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Valsartan in early-stage hypertrophic cardiomyopathy: a randomized phase 2 trial

Carolyn Y Ho1, Sharlene M Day2,3, Anna Axelsson4

  • 1Department of Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, MA, USA. cho@bwh.harvard.edu.

Nature Medicine
|September 24, 2021
PubMed

Insights

Valsartan treatment significantly improved cardiac structure and function in early-stage hypertrophic cardiomyopathy (HCM) patients. This study suggests valsartan may attenuate disease progression in HCM, offering a new therapeutic avenue.

Area of Science:

  • Cardiology
  • Genetics
  • Pharmacology

Background:

  • Hypertrophic cardiomyopathy (HCM) is a genetic heart muscle disease characterized by left ventricular hypertrophy and fibrosis.
  • Current treatments for HCM focus on symptom management, lacking therapies to modify disease progression.
  • Pathogenic variants in sarcomeric genes are common causes of HCM, leading to heart failure and arrhythmias.

Purpose of the Study:

  • To evaluate the safety and efficacy of valsartan in slowing disease progression in early-stage sarcomeric HCM.
  • To assess valsartan's impact on cardiac structure and function in patients with early HCM.
  • To determine if valsartan can serve as a disease-modifying therapy for HCM.

Main Methods:

  • A multi-center, double-blind, placebo-controlled phase 2 clinical trial.
  • 178 participants with early-stage sarcomeric HCM were randomized to receive valsartan or placebo for 2 years.
  • Primary outcome: a composite z-score integrating changes in LV wall thickness, mass, volumes, left atrial volume, diastolic/systolic velocities, and cardiac biomarkers.

Main Results:

  • Valsartan significantly improved cardiac structure and function compared to placebo (P=0.001).
  • The composite z-score showed a significant improvement in the valsartan group, meeting the primary endpoint.
  • Valsartan treatment was well-tolerated in both adult and pediatric participants.

Conclusions:

  • Valsartan demonstrated efficacy in attenuating disease progression in early-stage sarcomeric HCM.
  • This study highlights valsartan as a potential accessible and safe medication to modify HCM progression.
  • Further research may establish valsartan as a standard of care for early HCM management.

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