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Hypertrophic cardiomyopathy (HCM) is a heterogenous, predominantly autosomal dominant genetic heart disease with an estimated prevalence of 0.2% to 0.6% in the general population. Clinically, HCM ranges from being asymptomatic or causing mild exercise intolerance to result in severe debilitating symptoms, heart failure, and sudden cardiac death. Currently, HCM management is based on lifestyle modifications, familial screening along with genetic counseling, symptom control through pharmacotherapy, risk stratification for sudden cardiac death with or without defibrillator implantation, septal reduction therapy, and heart transplantation in select patients. It was not until recently that targeted medical therapies for HCM, such as cardiac myosin inhibitors (CMI), were studied in multicenter randomized controlled trials. Mavacamten is the first-in-class Food and Drug Administration-approved CMI that explicitly targets the pathophysiology of HCM with additional therapies like Aficamten currently being studied. In this chapter, we discuss the currently available literature related to different CMIs. We also discuss future directions in HCM pharmacotherapy and highlight unmet needs for this patient population.

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Current Evidence in Cardiac Myosin Inhibitors and Future Directions in Medical Therapy

  • 章节

  • 发表日期：

    2025年

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  • Milind Y. Desai,
  • Iacopo Olivotto,
  • Ahmad Masri

摘要

Hypertrophic cardiomyopathy (HCM) is a heterogenous, predominantly autosomal dominant genetic heart disease with an estimated prevalence of 0.2% to 0.6% in the general population. Clinically, HCM ranges from being asymptomatic or causing mild exercise intolerance to result in severe debilitating symptoms, heart failure, and sudden cardiac death. Currently, HCM management is based on lifestyle modifications, familial screening along with genetic counseling, symptom control through pharmacotherapy, risk stratification for sudden cardiac death with or without defibrillator implantation, septal reduction therapy, and heart transplantation in select patients. It was not until recently that targeted medical therapies for HCM, such as cardiac myosin inhibitors (CMI), were studied in multicenter randomized controlled trials. Mavacamten is the first-in-class Food and Drug Administration-approved CMI that explicitly targets the pathophysiology of HCM with additional therapies like Aficamten currently being studied. In this chapter, we discuss the currently available literature related to different CMIs. We also discuss future directions in HCM pharmacotherapy and highlight unmet needs for this patient population.