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Changing paradigm in the treatment of amyloidosis: From disease-modifying drugs to anti-fibril therapy
Cardiac amyloidosis is a rare, debilitating, and usually fatal disease increasingly recognized in clinical practice despite patients presenting with non-specific symptoms of cardiomyopathy. The current standard of care (SoC) focuses on preventing further amyloid formation and deposition, either with...
Autores principales: | , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
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Frontiers Media S.A.
2022
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9808970/ https://www.ncbi.nlm.nih.gov/pubmed/36606280 http://dx.doi.org/10.3389/fcvm.2022.1073503 |
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author | Quarta, C. Cristina Fontana, Marianna Damy, Thibaud Catini, Julia Simoneau, Damien Mercuri, Michele Garcia-Pavia, Pablo Maurer, Mathew S. Palladini, Giovanni |
author_facet | Quarta, C. Cristina Fontana, Marianna Damy, Thibaud Catini, Julia Simoneau, Damien Mercuri, Michele Garcia-Pavia, Pablo Maurer, Mathew S. Palladini, Giovanni |
author_sort | Quarta, C. Cristina |
collection | PubMed |
description | Cardiac amyloidosis is a rare, debilitating, and usually fatal disease increasingly recognized in clinical practice despite patients presenting with non-specific symptoms of cardiomyopathy. The current standard of care (SoC) focuses on preventing further amyloid formation and deposition, either with anti-plasma cell dyscrasia (anti-PCD) therapies in light-chain (AL) amyloidosis or stabilizers of transthyretin (TTR) in transthyretin amyloidosis (ATTR). The SoC is supplemented by therapies to treat the complications arising from organ dysfunction; for example, heart failure, arrhythmia, and proteinuria. Advancements in treatments have improved patient survival, especially for those whose disease is detected and for whom treatment is initiated at an early stage. However, there still are many unmet medical needs, particularly for patients with severe disease for whom morbidity and mortality remain high. There currently are no approved treatments to reverse amyloid infiltration and deplete the amyloid fibrils already deposited in organs, which can continue to cause progressive dysfunction. Anti-fibril therapies aimed at removing the deposited fibrils are being investigated for safety and efficacy in improving outcomes for patients with severe disease. However, there is no clinical evidence yet that removing deposited amyloid fibrils will improve organ function, thereby improving quality of life or extending life. Nevertheless, anti-fibril therapies are actively being investigated in clinical trials to evaluate their ability to complement and synergize with current SoC. |
format | Online Article Text |
id | pubmed-9808970 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2022 |
publisher | Frontiers Media S.A. |
record_format | MEDLINE/PubMed |
spelling | pubmed-98089702023-01-04 Changing paradigm in the treatment of amyloidosis: From disease-modifying drugs to anti-fibril therapy Quarta, C. Cristina Fontana, Marianna Damy, Thibaud Catini, Julia Simoneau, Damien Mercuri, Michele Garcia-Pavia, Pablo Maurer, Mathew S. Palladini, Giovanni Front Cardiovasc Med Cardiovascular Medicine Cardiac amyloidosis is a rare, debilitating, and usually fatal disease increasingly recognized in clinical practice despite patients presenting with non-specific symptoms of cardiomyopathy. The current standard of care (SoC) focuses on preventing further amyloid formation and deposition, either with anti-plasma cell dyscrasia (anti-PCD) therapies in light-chain (AL) amyloidosis or stabilizers of transthyretin (TTR) in transthyretin amyloidosis (ATTR). The SoC is supplemented by therapies to treat the complications arising from organ dysfunction; for example, heart failure, arrhythmia, and proteinuria. Advancements in treatments have improved patient survival, especially for those whose disease is detected and for whom treatment is initiated at an early stage. However, there still are many unmet medical needs, particularly for patients with severe disease for whom morbidity and mortality remain high. There currently are no approved treatments to reverse amyloid infiltration and deplete the amyloid fibrils already deposited in organs, which can continue to cause progressive dysfunction. Anti-fibril therapies aimed at removing the deposited fibrils are being investigated for safety and efficacy in improving outcomes for patients with severe disease. However, there is no clinical evidence yet that removing deposited amyloid fibrils will improve organ function, thereby improving quality of life or extending life. Nevertheless, anti-fibril therapies are actively being investigated in clinical trials to evaluate their ability to complement and synergize with current SoC. Frontiers Media S.A. 2022-12-20 /pmc/articles/PMC9808970/ /pubmed/36606280 http://dx.doi.org/10.3389/fcvm.2022.1073503 Text en Copyright © 2022 Quarta, Fontana, Damy, Catini, Simoneau, Mercuri, Garcia-Pavia, Maurer and Palladini. https://creativecommons.org/licenses/by/4.0/This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms. |
spellingShingle | Cardiovascular Medicine Quarta, C. Cristina Fontana, Marianna Damy, Thibaud Catini, Julia Simoneau, Damien Mercuri, Michele Garcia-Pavia, Pablo Maurer, Mathew S. Palladini, Giovanni Changing paradigm in the treatment of amyloidosis: From disease-modifying drugs to anti-fibril therapy |
title | Changing paradigm in the treatment of amyloidosis: From disease-modifying drugs to anti-fibril therapy |
title_full | Changing paradigm in the treatment of amyloidosis: From disease-modifying drugs to anti-fibril therapy |
title_fullStr | Changing paradigm in the treatment of amyloidosis: From disease-modifying drugs to anti-fibril therapy |
title_full_unstemmed | Changing paradigm in the treatment of amyloidosis: From disease-modifying drugs to anti-fibril therapy |
title_short | Changing paradigm in the treatment of amyloidosis: From disease-modifying drugs to anti-fibril therapy |
title_sort | changing paradigm in the treatment of amyloidosis: from disease-modifying drugs to anti-fibril therapy |
topic | Cardiovascular Medicine |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9808970/ https://www.ncbi.nlm.nih.gov/pubmed/36606280 http://dx.doi.org/10.3389/fcvm.2022.1073503 |
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