Monitoring Treatment Response and Advancing Therapy in Obstructive HCM

This episode, titled Monitoring Treatment Response and Advancing Therapy in Obstructive HCM, features panelists/expert [specialty] discussed the following critical questions:

How are you monitoring response to therapy after initiation on beta-blockers or calcium channel blockers?

The first cardiac myosin inhibitor, mavacamten, was FDA-approved in 2022 for symptomatic obstructive hypertrophic cardiomyopathy. Please describe the key clinical trials, EXPLORER-HCM and VALOR-HCM, and their associated safety and efficacy data that led to its FDA-approval?

Led by the moderator, the cardiology experts examined response to beta-blockers or calcium channel blockers, which is typically monitored through changes in symptoms, exercise tolerance, heart rate, and echocardiographic measures such as left ventricular outflow tract gradients. Follow-up assessments also help determine whether additional therapy is needed for persistent symptoms or obstruction. Mavacamten demonstrated significant efficacy in the EXPLORER-HCM trial, improving exercise capacity, reducing outflow tract gradients, and improving symptoms in patients with obstructive hypertrophic cardiomyopathy. In the VALOR-HCM trial, mavacamten reduced the need for septal reduction therapy in patients with severe disease, and across both studies it was generally well tolerated with careful monitoring of left ventricular ejection fraction.

Throughout the conversation, the experts provide a comprehensive reflection on the field and the factors that may shape how clinicians approach care moving forward.

In the next episode, Advancing Cardiac Myosin Inhibitor Therapy: Evidence, Monitoring, and Clinical Integration, panelists will continue their discussion on key clinical trial data for aficamten, including findings from MAPLE-HCM and SEQUOIA-HCM, and their implications for treatment of symptomatic obstructive hypertrophic cardiomyopathy. It also explores best practices for monitoring efficacy and safety, including navigating REMS programs and applying trial endpoints to real-world clinical decision-making.