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Observational Study
. 2025 Jul 1;26(7):381-385.
doi: 10.2459/JCM.0000000000001746. Epub 2025 Jun 20.

Myosin-ATPase inhibitor in real-world patients with obstructive HCM: a report by the Cardiomyopathies and Pericardial Diseases WG of the Italian Society of Cardiology

Cesare de Gregorio  1 , Paolo Bellocchi  1 , Anna Rosa Napoli  1 , Beatrice Musumeci  2 , Aniello Sammartino  2 , Giacomo Tini  2 , Giancarlo Todiere  3 , Andrea Barison  3 , Vladyslav Chubuchnyi  3 , Lia Crotti  4   5 , Valeria Rella  4 , Denisa Muraru  4   5 , Diego La Maestra  1 , Mariapaola Campisi  1 , Elena Biagini  6   7 , Maria Alessandra Schiavo  6   7 , Claudio Bergami  6   7 , Francesco Negri  8 , Giuseppe Limongelli  7   9 , Emanuele Monda  7   9 , Federica Verrillo  7   9 , Fabio Vagnarelli  10 , Carla Lofiego  10 , Paolo Tofoni  10 , Daniela Tomasoni  11 , Maria Giulia Bellicini  11 , Enrica Perugini  12 , Giacomo Dattolo  12 , Maurizio Sguazzotti  12 , Barbara Mabritto  13 , Giuseppe Musumeci  13 , Francesca Fumero  13 , Ines Paola Monte  14 , Denise Cristiana Faro  14 , Claudia Raineri  15 , Daniele Melis  15 , Chiara Calore  16 , Marika Martini  16 , Federica Re  17 , Lorenzo-Lupo Dei  17 , Marco Merlo  7   18 , Anna Reginato  7   18 , Federico Angriman  7   18 , Cinzia Forleo  19 , Andrea Igoren Guaricci  19 , Massimo Mapelli  20   21 , Giuseppe Patti  22 , Piergiuseppe Agostoni  20   21 , Camillo Autore  23 , Marco Metra  11 , Marco Canepa  24 , Silvia Castelletti  4 , Alberto Aimo  3 , Francesco Cappelli  25 , Iacopo Olivotto  25 , Gianfranco Sinagra  7   18 , Massimo Imazio  8   26
Affiliations
Observational Study

Myosin-ATPase inhibitor in real-world patients with obstructive HCM: a report by the Cardiomyopathies and Pericardial Diseases WG of the Italian Society of Cardiology

Cesare de Gregorio et al. J Cardiovasc Med (Hagerstown). .

Abstract

Introduction: Approximately two-thirds of patients suffering from hypertrophic cardiomyopathy present with an obstructive (HOCM) physiology. For years, medical therapy has been limited to beta blockers, verapamil and/or disopyramide. Recently, a novel class of drugs, the allosteric inhibitors of the cardiac-specific myosin head adenosine triphosphatase (ATPase), have been demonstrated to be effective in relieving the dynamic obstruction and related clinical condition. In July 2024 the Cardiomyopathies and Pericardial Diseases WG of the Italian Society of Cardiology started with a nationwide multicentre registry aimed at investigating the pathophysiology of dynamic obstruction in real-world patients with HOCM. Based on the medical records, this brief report deals with the proportion of patients who were eligible for Mavacamten based on the Explorer-HCM entry criteria, and then admitted for compassionate use by the end of 2024.

Methods and results: The Hypertrophic Obstructive Physiology Study (HOPS) was designed as a registry on consecutive adult patients admitted to 19 tertiary Cardiac Centres in Italy until June 2024. A total of 424 patients, 53% males, aged 64 ± 13 years, were included. We retrospectively recognized 200 Mavacamten-eligible patients (47.2%) on 5 Explorer-HCM requirements. Forty out of this latter group, along with 15 more patients on 4 criteria, were admitted to the compassionate use programme ( n = 55, 13% of the whole population). Forty-three showed subaortic obstruction and 12 a mid-ventricular variant. Ethical committee approval items varied among centres and regions.

Discussion: This study confirmed our recent demonstration that approximately half of real-world HOCM patients are suitable for Mavacamten therapy based on the full Explorer-HCM trial entry criteria. Due to the current limitation of compassionate use programmes in Italy, only one in four patients was admitted for treatment.

Keywords: heart failure; hypertrophic cardiomyopathy; myosin ATP-ase inhibitors; obstructive physiology; treatment.

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