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Published on: November 2, 2020
An overview of the treatments for hypertrophic cardiomyopathy
Wenna Xu1, Fuyu Zhu1, Yue Zhang2
1Department of Cardiology, The Affiliated Suzhou Hospital of Nanjing Medical University, Suzhou Municipal Hospital, Gusu School, Nanjing Medical University, Suzhou, Jiangsu, China.
Insights
Hypertrophic cardiomyopathy (HCM), a common inherited heart disease, is now treatable, but significant therapeutic needs remain. This review covers HCM pathogenesis, treatments, and future directions.
Area of Science:
- Cardiology
- Genetics
- Molecular Biology
Background:
- Hypertrophic cardiomyopathy (HCM) affects ~0.2% of the population globally.
- It is primarily caused by sarcomere gene mutations leading to left ventricular hypertrophy (LVH).
- Pathological hallmarks include cardiomyocyte hypertrophy, disarray, and fibrosis, causing diastolic dysfunction, outflow tract obstruction, and arrhythmias.
Purpose of the Study:
- To review current advances in Hypertrophic Cardiomyopathy (HCM) treatment.
- To discuss the pathogenesis, classification, and therapeutic strategies for HCM.
- To highlight unmet needs and future prospects in HCM management.
Main Methods:
- Comprehensive literature review of over 100 references from the past four decades.
- Analysis of current pharmacotherapy, invasive treatments, and emerging gene therapies for HCM.
- Synthesis of information on HCM pathogenesis, clinical presentation, and treatment outcomes.
Main Results:
- HCM, once considered untreatable, now has significantly reduced mortality rates due to improved medical standards.
- Pharmacological and invasive treatments have advanced, but challenges in managing symptoms and complications persist.
- Gene therapy represents a promising future avenue for treating the genetic basis of HCM.
Conclusions:
- While therapeutic options for HCM have improved, substantial unmet needs remain.
- A multi-faceted approach combining pharmacotherapy, invasive procedures, and potentially gene therapy is crucial for optimal HCM management.
- Continued research into pathogenesis and novel therapeutic strategies is essential for addressing the complexities of HCM.
Abstract:
Hypertrophic cardiomyopathy (HCM) is a very prevalent inherited disease with a wide global distribution and a prevalence rate of approximately 0.2% in the general population. Left ventricular hypertrophy (LVH) caused by sarcomere mutation is the primary reason of HCM. The histopathology feature is that cardiomyocyte hypertrophy, myocyte disorder and myocardial fibrosis lead to diminished diastolic function, left ventricular outflow tract obstruction (LVOTO) and arrhythmia, all of which result in serious cardiac complications. Previously, HCM was considered a malignant disease that was almost untreatable. With the improvement of medical standards and increasing awareness of HCM, it has become a highly treatable disease in contemporary times, with a significant decrease in mortality rates. However, there are still significant unmet requirements in the therapy of HCM. This paper draws on more than 100 references from the past four decades and summarizes current advances in the treatment of HCM. The article will review the pathogenesis and types, recent development in pharmacotherapy, invasive treatments and gene therapies, as well as dilemma and future development of HCM.
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