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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
Daniel Kim Daniel Kim *Emily Chu Emily Chu *Emily Keamy-Minor Emily Keamy-Minor *Ishan Paranjpe Ishan Paranjpe *Wilson L. Tang Wilson L. Tang Jack W. O'sullivan Jack W. O'sullivan *Yaanik B. Desai Yaanik B. Desai *Michael B. Liu Michael B. Liu *Elise Munsey Elise Munsey *Kimberly Hecker Kimberly Hecker *Isabella Cuenco Isabella Cuenco *Beth Kao Beth Kao *Ellen Bacolor Ellen Bacolor *Colleen Bonnett Colleen Bonnett *Andrea Linder Andrea Linder Kathleen Lacar Kathleen Lacar *Nancy Robles Nancy Robles *Cindy Lamendola Cindy Lamendola *Allysonne Smith Allysonne Smith *Josh Knowles Josh Knowles Marco V. Perez Marco V. Perez *Masataka Kawana Masataka Kawana Karim Sallam Karim Sallam Chad S. Weldy Chad S. Weldy Matthew T. Wheeler Matthew T. Wheeler Victoria N. Parikh Victoria N. Parikh *Heidi Salisbury Heidi Salisbury *Euan A. Ashley Euan A. Ashley *
  • Stanford University, Stanford, United States

The final, formatted version of the article will be published soon.

    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

    Disclaimer: All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.