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BRIEF RESEARCH REPORT article
Front. Cardiovasc. Med.
Sec. Cardiovascular Pharmacology and Drug Discovery
Volume 11 - 2024 |
doi: 10.3389/fcvm.2024.1429230
This article is part of the Research Topic Novel Pharmacologic Therapeutics in Cardiovascular Diseases View all 4 articles
One-year real-world experience with mavacamten and its physiologic effects on obstructive hypertrophic cardiomyopathy
Provisionally accepted- Stanford University, Stanford, United States
Mavacamten is a first-in-class cardiac myosin ATPase inhibitor, approved by the United States Food and Drug Administration for the treatment of hypertrophic cardiomyopathy with obstructive physiology (oHCM). Here, we present the real-world use of mavacamten in 50 patients with oHCM at a tertiary care referral center. In both our highlighted case and in our aggregate data, we report significant improvement in wall thickness, mitral regurgitation, left ventricular outflow tract obstruction and New York Heart Association symptom class. Moreover, in our center’s experience, neither arrhythmia burden, nor contractility have worsened in the vast majority of patients: we note a clinically insignificant mean decrease in left ventricular ejection fraction (LVEF), with only two patients requiring temporary mavacamten discontinuance for LVEF < 50%. Adverse events were rare, unrelated to mavacamten itself, and seen solely in patients with disease too advanced to have been represented in clinical trials. Moreover, our multidisciplinary pathway enabled us to provide a large number of patients with a novel closely-monitored therapeutic within just a few months of commercial availability. These data lead us to conclude that mavacamten, as a first-in-class cardiac myosin inhibitor, is safe and efficacious in real-world settings.
Keywords: mavacamten, MYK-461, hypertrophic cardiomyopathy, cardiac myosin inhibitor, hypertrophic cardiomyopathy with obstruction (oHCM), hypertrophic obstructive cardiomyopathy (HOCM), left ventricular outflow tract obstruction LV -left ventricle, LVEF -left ventricular ejection fraction, LVOTv -left ventricular outflow tract obstructive gradient with Valsalva maneuver, LVOTr -left ventricular outflow tract obstructive gradient at rest, MCS -mechanical circulatory support, MR -mitral regurgitation, NYHA -New York Heart Association, RVSP -right ventricular systolic pressure
Received: 07 May 2024; Accepted: 13 Aug 2024.
Copyright: © 2024 Kim, Chu, Keamy-Minor, Paranjpe, Tang, O'sullivan, Desai, Liu, Munsey, Hecker, Cuenco, Kao, Bacolor, Bonnett, Linder, Lacar, Robles, Lamendola, Smith, Knowles, Perez, Kawana, Sallam, Weldy, Wheeler, Parikh, Salisbury and Ashley. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence:
Daniel Kim, Stanford University, Stanford, United States
Emily Chu, Stanford University, Stanford, United States
Emily Keamy-Minor, Stanford University, Stanford, United States
Ishan Paranjpe, Stanford University, Stanford, United States
Jack W. O'sullivan, Stanford University, Stanford, United States
Yaanik B. Desai, Stanford University, Stanford, United States
Michael B. Liu, Stanford University, Stanford, United States
Elise Munsey, Stanford University, Stanford, United States
Kimberly Hecker, Stanford University, Stanford, United States
Isabella Cuenco, Stanford University, Stanford, United States
Beth Kao, Stanford University, Stanford, United States
Ellen Bacolor, Stanford University, Stanford, United States
Colleen Bonnett, Stanford University, Stanford, United States
Kathleen Lacar, Stanford University, Stanford, United States
Nancy Robles, Stanford University, Stanford, United States
Cindy Lamendola, Stanford University, Stanford, United States
Allysonne Smith, Stanford University, Stanford, United States
Marco V. Perez, Stanford University, Stanford, United States
Victoria N. Parikh, Stanford University, Stanford, United States
Heidi Salisbury, Stanford University, Stanford, United States
Euan A. Ashley, Stanford University, Stanford, United States
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