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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...

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Autores principales: Quarta, C. Cristina, Fontana, Marianna, Damy, Thibaud, Catini, Julia, Simoneau, Damien, Mercuri, Michele, Garcia-Pavia, Pablo, Maurer, Mathew S., Palladini, Giovanni
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2022
Materias:
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.
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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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