Efficacy and safety of mavacamten in non-obstructive hypertrophic cardiomyopathy: A systematic review and meta-analysis of randomized trials

Background: Non-obstructive hypertrophic cardiomyopathy (nHCM) remains a therapeutic challenge, with limited pharmacological options beyond symptomatic management. Mavacamten, a selective allosteric cardiac myosin inhibitor, has shown benefit in obstructive HCM, but its role in nHCM is uncertain. Methods: ), New York Heart Association (NYHA) functional class, and treatment-emergent adverse events (TEAEs). Pooled mean differences (MDs) and risk ratios (RRs) were estimated using random- or fixed-effects models based on heterogeneity. Results: = 40.8%). Atrial fibrillation occurred more frequently with mavacamten (RR: 1.41; 95% CI: 1.28 to 1.56), whereas palpitations and dizziness did not differ significantly. Conclusions: Mavacamten does not significantly enhance symptoms, functional capacity, or health status in nHCM, though safety is comparable to placebo. Ongoing monitoring for arrhythmias and reduced ejection fraction is essential. Larger, longer-term studies are needed to clarify its role in this population.

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Journal
IJC Heart & Vasculature
Published
2026-04-16
DOI
https://doi.org/10.1016/j.ijcha.2026.101908
Primary Topic
Cardiomyopathy and Myosin Studies
Type
article
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article

Efficacy and safety of mavacamten in non-obstructive hypertrophic cardiomyopathy: A systematic review and meta-analysis of randomized trials

Mazen Albaghdadi, Noman Khalid, Salman Muddassir, Michael Ayers et al.
IJC Heart & Vasculature
Cardiomyopathy and Myosin Studies
article

Efficacy and safety of mavacamten in non-obstructive hypertrophic cardiomyopathy: A systematic review and meta-analysis of randomized trials

Mazen Albaghdadi, Noman Khalid, Salman Muddassir, Michael Ayers, Mustafa Bin Tahir, Husna Irfan Thalib, Muhammad Adil Afzal, Nagaraja D. Sharma, Michael D. Moore, Ahmad Mushtaq, Wilbert S. Aronow
article en

Abstract

Background: Non-obstructive hypertrophic cardiomyopathy (nHCM) remains a therapeutic challenge, with limited pharmacological options beyond symptomatic management. Mavacamten, a selective allosteric cardiac myosin inhibitor, has shown benefit in obstructive HCM, but its role in nHCM is uncertain. Methods: ), New York Heart Association (NYHA) functional class, and treatment-emergent adverse events (TEAEs). Pooled mean differences (MDs) and risk ratios (RRs) were estimated using random- or fixed-effects models based on heterogeneity. Results: = 40.8%). Atrial fibrillation occurred more frequently with mavacamten (RR: 1.41; 95% CI: 1.28 to 1.56), whereas palpitations and dizziness did not differ significantly. Conclusions: Mavacamten does not significantly enhance symptoms, functional capacity, or health status in nHCM, though safety is comparable to placebo. Ongoing monitoring for arrhythmias and reduced ejection fraction is essential. Larger, longer-term studies are needed to clarify its role in this population.

IJC Heart & VasculatureVol. 64
University of Central Florida (US), Westchester Medical Center (US), NewYork–Presbyterian Brooklyn Methodist Hospital (US), New York Medical College (US), HCA Healthcare (US), King Abdulaziz Medical City (SA), St. Joseph’s University Medical Center (US), Naples Anesthesia & Physician Associates (US), Shaikh Zayed Postgraduate Medical Institute (PK), Batterjee Medical College, University of Virginia (US)
Good health and well-being
Openalex Percentile: Top 24%
Cardiomyopathy and Myosin Studies
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