A New Treatment for Obstructive Hypertrophic Cardiomyopathy?

A phase 2 study yields promising, although preliminary, results for this difficult-to-treat disorder.

The obstructive phenotype of hypertrophic cardiomyopathy (HCM) entails left ventricular hypercontractility with left ventricular outflow tract (LVOT) obstruction and can result in exercise intolerance, exertional dyspnea, chest pain, or fatigue despite management with negative inotropes like β-blockers and nondihydropyridine calcium channel blockers. In the disorder, mutations in the cardiac myosin heavy chain are thought to contribute to hypercontractility and abnormal myocardial compliance. The non-FDA-approved drug mavacamten is a small molecule that reduces hypercontractility by inhibiting the binding of β-cardiac myosin to actin. These researchers conducted a manufacturer-funded, open-label, phase 2 study (PIONEER-HCM; NCT02842242) exa…

Reviewing Author

Frederick A. Masoudi, MD, MSPH, FACC, FAHA

Disclosures

Consultant/Advisory BoardBristol Myers Squibb; CPC Clinical Research

Grant/Research SupportNational Heart, Lung, and Blood Institute

Editorial BoardsUpToDate; American College of Cardiology Self-Assessment Program (SAP)

Leadership Positions in Professional SocietiesAmerican College of Cardiology (Chair, Innovations Committee)

Frederick A. Masoudi, MD, MSPH, FACC, FAHA

Disclosures

Consultant/Advisory BoardBristol Myers Squibb; CPC Clinical Research

Grant/Research SupportNational Heart, Lung, and Blood Institute

Editorial BoardsUpToDate; American College of Cardiology Self-Assessment Program (SAP)

Leadership Positions in Professional SocietiesAmerican College of Cardiology (Chair, Innovations Committee)

A New Treatment for Obstructive Hypertrophic Cardiomyopathy?

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Reviewing Author