Is Mavacamten Effective and Safe for Adolescents with Obstructive Hypertrophic Cardiomyopathy?

Clinical Takeaway

: In the first trial of mavacamten in this population, it reduced left ventricular outflow tract obstruction.

Context

Mavacamten, a cardiac myosin inhibitor, has revolutionized the management of symptomatic obstructive hypertrophic cardiomyopathy (oHCM). Randomized trials established its positive effect on left ventricular outflow tract (LVOT) obstruction and physical function, alongside an acceptable safety profile. However, these trials were limited to adults.

In a randomized, industry-sponsored trial, 44 adolescents (mean age, 14.7 years; 30% female) with symptomatic oHCM received either placebo or mavacamten initiated at a dose of 2.5 mg or 5.0 mg daily, based on body weight, and then titrated for tolerability up to 15 mg daily. Most (84%) had New York Heart Association class II symptoms. Background therapies, including β-blockers, calcium channe…

Reviewing Author

Frederick A. Masoudi, M.D., M.S.P.H., M.A.C.C., F.A.H.A.

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)