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Pharmacological Management of Hypertrophic Cardiomyopathy: From Bench to Bedside

Hypertrophic cardiomyopathy (HCM), the most common inherited heart disease, is still orphan of a specific drug treatment. The erroneous consideration of HCM as a rare disease has hampered the design and conduct of large, randomized trials in the last 50 years, and most of the indications in the curr...

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Autores principales: Palandri, Chiara, Santini, Lorenzo, Argirò, Alessia, Margara, Francesca, Doste, Ruben, Bueno-Orovio, Alfonso, Olivotto, Iacopo, Coppini, Raffaele
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer International Publishing 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9209358/
https://www.ncbi.nlm.nih.gov/pubmed/35696053
http://dx.doi.org/10.1007/s40265-022-01728-w
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author Palandri, Chiara
Santini, Lorenzo
Argirò, Alessia
Margara, Francesca
Doste, Ruben
Bueno-Orovio, Alfonso
Olivotto, Iacopo
Coppini, Raffaele
author_facet Palandri, Chiara
Santini, Lorenzo
Argirò, Alessia
Margara, Francesca
Doste, Ruben
Bueno-Orovio, Alfonso
Olivotto, Iacopo
Coppini, Raffaele
author_sort Palandri, Chiara
collection PubMed
description Hypertrophic cardiomyopathy (HCM), the most common inherited heart disease, is still orphan of a specific drug treatment. The erroneous consideration of HCM as a rare disease has hampered the design and conduct of large, randomized trials in the last 50 years, and most of the indications in the current guidelines are derived from small non-randomized studies, case series, or simply from the consensus of experts. Guideline-directed therapy of HCM includes non-selective drugs such as disopyramide, non-dihydropyridine calcium channel blockers, or β-adrenergic receptor blockers, mainly used in patients with symptomatic obstruction of the outflow tract. Following promising preclinical studies, several drugs acting on potential HCM-specific targets were tested in patients. Despite the huge efforts, none of these studies was able to change clinical practice for HCM patients, because tested drugs were proven to be scarcely effective or hardly tolerated in patients. However, novel compounds have been developed in recent years specifically for HCM, addressing myocardial hypercontractility and altered energetics in a direct manner, through allosteric inhibition of myosin. In this paper, we will critically review the use of different classes of drugs in HCM patients, starting from “old” established agents up to novel selective drugs that have been recently trialed in patients.
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spelling pubmed-92093582022-06-22 Pharmacological Management of Hypertrophic Cardiomyopathy: From Bench to Bedside Palandri, Chiara Santini, Lorenzo Argirò, Alessia Margara, Francesca Doste, Ruben Bueno-Orovio, Alfonso Olivotto, Iacopo Coppini, Raffaele Drugs Review Article Hypertrophic cardiomyopathy (HCM), the most common inherited heart disease, is still orphan of a specific drug treatment. The erroneous consideration of HCM as a rare disease has hampered the design and conduct of large, randomized trials in the last 50 years, and most of the indications in the current guidelines are derived from small non-randomized studies, case series, or simply from the consensus of experts. Guideline-directed therapy of HCM includes non-selective drugs such as disopyramide, non-dihydropyridine calcium channel blockers, or β-adrenergic receptor blockers, mainly used in patients with symptomatic obstruction of the outflow tract. Following promising preclinical studies, several drugs acting on potential HCM-specific targets were tested in patients. Despite the huge efforts, none of these studies was able to change clinical practice for HCM patients, because tested drugs were proven to be scarcely effective or hardly tolerated in patients. However, novel compounds have been developed in recent years specifically for HCM, addressing myocardial hypercontractility and altered energetics in a direct manner, through allosteric inhibition of myosin. In this paper, we will critically review the use of different classes of drugs in HCM patients, starting from “old” established agents up to novel selective drugs that have been recently trialed in patients. Springer International Publishing 2022-06-13 2022 /pmc/articles/PMC9209358/ /pubmed/35696053 http://dx.doi.org/10.1007/s40265-022-01728-w Text en © The Author(s) 2022 https://creativecommons.org/licenses/by-nc/4.0/Open AccessThis article is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License, which permits any non-commercial use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by-nc/4.0/ (https://creativecommons.org/licenses/by-nc/4.0/) .
spellingShingle Review Article
Palandri, Chiara
Santini, Lorenzo
Argirò, Alessia
Margara, Francesca
Doste, Ruben
Bueno-Orovio, Alfonso
Olivotto, Iacopo
Coppini, Raffaele
Pharmacological Management of Hypertrophic Cardiomyopathy: From Bench to Bedside
title Pharmacological Management of Hypertrophic Cardiomyopathy: From Bench to Bedside
title_full Pharmacological Management of Hypertrophic Cardiomyopathy: From Bench to Bedside
title_fullStr Pharmacological Management of Hypertrophic Cardiomyopathy: From Bench to Bedside
title_full_unstemmed Pharmacological Management of Hypertrophic Cardiomyopathy: From Bench to Bedside
title_short Pharmacological Management of Hypertrophic Cardiomyopathy: From Bench to Bedside
title_sort pharmacological management of hypertrophic cardiomyopathy: from bench to bedside
topic Review Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9209358/
https://www.ncbi.nlm.nih.gov/pubmed/35696053
http://dx.doi.org/10.1007/s40265-022-01728-w
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