Cargando…

Emerging Therapeutics for the Treatment of Light Chain and Transthyretin Amyloidosis

Cardiac amyloidosis is a restrictive cardiomyopathy that results from the deposition of misfolded light chain or transthyretin proteins, most commonly, in cardiac tissue. Traditionally, treatment options for light chain (AL) and transthyretin (ATTR) amyloidosis have been limited. However, there are...

Descripción completa

Detalles Bibliográficos
Autores principales: Zhang, Kathleen W., Stockerl-Goldstein, Keith E., Lenihan, Daniel J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6609907/
https://www.ncbi.nlm.nih.gov/pubmed/31312767
http://dx.doi.org/10.1016/j.jacbts.2019.02.002
_version_ 1783432406104014848
author Zhang, Kathleen W.
Stockerl-Goldstein, Keith E.
Lenihan, Daniel J.
author_facet Zhang, Kathleen W.
Stockerl-Goldstein, Keith E.
Lenihan, Daniel J.
author_sort Zhang, Kathleen W.
collection PubMed
description Cardiac amyloidosis is a restrictive cardiomyopathy that results from the deposition of misfolded light chain or transthyretin proteins, most commonly, in cardiac tissue. Traditionally, treatment options for light chain (AL) and transthyretin (ATTR) amyloidosis have been limited. However, there are now multiple novel therapeutics in development and several therapeutics recently approved that promise to revolutionize clinical management of AL and ATTR. Most of these agents disrupt specific stages of amyloidogenesis such as light chain or transthyretin protein production, formation of amyloidogenic intermediates, or amyloid fibril aggregation. Others aim to remove existing amyloid tissue deposits using monoclonal antibody technology. Although these advances represent an important step forward in the care of cardiac amyloidosis patients, additional studies are needed to define the optimal treatment paradigms for AL and ATTR and to validate clinical, imaging, or serum biomarker strategies that may confirm a cardiac response to therapy.
format Online
Article
Text
id pubmed-6609907
institution National Center for Biotechnology Information
language English
publishDate 2019
publisher Elsevier
record_format MEDLINE/PubMed
spelling pubmed-66099072019-07-16 Emerging Therapeutics for the Treatment of Light Chain and Transthyretin Amyloidosis Zhang, Kathleen W. Stockerl-Goldstein, Keith E. Lenihan, Daniel J. JACC Basic Transl Sci STATE-OF-THE-ART REVIEW Cardiac amyloidosis is a restrictive cardiomyopathy that results from the deposition of misfolded light chain or transthyretin proteins, most commonly, in cardiac tissue. Traditionally, treatment options for light chain (AL) and transthyretin (ATTR) amyloidosis have been limited. However, there are now multiple novel therapeutics in development and several therapeutics recently approved that promise to revolutionize clinical management of AL and ATTR. Most of these agents disrupt specific stages of amyloidogenesis such as light chain or transthyretin protein production, formation of amyloidogenic intermediates, or amyloid fibril aggregation. Others aim to remove existing amyloid tissue deposits using monoclonal antibody technology. Although these advances represent an important step forward in the care of cardiac amyloidosis patients, additional studies are needed to define the optimal treatment paradigms for AL and ATTR and to validate clinical, imaging, or serum biomarker strategies that may confirm a cardiac response to therapy. Elsevier 2019-06-24 /pmc/articles/PMC6609907/ /pubmed/31312767 http://dx.doi.org/10.1016/j.jacbts.2019.02.002 Text en © 2019 The Authors http://creativecommons.org/licenses/by-nc-nd/4.0/ This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).
spellingShingle STATE-OF-THE-ART REVIEW
Zhang, Kathleen W.
Stockerl-Goldstein, Keith E.
Lenihan, Daniel J.
Emerging Therapeutics for the Treatment of Light Chain and Transthyretin Amyloidosis
title Emerging Therapeutics for the Treatment of Light Chain and Transthyretin Amyloidosis
title_full Emerging Therapeutics for the Treatment of Light Chain and Transthyretin Amyloidosis
title_fullStr Emerging Therapeutics for the Treatment of Light Chain and Transthyretin Amyloidosis
title_full_unstemmed Emerging Therapeutics for the Treatment of Light Chain and Transthyretin Amyloidosis
title_short Emerging Therapeutics for the Treatment of Light Chain and Transthyretin Amyloidosis
title_sort emerging therapeutics for the treatment of light chain and transthyretin amyloidosis
topic STATE-OF-THE-ART REVIEW
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6609907/
https://www.ncbi.nlm.nih.gov/pubmed/31312767
http://dx.doi.org/10.1016/j.jacbts.2019.02.002
work_keys_str_mv AT zhangkathleenw emergingtherapeuticsforthetreatmentoflightchainandtransthyretinamyloidosis
AT stockerlgoldsteinkeithe emergingtherapeuticsforthetreatmentoflightchainandtransthyretinamyloidosis
AT lenihandanielj emergingtherapeuticsforthetreatmentoflightchainandtransthyretinamyloidosis