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Published on: September 25, 2017
Pharmacological treatment of hypertrophic cardiomyopathy: current practice and novel perspectives
Enrico Ammirati1, Rachele Contri2, Raffaele Coppini3
1De Gasperis Cardio Centre, ASST Grande Ospedale Metropolitano Niguarda, Milan, Italy.
Insights
Hypertrophic cardiomyopathy (HCM) treatment is evolving from generic drugs to targeted therapies. New research promises disease-specific pharmacological treatments for this rare heart condition.
Area of Science:
- Cardiology
- Pharmacology
- Translational Research
Background:
- Hypertrophic cardiomyopathy (HCM) has historically lacked disease-specific treatments, relying on drugs developed for other conditions.
- Current management of HCM involves established therapies, requiring expertise for complex cases and decisions on when treatment is not indicated.
Purpose of the Study:
- To review current time-honored therapies for hypertrophic cardiomyopathy (HCM) in a practical, real-world context.
- To assess specific challenges in HCM management, including dynamic outflow obstruction, end-stage heart failure, and arrhythmia prevention.
- To discuss emerging pharmacological approaches and their implications for future HCM research.
Main Methods:
- Review of existing literature on hypertrophic cardiomyopathy (HCM) therapies.
- Analysis of practical application, including dosing, titration, and risk-benefit profiles of current agents.
- Discussion of novel therapeutic strategies currently in clinical research.
Main Results:
- Established therapies can manage HCM manifestations but require specialized knowledge for challenging situations.
- Specific HCM-related issues like outflow obstruction, heart failure progression, and arrhythmias necessitate tailored treatment strategies.
- Significant scientific and economic interest is driving the development of new HCM-specific drugs.
Conclusions:
- Hypertrophic cardiomyopathy (HCM) is poised for advancements in disease-specific pharmacological treatments.
- Emerging therapies targeting myocardial metabolism, sodium current, and myosin function show promise.
- Future research directions include further clinical investigation of these novel approaches for HCM.
Abstract:
Hypertrophic cardiomyopathy (HCM) is entering a phase of intense translational research that holds promise for major advances in disease-specific pharmacological therapy. For over 50 years, however, HCM has largely remained an orphan disease, and patients are still treated with old drugs developed for other conditions. While judicious use of the available armamentarium may control the clinical manifestations of HCM in most patients, specific experience is required in challenging situations, including deciding when not to treat. The present review revisits the time-honoured therapies available for HCM, in a practical perspective reflecting real-world scenarios. Specific agents are presented with doses, titration strategies, pros and cons. Peculiar HCM dilemmas such as treatment of dynamic outflow obstruction, heart failure caused by end-stage progression and prevention of atrial fibrillation and ventricular arrhythmias are assessed. In the near future, the field of HCM drug therapy will rapidly expand, based on ongoing efforts. Approaches such as myocardial metabolic modulation, late sodium current inhibition and allosteric myosin inhibition have moved from pre-clinical to clinical research, and reflect a surge of scientific as well as economic interest by academia and industry alike. These exciting developments, and their implications for future research, are discussed.
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