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Updated: Jul 31, 2025

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Stage-specific therapy for hypertrophic cardiomyopathy

Alessia Argirò1, Mattia Zampieri1,2, Alberto Marchi1,2

  • 1Cardiomyopathy Unit, Cardiothoracovascular Department, Careggi University Hospital, Largo Brambilla, 3 50134 Florence, Italy.

European Heart Journal Supplements : Journal of the European Society of Cardiology
|May 1, 2023
PubMed

Insights

Hypertrophic cardiomyopathy (HCM) is an inherited heart muscle disease. New targeted therapies show promise for improving symptoms and function across all disease stages.

Area of Science:

  • Cardiology
  • Genetics
  • Pharmacology

Background:

  • Hypertrophic cardiomyopathy (HCM) is the most common inherited myocardial disease.
  • It is characterized by unexplained left ventricular hypertrophy.
  • Complications include heart failure and arrhythmias like atrial fibrillation and ventricular arrhythmias.

Purpose of the Study:

  • To review current and emerging treatment strategies for hypertrophic cardiomyopathy (HCM).
  • To outline therapeutic approaches tailored to different stages of HCM natural history.
  • To highlight recent advancements in pharmacological agents targeting HCM pathophysiology.

Main Methods:

  • Review of current literature on hypertrophic cardiomyopathy (HCM) treatments.
  • Analysis of clinical trial data for novel pharmacological agents.
  • Synthesis of treatment options based on disease progression: pre-phenotype, classic phenotype, adverse remodeling, and overt dysfunction.

Main Results:

  • Established treatments include septal reduction, ICDs, and medications like beta-blockers.
  • New pharmacological agents targeting HCM pathophysiology demonstrate encouraging results.
  • Improvements in functional capacity and symptom reduction are observed in clinical trials.

Conclusions:

  • Treatment for HCM requires a phased approach, adapting to disease progression.
  • Emerging therapies offer new hope for managing symptoms and improving outcomes in HCM patients.
  • Personalized treatment strategies are crucial for addressing the diverse clinical manifestations of HCM.

Keywords:
Heart failureHypertrophic cardiomyopathyTherapy

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