Frontiers in Cardiovascular Medicine (Aug 2024)

One-year real-world experience with mavacamten and its physiologic effects on obstructive hypertrophic cardiomyopathy

  • Daniel Seung Kim,
  • Daniel Seung Kim,
  • Daniel Seung Kim,
  • Daniel Seung Kim,
  • Daniel Seung Kim,
  • Emily L. Chu,
  • Emily E. Keamy-Minor,
  • Ishan Dhananjay Paranjpe,
  • Wilson L. Tang,
  • Jack W. O’Sullivan,
  • Jack W. O’Sullivan,
  • Jack W. O’Sullivan,
  • Yaanik B. Desai,
  • Yaanik B. Desai,
  • Michael B. Liu,
  • Michael B. Liu,
  • Elise Munsey,
  • Kimberly Hecker,
  • Isabella Cuenco,
  • Beth Kao,
  • Ellen Bacolor,
  • Colleen Bonnett,
  • Andrea Linder,
  • Kathleen Lacar,
  • Nancy Robles,
  • Cindy Lamendola,
  • Allysonne Smith,
  • Joshua W. Knowles,
  • Joshua W. Knowles,
  • Joshua W. Knowles,
  • Joshua W. Knowles,
  • Marco V. Perez,
  • Marco V. Perez,
  • Marco V. Perez,
  • Marco V. Perez,
  • Masataka Kawana,
  • Masataka Kawana,
  • Masataka Kawana,
  • Karim I. Sallam,
  • Karim I. Sallam,
  • Karim I. Sallam,
  • Chad S. Weldy,
  • Chad S. Weldy,
  • Chad S. Weldy,
  • Matthew T. Wheeler,
  • Matthew T. Wheeler,
  • Matthew T. Wheeler,
  • Matthew T. Wheeler,
  • Victoria N. Parikh,
  • Victoria N. Parikh,
  • Victoria N. Parikh,
  • Victoria N. Parikh,
  • Heidi Salisbury,
  • Euan A. Ashley,
  • Euan A. Ashley,
  • Euan A. Ashley,
  • Euan A. Ashley,
  • Euan A. Ashley,
  • Euan A. Ashley,
  • Euan A. Ashley,
  • the Stanford Center for Inherited Cardiovascular Disease,
  • Karim I Sallam,
  • Masataka Kawana,
  • Chad S Weldy,
  • Marco Perez,
  • Joshua W Knowles,
  • Jason Tso,
  • Cindy Lamendola,
  • Allysonne Smith,
  • Nancy Robles,
  • Colleen Bonnett,
  • Ellen Bacolor,
  • Kimberly Hecker,
  • Isabella Cuenco,
  • Beth Kao,
  • Elise Munsey,
  • Andrea Linder,
  • Kathleen Lacar,
  • Julia Platt,
  • Chloe Reuter,
  • Tia Moscarello,
  • Ryan Murtha,
  • Jennifer Kohler,
  • Hannah Ison,
  • Mitchel Pariani,
  • Anusha Klinder,
  • Priya Nair,
  • Jennifer Marino,
  • Andrea Linder,
  • Ruchi Patel,
  • Matthew T Wheeler,
  • Euan A Ashley,
  • Victoria N Parikh

DOI
https://doi.org/10.3389/fcvm.2024.1429230
Journal volume & issue
Vol. 11

Abstract

Read online

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