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. 2025 Jan 15:11:1477487.
doi: 10.3389/fcvm.2024.1477487. eCollection 2024.

Efficacy and safety of cardiac myosin inhibitors for symptomatic hypertrophic cardiomyopathy: a meta-analysis of randomized controlled trials

Anas Abunada  1 , Madiha Shah  1 , Ateesh Kumar  2 , Syeda Lamiya Mir  2 , Dinesh Kumar  1 , Saboor Ahmed  2 , Muhammad Tanzeel  3 , Vikash Kumar  4 , Aashish Meghjiani  2 , Muhammad Basit Ali Siddiqui  2 , Govinda Khatri  2 , Aneesh Rai  2 , Fnu Deepak  3 , Ayush Kumar  5
Affiliations

Efficacy and safety of cardiac myosin inhibitors for symptomatic hypertrophic cardiomyopathy: a meta-analysis of randomized controlled trials

Anas Abunada et al. Front Cardiovasc Med. .

Abstract

Introduction: Hypertrophic cardiomyopathy (HCM) is a common genetic heart disorder. It is characterized by left ventricular hypertrophy and impaired cardiac function, with forms categorized into obstructive (oHCM) and nonobstructive (nHCM). Traditional treatments address symptoms but not the underlying disease mechanism, highlighting the need for novel therapies. Cardiac myosin inhibitors such as mavacamten and aficamten present potential new treatment options.

Methods: A meta-analysis of randomized controlled trials (RCTs) was conducted following PRISMA guidelines. Studies comparing cardiac myosin inhibitors with placebo were reviewed, and outcomes related to NYHA functional class, Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS), LVOT gradients, and left ventricular ejection fraction (LVEF) were analyzed.

Results: Six RCTs involving 826 participants demonstrated that mavacamten and aficamten significantly improved NYHA functional class and KCCQ-CSS scores. The incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) was similar between the treatment and control groups, indicating a comparable safety profile.

Conclusion: Cardiac myosin inhibitors are effective in improving cardiac function and reducing LVOT obstruction in HCM patients. They offer a promising alternative to current treatments, with a safety profile comparable to placebo. Further research is needed to confirm long-term benefits.

Keywords: LVOT obstruction; aficamten; cardiac myosin inhibitors; hypertrophic cardiomyopathy; mavacamten.

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Conflict of interest statement

The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.

Figures

Figure 1
Figure 1
Prisma flow diagram.
Figure 2
Figure 2
(A) Risk of bias summary. (B) Risk of bias graph.
Figure 3
Figure 3
Forest plot for improvement in NYHA functional class.
Figure 4
Figure 4
Forest plot for improvement in KCCQ-CSS.
Figure 5
Figure 5
Forest plot for mean Valsalva LVOT peak gradient.
Figure 6
Figure 6
Forest plot for mean rest LVOT peak gradient.
Figure 7
Figure 7
Forest plot for change in LVEF.
Figure 8
Figure 8
Forest plot for post-exercise LVOT gradient.
Figure 9
Figure 9
Forest plot for TEAEs.
Figure 10
Figure 10
Forest plot for SAEs.
See this image and copyright information in PMC

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