Is Mavacamten Effective for Nonobstructive Hypertrophic Cardiomyopathy?

Clinical Takeaway

: The myosin inhibitor falls short, in contrast with its benefits in the obstructive phenotype.

Context

Hypertrophic cardiomyopathy (HCM) is phenotypically heterogeneous, including the extent to which dynamic left ventricular outflow tract (LVOT) obstruction is present. Some individuals with HCM do not have significant LVOT obstruction but are symptomatic — in some cases, severely so. The cardiac myosin inhibitor mavacamten, when used to treat the obstructive phenotype, reduces LVOT gradient and improves symptoms, functional capacity, and quality of life; however, its efficacy in individuals with nonobstructive HCM is unclear.

In a manufacturer-funded trial, investigators randomized 580 adults with symptomatic nonobstructive HCM and preserved left ventricular ejection fraction to receive either mavacamten (initially 5 mg daily, titrated…

Reviewing Author

Frederick Masoudi, MD, MSPH, MACC, 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)