de Gregorio Cesare, Bellocchi Paolo, Napoli Anna Rosa, Musumeci Beatrice, Sammartino Aniello, Tini Giacomo, Todiere Giancarlo, Barison Andrea, Chubuchnyi Vladyslav, Crotti Lia, Rella Valeria, Muraru Denisa, La Maestra Diego, Campisi Mariapaola, Biagini Elena, Schiavo Maria Alessandra, Bergami Claudio, Negri Francesco, Limongelli Giuseppe, Monda Emanuele, Verrillo Federica, Vagnarelli Fabio, Lofiego Carla, Tofoni Paolo, Tomasoni Daniela, Bellicini Maria Giulia, Perugini Enrica, Dattolo Giacomo, Sguazzotti Maurizio, Mabritto Barbara, Musumeci Giuseppe, Fumero Francesca, Monte Ines Paola, Faro Denise Cristiana, Raineri Claudia, Melis Daniele, Calore Chiara, Martini Marika, Re Federica, Dei Lorenzo-Lupo, Merlo Marco, Reginato Anna, Angriman Federico, Forleo Cinzia, Guaricci Andrea Igoren, Mapelli Massimo, Patti Giuseppe, Agostoni Piergiuseppe, Autore Camillo, Metra Marco, Canepa Marco, Castelletti Silvia, Aimo Alberto, Cappelli Francesco, Olivotto Iacopo, Sinagra Gianfranco, Imazio Massimo
Department of Clinical and Experimental Medicine. Outpatient heart failure and cardiomyopathy office. University Hospital of Messina.
Department of Clinical and Molecular Medicine, Sapienza University of Roma.
J Cardiovasc Med (Hagerstown). 2025 Jul 1;26(7):381-385. doi: 10.2459/JCM.0000000000001746. Epub 2025 Jun 20.
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.
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.
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.
大约三分之二的肥厚型心肌病患者表现为梗阻性（HOCM）生理特征。多年来，药物治疗一直局限于β受体阻滞剂、维拉帕米和/或丙吡胺。最近，一类新型药物，即心脏特异性肌球蛋白头部三磷酸腺苷酶（ATPase）的变构抑制剂，已被证明可有效缓解动态梗阻及相关临床症状。2024年7月，意大利心脏病学会心肌病和心包疾病工作组启动了一项全国多中心注册研究，旨在调查现实世界中HOCM患者动态梗阻的病理生理学。基于病历，本简要报告涉及根据探索者-HCM入组标准符合玛伐卡坦治疗条件，然后在2024年底前被批准用于同情用药的患者比例。
肥厚性梗阻性生理研究（HOPS）设计为一项针对意大利19个三级心脏中心截至2024年6月收治的连续成年患者的注册研究。共纳入424例患者，其中男性占53%，年龄为64±13岁。我们根据探索者-HCM的5项要求回顾性识别出200例符合玛伐卡坦治疗条件的患者（47.2%）。后一组中的40例患者，以及另外15例符合4项标准的患者，被纳入同情用药计划（n = 55，占总人群的13%）。43例表现为主动脉下梗阻，12例为心室中部变异型。伦理委员会批准项目因中心和地区而异。
本研究证实了我们最近的一项发现，即根据探索者-HCM试验的完整入组标准，大约一半的现实世界HOCM患者适合接受玛伐卡坦治疗。由于意大利目前同情用药计划的限制，只有四分之一的患者被批准接受治疗。
