Emerging pharmacologic and structural therapies for hypertrophic cardiomyopathy
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Published on: June 14, 2016
Daniel J Philipson1, Eugene C DePasquale2, Eric H Yang3
1Department of Medicine, UCLA, 200 UCLA Medical Plaza Suite 420, Los Angeles, CA, 90095, USA. dphilipson@mednet.ucla.edu.
Insights
New therapies are emerging for hypertrophic cardiomyopathy, a common inherited heart condition. These innovative treatments aim to address the disease
Area of Science:
- Cardiology
- Genetics
- Pharmacology
Background:
- Hypertrophic cardiomyopathy (HCM) is the most prevalent inherited cardiac disorder.
- Current treatments for HCM primarily manage symptoms and do not alter disease progression.
- Established interventions for refractory outflow tract obstruction include surgical myectomy and alcohol septal ablation.
Purpose of the Study:
- To review novel and emerging therapeutic strategies for hypertrophic cardiomyopathy.
- To highlight advancements targeting the molecular and hemodynamic underpinnings of HCM.
Main Methods:
- Review of current literature on innovative hypertrophic cardiomyopathy therapeutics.
- Categorization of emerging treatments including pharmacologic and structural interventions.
Main Results:
- Emerging pharmacologic therapies include: attenuation of sarcomeric calcium sensitivity, cardiac myosin inhibition, metabolic modulation, and renin-angiotensin-aldosterone system inhibition.
- Structural interventions under development include percutaneous mitral valve plication and endocardial radiofrequency ablation.
Conclusions:
- These novel therapeutic strategies represent significant advancements in hypertrophic cardiomyopathy treatment.
- Ongoing development holds promise for improved management of hypertrophic cardiomyopathy patients.
Abstract:
Hypertrophic cardiomyopathy is the most common inherited heart disease. Although it was first described over 50 years ago, there has been little in the way of novel disease-specific therapeutic development for these patients. Current treatment practice largely aims at symptomatic control using old drugs made for other diseases and does little to modify the disease course. Septal reduction by surgical myectomy or percutaneous alcohol septal ablation are well-established treatments for pharmacologic-refractory left ventricular outflow tract obstruction in hypertrophic cardiomyopathy patients. In recent years, there has been a relative surge in the development of innovative therapeutics, which aim to target the complex molecular pathophysiology and resulting hemodynamics that underlie hypertrophic cardiomyopathy. Herein, we review the new and emerging therapeutics for hypertrophic cardiomyopathy, which include pharmacologic attenuation of sarcomeric calcium sensitivity, allosteric inhibition of cardiac myosin, myocardial metabolic modulation, and renin-angiotensin-aldosterone system inhibition, as well as structural intervention by percutaneous mitral valve plication and endocardial radiofrequency ablation of septal hypertrophy. In conclusion, while further development of these therapeutic strategies is ongoing, they each mark a significant and promising advancement in treatment for hypertrophic cardiomyopathy patients.
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