One-Year Real-World Experience with Mavacamten and its Physiologic Effects on Obstructive Hypertrophic Cardiomyopathy

Daniel Kim; Emily Chu; Emily Keamy-Minor; Ishan Paranjpe; Wilson Tang; Jack O'Sullivan; Yaanik Desai; Michael Liu; Elise Munsey; Kimberly Hecker; Isabella Cuenco; Beth Kao; Ellen Bacolor; Colleen Bonnett; Andrea Linder; Kathleen Lacar; Nancy Robles; Cynthia Lamendola; Allysonne Smith; Joshua Knowles; Marco Perez; Masataka Kawana; Karim Sallam; Chad Weldy; Matthew Wheeler; Victoria Parikh; Heidi Salisbury; Euan Ashley

doi:10.20944/preprints202311.1692.v1

How to cite: Kim, D.; Chu, E.; Keamy-Minor, E.; Paranjpe, I.; Tang, W.; O'Sullivan, J.; Desai, Y.; Liu, M.; Munsey, E.; Hecker, K.; Cuenco, I.; Kao, B.; Bacolor, E.; Bonnett, C.; Linder, A.; Lacar, K.; Robles, N.; Lamendola, C.; Smith, A.; Knowles, J.; Perez, M.; Kawana, M.; Sallam, K.; Weldy, C.; Wheeler, M.; Parikh, V.; Salisbury, H.; Ashley, E. One-Year Real-World Experience with Mavacamten and its Physiologic Effects on Obstructive Hypertrophic Cardiomyopathy. Preprints 2023, 2023111692. https://doi.org/10.20944/preprints202311.1692.v1 Kim, D.; Chu, E.; Keamy-Minor, E.; Paranjpe, I.; Tang, W.; O'Sullivan, J.; Desai, Y.; Liu, M.; Munsey, E.; Hecker, K.; Cuenco, I.; Kao, B.; Bacolor, E.; Bonnett, C.; Linder, A.; Lacar, K.; Robles, N.; Lamendola, C.; Smith, A.; Knowles, J.; Perez, M.; Kawana, M.; Sallam, K.; Weldy, C.; Wheeler, M.; Parikh, V.; Salisbury, H.; Ashley, E. One-Year Real-World Experience with Mavacamten and its Physiologic Effects on Obstructive Hypertrophic Cardiomyopathy. Preprints 2023, 2023111692. https://doi.org/10.20944/preprints202311.1692.v1

Abstract

We describe real-world use of mavacamten in 50 patients with oHCM. Consistent with EXPLORER-HCM and VALOR-HCM, we report significant improvement in wall thickness, mitral regurgitation, left ventricular outflow tract obstruction and NYHA 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. 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.

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Copyright: This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.

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One-Year Real-World Experience with Mavacamten and its Physiologic Effects on Obstructive Hypertrophic Cardiomyopathy

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