adults. METHODS: Retrospective analysis of prospectively collected data at a single center in Mainz, Germany, revealed that 45 adult patients (21 men, 24 women) had received agalsidase alfa for approximate...

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Autores principales: Kampmann, Christoph, Perrin, Amandine, Beck, Michael
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2015
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4587871/
https://www.ncbi.nlm.nih.gov/pubmed/26416388
http://dx.doi.org/10.1186/s13023-015-0338-2
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author Kampmann, Christoph
Perrin, Amandine
Beck, Michael
author_facet Kampmann, Christoph
Perrin, Amandine
Beck, Michael
author_sort Kampmann, Christoph
collection PubMed
description BACKGROUND: To explore long-term effects of agalsidase alfa on Fabry disease cardiomyopathy in adults. METHODS: Retrospective analysis of prospectively collected data at a single center in Mainz, Germany, revealed that 45 adult patients (21 men, 24 women) had received agalsidase alfa for approximately 10 years. Data were extracted for cardiac and heart failure status, echocardiographic evaluations of cardiac structure and function, and renal function at treatment start and during agalsidase alfa treatment. RESULTS: After 10 years of agalsidase alfa treatment, heart failure classification had improved by at least 1 class in 22/42 patients, and angina scores were stable or improved in 41/42 patients. During treatment, no patients without left ventricular hypertrophy (LVH) at treatment initiation developed LVH, and no patients with LVH at treatment initiation showed a decline in left ventricular mass. CONCLUSIONS: Approximately 10 years of agalsidase alfa treatment appeared to have beneficial effects for controlling progression and improving some symptoms of Fabry-associated cardiomyopathy.
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spelling pubmed-45878712015-09-30 Effectiveness of agalsidase alfa enzyme replacement in Fabry disease: cardiac outcomes after 10 years’ treatment Kampmann, Christoph Perrin, Amandine Beck, Michael Orphanet J Rare Dis Research BACKGROUND: To explore long-term effects of agalsidase alfa on Fabry disease cardiomyopathy in adults. METHODS: Retrospective analysis of prospectively collected data at a single center in Mainz, Germany, revealed that 45 adult patients (21 men, 24 women) had received agalsidase alfa for approximately 10 years. Data were extracted for cardiac and heart failure status, echocardiographic evaluations of cardiac structure and function, and renal function at treatment start and during agalsidase alfa treatment. RESULTS: After 10 years of agalsidase alfa treatment, heart failure classification had improved by at least 1 class in 22/42 patients, and angina scores were stable or improved in 41/42 patients. During treatment, no patients without left ventricular hypertrophy (LVH) at treatment initiation developed LVH, and no patients with LVH at treatment initiation showed a decline in left ventricular mass. CONCLUSIONS: Approximately 10 years of agalsidase alfa treatment appeared to have beneficial effects for controlling progression and improving some symptoms of Fabry-associated cardiomyopathy. BioMed Central 2015-09-29 /pmc/articles/PMC4587871/ /pubmed/26416388 http://dx.doi.org/10.1186/s13023-015-0338-2 Text en © Kampmann et al. 2015 Open AccessThis article is distributed under the terms of the Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated.
spellingShingle Research
Kampmann, Christoph
Perrin, Amandine
Beck, Michael
Effectiveness of agalsidase alfa enzyme replacement in Fabry disease: cardiac outcomes after 10 years’ treatment
title Effectiveness of agalsidase alfa enzyme replacement in Fabry disease: cardiac outcomes after 10 years’ treatment
title_full Effectiveness of agalsidase alfa enzyme replacement in Fabry disease: cardiac outcomes after 10 years’ treatment
title_fullStr Effectiveness of agalsidase alfa enzyme replacement in Fabry disease: cardiac outcomes after 10 years’ treatment
title_full_unstemmed Effectiveness of agalsidase alfa enzyme replacement in Fabry disease: cardiac outcomes after 10 years’ treatment
title_short Effectiveness of agalsidase alfa enzyme replacement in Fabry disease: cardiac outcomes after 10 years’ treatment
title_sort effectiveness of agalsidase alfa enzyme replacement in fabry disease: cardiac outcomes after 10 years’ treatment
topic Research
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4587871/
https://www.ncbi.nlm.nih.gov/pubmed/26416388
http://dx.doi.org/10.1186/s13023-015-0338-2
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