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Hypertrophic cardiomyopathy: the future of treatment

C Vaughan Tuohy1, Sanjiv Kaul1, Howard K Song1

  • 1Oregon Health and Sciences University (OHSU), Division of Cardiovascular Medicine, Knight Cardiovascular Institute, Portland, OR, USA.

European Journal of Heart Failure
|January 11, 2020
PubMed

Insights

Hypertrophic cardiomyopathy (HCM) treatments are advancing beyond symptom management. New drugs, procedures, and gene editing offer hope for addressing the genetic causes and improving outcomes for this common inherited heart condition.

Area of Science:

  • Cardiology
  • Genetics
  • Pharmacology

Background:

  • Hypertrophic cardiomyopathy (HCM) is a prevalent genetic heart disease caused by sarcomeric mutations.
  • It leads to left ventricular hypertrophy, fibrosis, and impaired cardiac function.
  • Current therapies manage symptoms but have unmet needs for obstructive and non-obstructive HCM phenotypes.

Purpose of the Study:

  • To review recent advancements in hypertrophic cardiomyopathy (HCM) treatment.
  • To highlight novel therapeutic strategies targeting molecular and genetic aspects of HCM.
  • To discuss emerging procedural and gene-based interventions.

Main Methods:

  • Review of current literature on pharmacotherapy for HCM.
  • Analysis of innovative procedural techniques for septal reduction and mitral valve manipulation.
  • Exploration of gene-editing technologies for correcting underlying genetic defects in HCM.

Main Results:

  • Novel pharmacotherapies directly address HCM's molecular mechanisms.
  • Minimally-invasive procedures offer alternatives to traditional septal reduction therapy.
  • Gene-based therapies present potential for correcting the root genetic causes of HCM.

Conclusions:

  • Significant progress is being made in hypertrophic cardiomyopathy (HCM) treatment.
  • Emerging therapies target molecular pathways, structural abnormalities, and genetic origins.
  • These advancements promise improved management for both obstructive and non-obstructive HCM.

Keywords:
CRISPR/Cas9Genome editingHigh-intensity focused ultrasoundHypertrophic cardiomyopathyMavacamtenMitraClipMyectomyPapillary musclePercutaneous mitral valve repairRadiofrequency ablation

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