Cesare de Gregorio, Paolo Bellocchi, Anna Rosa Napoli, Beatrice Musumeci, Aniello Sammartino, Giacomo Tini, Giancarlo Todiere, Andrea Barison, Vladyslav Chubuchnyi, Lia Crotti, Valeria Rella, Denisa Muraru, Diego La Maestra, Mariapaola Campisi, Elena Biagini, Maria Alessandra Schiavo, Claudio Bergami, Francesco Negri, Giuseppe Limongelli, Emanuele Monda, Federica Verrillo, Fabio Vagnarelli, Carla Lofiego, Paolo Tofoni, Daniela Tomasoni, Maria Giulia Bellicini, Enrica Perugini, Giacomo Dattolo, Maurizio Sguazzotti, Barbara Mabritto, Giuseppe Musumeci, Francesca Fumero, Ines Paola Monte, Denise Cristiana Faro, Claudia Raineri, Daniele Melis, Chiara Calore, Marika Martini, Federica Re, Lorenzo-Lupo Dei, Marco Merlo, Anna Reginato, Federico Angriman, Cinzia Forleo, Andrea Igoren Guaricci, Massimo Mapelli, Giuseppe Patti, Piergiuseppe Agostoni, Camillo Autore, Marco Metra, Marco Canepa, Silvia Castelletti, Alberto Aimo, Francesco Cappelli, Iacopo Olivotto, Gianfranco Sinagra, Massimo Imazio
{"title":"肌球蛋白- atp酶抑制剂在现实世界中阻塞性HCM患者中的作用：意大利心脏病学会心肌病和心包疾病工作组的一份报告。","authors":"Cesare de Gregorio, Paolo Bellocchi, Anna Rosa Napoli, Beatrice Musumeci, Aniello Sammartino, Giacomo Tini, Giancarlo Todiere, Andrea Barison, Vladyslav Chubuchnyi, Lia Crotti, Valeria Rella, Denisa Muraru, Diego La Maestra, Mariapaola Campisi, Elena Biagini, Maria Alessandra Schiavo, Claudio Bergami, Francesco Negri, Giuseppe Limongelli, Emanuele Monda, Federica Verrillo, Fabio Vagnarelli, Carla Lofiego, Paolo Tofoni, Daniela Tomasoni, Maria Giulia Bellicini, Enrica Perugini, Giacomo Dattolo, Maurizio Sguazzotti, Barbara Mabritto, Giuseppe Musumeci, Francesca Fumero, Ines Paola Monte, Denise Cristiana Faro, Claudia Raineri, Daniele Melis, Chiara Calore, Marika Martini, Federica Re, Lorenzo-Lupo Dei, Marco Merlo, Anna Reginato, Federico Angriman, Cinzia Forleo, Andrea Igoren Guaricci, Massimo Mapelli, Giuseppe Patti, Piergiuseppe Agostoni, Camillo Autore, Marco Metra, Marco Canepa, Silvia Castelletti, Alberto Aimo, Francesco Cappelli, Iacopo Olivotto, Gianfranco Sinagra, Massimo Imazio","doi":"10.2459/JCM.0000000000001746","DOIUrl":null,"url":null,"abstract":"<p><strong>Introduction: </strong>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.</p><p><strong>Methods and results: </strong>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.</p><p><strong>Discussion: </strong>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.</p>","PeriodicalId":15228,"journal":{"name":"Journal of Cardiovascular Medicine","volume":" ","pages":"381-385"},"PeriodicalIF":2.0000,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"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.\",\"authors\":\"Cesare de Gregorio, Paolo Bellocchi, Anna Rosa Napoli, Beatrice Musumeci, Aniello Sammartino, Giacomo Tini, Giancarlo Todiere, Andrea Barison, Vladyslav Chubuchnyi, Lia Crotti, Valeria Rella, Denisa Muraru, Diego La Maestra, Mariapaola Campisi, Elena Biagini, Maria Alessandra Schiavo, Claudio Bergami, Francesco Negri, Giuseppe Limongelli, Emanuele Monda, Federica Verrillo, Fabio Vagnarelli, Carla Lofiego, Paolo Tofoni, Daniela Tomasoni, Maria Giulia Bellicini, Enrica Perugini, Giacomo Dattolo, Maurizio Sguazzotti, Barbara Mabritto, Giuseppe Musumeci, Francesca Fumero, Ines Paola Monte, Denise Cristiana Faro, Claudia Raineri, Daniele Melis, Chiara Calore, Marika Martini, Federica Re, Lorenzo-Lupo Dei, Marco Merlo, Anna Reginato, Federico Angriman, Cinzia Forleo, Andrea Igoren Guaricci, Massimo Mapelli, Giuseppe Patti, Piergiuseppe Agostoni, Camillo Autore, Marco Metra, Marco Canepa, Silvia Castelletti, Alberto Aimo, Francesco Cappelli, Iacopo Olivotto, Gianfranco Sinagra, Massimo Imazio\",\"doi\":\"10.2459/JCM.0000000000001746\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Introduction: </strong>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.</p><p><strong>Methods and results: </strong>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.</p><p><strong>Discussion: </strong>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.</p>\",\"PeriodicalId\":15228,\"journal\":{\"name\":\"Journal of Cardiovascular Medicine\",\"volume\":\" \",\"pages\":\"381-385\"},\"PeriodicalIF\":2.0000,\"publicationDate\":\"2025-07-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Journal of Cardiovascular Medicine\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.2459/JCM.0000000000001746\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2025/6/20 0:00:00\",\"PubModel\":\"Epub\",\"JCR\":\"Q2\",\"JCRName\":\"CARDIAC & CARDIOVASCULAR SYSTEMS\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of Cardiovascular Medicine","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.2459/JCM.0000000000001746","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2025/6/20 0:00:00","PubModel":"Epub","JCR":"Q2","JCRName":"CARDIAC & CARDIOVASCULAR SYSTEMS","Score":null,"Total":0}
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.
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.
期刊介绍：
Journal of Cardiovascular Medicine is a monthly publication of the Italian Federation of Cardiology. It publishes original research articles, epidemiological studies, new methodological clinical approaches, case reports, design and goals of clinical trials, review articles, points of view, editorials and Images in cardiovascular medicine.
Submitted articles undergo a preliminary review by the editor. Some articles may be returned to authors without further consideration. Those being considered for publication will undergo further assessment and peer-review by the editors and those invited to do so from a reviewer pool.
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