Baseline clinical profile of patients with obstructive hypertrophic cardiomyopathy in the Italian Mavacamten early access program

IF 3.3 2区医学 Q2 CARDIAC & CARDIOVASCULAR SYSTEMS

International journal of cardiology Pub Date : 2026-04-01 Epub Date: 2026-01-08 DOI:10.1016/j.ijcard.2025.134152

G. Bonacchi , A. Del Franco , P. Agostoni , E. Agricola , E. Ammirati , P. Baratta , F. Barocelli , E. Bertero , E. Biagini , G. Boriani , P. Calabrò , L. Calò , C. Calore , S. Carigi , E. Carluccio , N. Cerrato , C. Chimenti , A. Chinaglia , C. Chiriatti , L. Crotti , I. Olivotto

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引用次数: 0

Abstract

Background

Obstructive hypertrophic cardiomyopathy (oHCM) has recently seen the introduction of mavacamten, a disease-specific therapy that alleviates obstruction and symptoms with potential effects on disease expression. Since most patients remain symptomatic with conventional therapies, mavacamten constitutes an efficacious treatment strategy

Methods

The early access program (EAP) for mavacamten treatment ran from September 2023 to September 2024. We collected clinical and echocardiographic data prospectively

Results

337 patients with oHCM (53% male, mean age 61.4 ± 12.3 years) were receiving mavacamten treatment from 54 centers in Italy. The EAP cohort was older and showed a more advanced cardiac phenotype, showing lower left ventricular ejection fraction (LVEF) (66 ± 6% vs 74 ± 6% in EXPLORER-HCM cohort, p < 0.0001) and higher left atrial volume index (51.1 ± 16.4 ml/m² vs 40.5 ± 12.3 ml/m², p < 0.0001. Only 74 (22%) patients in the EAP group were candidates for septal reduction therapy (SRT). Echocardiographic parameters like LVEF, maximal wall thickness and LAVI as well as pharmacologic therapy showed no difference between subgroups, while LVOT obstruction gradient was higher in SRT candidates (40 vs 63 mmHg, p < 0.0001)..

Conclusion

The compassionate use program for mavacamten in Italy demonstrated significant engagement. The Italian oHCM cohort were older and exhibited more advanced disease stages compared to those in the EXPLORER-HCM trial. Only one-quarter of the patients were eligible for SRT, they did not show a more severe biomarker or echocardiographic parameters compared to those without indication. Mavacamten provides an additional option for oHCM patients unresponsive to standard therapy, especially those symptomatic but ineligible for SRT

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意大利Mavacamten早期准入项目中梗阻性肥厚性心肌病患者的基线临床概况

背景：梗阻性肥厚性心肌病（oHCM）最近引入了马伐卡坦，这是一种疾病特异性治疗，可缓解梗阻和症状，并对疾病表达有潜在影响。由于大多数患者在常规治疗下仍有症状，因此马伐卡坦是一种有效的治疗策略。方法：马伐卡坦治疗的早期准入项目（EAP）从2023年9月持续到2024年9月。结果：来自意大利54个中心的337例oHCM患者（53%为男性，平均年龄61.4 ± 12.3 岁）接受了马伐卡坦治疗。EAP群年长的,显示出更高级的心脏表型,显示降低左心室射血分数(LVEF)(66年 ± 6% vs 74 ±EXPLORER-HCM队列 6%,p 2和40.5 ±12.3  ml / m2, p 结论:同情使用计划在意大利mavacamten证明重要的参与。与EXPLORER-HCM试验相比，意大利oHCM队列年龄较大，表现出更晚期的疾病阶段。只有四分之一的患者符合SRT的条件，与没有适应症的患者相比，他们没有表现出更严重的生物标志物或超声心动图参数。Mavacamten为对标准治疗无反应的oHCM患者提供了额外的选择，特别是那些有症状但不适合SRT的患者。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

International journal of cardiology 医学-心血管系统

CiteScore

6.80

自引率

5.70%

发文量

758

审稿时长

44 days

期刊介绍： The International Journal of Cardiology is devoted to cardiology in the broadest sense. Both basic research and clinical papers can be submitted. The journal serves the interest of both practicing clinicians and researchers. In addition to original papers, we are launching a range of new manuscript types, including Consensus and Position Papers, Systematic Reviews, Meta-analyses, and Short communications. Case reports are no longer acceptable. Controversial techniques, issues on health policy and social medicine are discussed and serve as useful tools for encouraging debate.