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Background <p>Mavacamten is the first representative of the so-called myosin inhibitors that has been approved for clinical use in hypertrophic obstructive cardiomyopathy (HOCM).</p> Aim <p>Scientific background, current study situation, and first clinical experiences are described based on a&#xa0;case series of real-world treatment with mavacamten over a period of 12&#xa0;months.</p> Material and methods <p>The article examines the baseline characteristics as well as clinical and echocardiographic courses of 11 patients who were treated with mavacamten within the compassionate use program.</p> Results and discussion <p>Based on results of the two multicenter studies EXPLORER-HCM and VALOR-HCM, mavacamten was made available in Germany in December 2022 for the treatment of patients with HOCM initially through a&#xa0;compassionate use program. In the meantime, the drug has been approved for regular clinical use. We present the experiences with 11&#xa0;HOCM patients who were treated with mavacamten due to clinical indications at the German Heart Center of the Charité (Deutsches Herzzentrum der Charité). Under these real-world conditions over 12&#xa0;months, the positive results of the phase 3 approval trials were confirmed.</p>

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Myosininhibition mit Mavacamten zur Therapie der Hypertrophen Kardiomyopathie

  • 期刊论文

  • 发表日期：

    2025年12月19日

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  • Daniel Messroghli,
  • Anne Theresa Bingel,
  • Stefanie Maria Werhahn,
  • Viera Podracka,
  • Henryk Dreger,
  • Fabian Knebel

摘要

Background

Mavacamten is the first representative of the so-called myosin inhibitors that has been approved for clinical use in hypertrophic obstructive cardiomyopathy (HOCM).

Aim

Scientific background, current study situation, and first clinical experiences are described based on a case series of real-world treatment with mavacamten over a period of 12 months.

Material and methods

The article examines the baseline characteristics as well as clinical and echocardiographic courses of 11 patients who were treated with mavacamten within the compassionate use program.

Results and discussion

Based on results of the two multicenter studies EXPLORER-HCM and VALOR-HCM, mavacamten was made available in Germany in December 2022 for the treatment of patients with HOCM initially through a compassionate use program. In the meantime, the drug has been approved for regular clinical use. We present the experiences with 11 HOCM patients who were treated with mavacamten due to clinical indications at the German Heart Center of the Charité (Deutsches Herzzentrum der Charité). Under these real-world conditions over 12 months, the positive results of the phase 3 approval trials were confirmed.