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Hypertrophic Cardiomyopathy: From Medical Treatment to Advanced Heart Failure Therapies

Matylda Mazur1, Wojciech Braksator2, Eric Popjes3

  • 1Heart and Vascular Institute, Kaufman Center for Heart Failure, Cleveland Clinic Foundation, 9500 Euclid Avenue, Cleveland, OH, 44195, USA. matyldamazur@gmail.com.

Current Cardiology Reports
|July 11, 2024
PubMed

Insights

Mavacamten offers symptom relief for hypertrophic cardiomyopathy (HCM) patients, potentially avoiding surgery. Heart transplantation remains crucial for advanced cases, but organ allocation challenges persist for HCM patients.

Area of Science:

  • Cardiology
  • Genetics
  • Pharmacology

Background:

  • Hypertrophic cardiomyopathy (HCM) is a complex genetic heart condition with varied clinical presentations.
  • A significant minority of HCM patients develop severe symptoms refractory to medical treatment.
  • Debate exists regarding optimal therapies and heart transplantation (HT) listing for HCM patients.

Purpose of the Study:

  • To review novel medical therapies for HCM.
  • To discuss challenges in heart transplantation listing for HCM patients.
  • To evaluate current treatment paradigms and future directions in HCM management.

Main Methods:

  • Review of recent clinical trials and FDA approvals.
  • Analysis of treatment outcomes for medical and surgical interventions.
  • Evaluation of heart transplantation outcomes and waitlist challenges.

Main Results:

  • Mavacamten, a cardiac myosin inhibitor, is FDA-approved, providing symptom relief and potentially delaying/avoiding septal reduction therapy for HCM with left ventricular outflow obstruction (LVOTO).
  • Heart transplantation is the gold standard for refractory end-stage heart failure in HCM, but concerns about organ allocation persist.
  • Post-HT outcomes are favorable, yet HT waitlist mortality remains high. Left ventricular assist devices are a viable option for select HCM patients.

Conclusions:

  • Mavacamten represents a significant advancement in medical therapy for symptomatic HCM with LVOTO.
  • Addressing organ allocation disparities is critical to ensure equitable access to heart transplantation for HCM patients.
  • Continued research and therapeutic innovation are essential for improving long-term outcomes in hypertrophic cardiomyopathy.
Abstract

Keywords:
Cardiac myosin inhibitorsHeart failureHeart transplantHypertrophic cardiomyopathyLeft ventricular assist device

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