Key result
Targeted therapies emerge for hypertrophic heart disease, led by cardiac myosin inhibitors and gene therapies.
Why the study?
Advancing pathophysiological understanding has identified several therapeutic targets, prompting the need to review existing and emerging tailored treatments for diverse causes of hypertrophic heart disease.
This review summarizes the evolving landscape of targeted therapies for various etiologies of hypertrophic heart disease in both pediatric and adult populations.
Answers are grounded in this paper and cite their sources.
Supports etiology-specific therapies in hypertrophic heart disease; leaves open questions on efficacy pending prospective trials.
Hypertrophic cardiomyopathy is a myocardial disease defined by an increased left ventricular wall thickness not solely explained by abnormal loading conditions. It is often genetically determined, with sarcomeric gene mutations accounting for around 50% of cases. Several conditions, including syndromic, metabolic, infiltrative, and neuromuscular diseases, may present with left ventricular hypertrophy, mimicking the hypertrophic cardiomyopathy phenotype but showing a different pathophysiology, clinical course, and outcome. Despite being rare, they are collectively responsible for a large proportion of patients presenting with hypertrophic heart disease, and their timely diagnosis can significantly impact patients' management. The understanding of disease pathophysiology has advanced over the last few years, and several therapeutic targets have been identified, leading to a new era of tailored treatments applying to different etiologies associated with left ventricular hypertrophy. This review aims to provide an overview of the existing and emerging therapies for the principal causes of hypertrophic heart disease, discussing the potential impact on patients' management and clinical outcome.
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Monda et al. (2023) conducted a review in Hypertrophic Heart Disease. Targeted therapies (e.g., cardiac myosin inhibitors, enzyme replacement, gene therapy) was evaluated. Recent advances in understanding the pathophysiology of hypertrophic heart disease have led to the development of tailored treatments, such as cardiac myosin inhibitors and gene therapies, for various etiologies.
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