What is the clinical evidence from this study?

Study design: Meta-Analysis. Population: Hypertrophic Cardiomyopathy (n=938). Intervention: Cardiac myosin inhibitors (mavacamten, aficamten) vs. Placebo or baseline. Primary outcome: Interventricular septum thickness (MD -1.77, 95% CI -3.30 to -0.23, p=0.0240).

What does this research mean for the field?

Cardiac myosin inhibitors significantly reduce interventricular septum thickness by 1.77 mm and left ventricular mass index by 18.15 g/m2 in patients with hypertrophic cardiomyopathy. Novelty: ClaimNovelty.SYNTHESIS. Consensus alignment: ConsensusAlignment.SUPPORTS_CONSENSUS.

February 2, 2026Open Access

Effect of Cardiac Myosin Inhibitors on Echocardiographic Features of Cardiac Structure and Function in Hypertrophic Cardiomyopathy: A Systematic Review and Meta-Analysis

YLYang LuChinese Academy of Medical Sciences & Peking Union Medical College YZYuanyuan ZhuNanjing Maternity and Child Health Care Hospital ZTZhuang TianChinese Academy of Medical Sciences & Peking Union Medical College

Key Result

Cardiac myosin inhibitors significantly reduced interventricular septum thickness (MD -1.77) and left ventricular mass index (MD -18.15) in patients with hypertrophic cardiomyopathy.

Key Points

The aim was to evaluate the effects of cardiac myosin inhibitors on echocardiographic features in patients with hypertrophic cardiomyopathy.
Conducted a systematic review and meta-analysis of studies up to September 2025.
Included studies that assessed the effects of cardiac myosin inhibitors on echocardiographic features in HCM patients.
Analyzed data from randomized controlled trials and long-term cohort studies.
CMIs significantly reduced interventricular septum thickness by 1.77 mm.
Left ventricular mass index decreased by 18.15 g/m² with CMIs.
Left ventricular ejection fraction showed a reduction of 3.22% with CMIs.
Echocardiographic left atrial volume index improved by 5.75 mL/m².
No association was found between CMIs and atrial arrhythmias.

Study Design

Type

Meta-Analysis (n=938)

Structured PICO

Do cardiac myosin inhibitors improve echocardiographic features of cardiac structure and function in adults with hypertrophic cardiomyopathy?

Population

938 adults aged 18 years and older with a confirmed diagnosis of hypertrophic cardiomyopathy (HCM), including both obstructive (oHCM) and nonobstructive (nHCM) types, pooled from 10 studies (5 RCTs, 3 echocardiographic sub-studies, and 2 long-term cohort studies).

Intervention

Cardiac myosin inhibitors (CMIs) as a class, specifically mavacamten and aficamten, administered for durations ranging from 16 to 128 weeks.

Comparator

Placebo (in the randomized controlled trials) or baseline measurements (in the single-arm cohort studies).

Outcome

Echocardiographic measures of cardiac structure and function, including interventricular septum thickness (IVST), left ventricular mass index (LVMI), left ventricular ejection fraction (LVEF), left atrial volume index (LAVI), and septal E/e' ratio.surrogate

Cardiac myosin inhibitors significantly improve left ventricular structure and diastolic function in patients with hypertrophic cardiomyopathy, but they also reduce left ventricular ejection fraction, necessitating careful monitoring.

Main Result

Effect estimate: MD -1.77 (95% CI -3.30 to -0.23)

p-value: p=0.0240

Limitations

Small number of eligible RCTs precluded a methodologically rigorous evaluation of publication bias
Included studies presented a short mean follow-up period
Only included RCTs involving obstructive HCM, so conclusions cannot be applied to nonobstructive HCM
Unable to assess the impact of CMIs on mitral regurgitation, cardiac magnetic resonance, or cardiac biomarkers

Abstract

Background: Recent advancements have introduced novel cardiac myosin inhibitors (CMIs) that have demonstrated significant efficacy in treating hypertrophic cardiomyopathy (HCM). This meta-analysis aimed to clarify the current understanding of the impact of CMIs on echocardiographic cardiac structure and function in patients with HCM. Methods: A comprehensive search of the PubMed, Cochrane Library, and Embase databases was conducted from inception until September 14, 2025. The studies reporting the impact of CMIs on echocardiographic cardiac structure and function in HCM patients were included. Results: Ultimately, this meta-analysis included 10 studies: five randomized controlled trials (RCTs), three echocardiographic sub-studies derived from RCTs, and two long-term cohort studies. A total of 938 patients were enrolled in these studies. This meta-analysis revealed that CMIs significantly reduce interventricular septum thickness (mean difference (MD): –1.77, 95% confidence interval (CI): –3.30 to –0.23; p = 0.0240). CMIs were also shown to significantly reduce left ventricular mass index (MD: –18.15, 95% CI: –32.65 to –3.65; p = 0.0141). Moreover, the pooled results demonstrated that administering CMIs can significantly reduce left ventricular ejection fraction (MD: –3.22, 95% CI: –5.60 to –0.85; p = 0.0078). CMIs also significantly improved echocardiographic parameters of left ventricular diastolic function, such as the left atrial volume index (MD: –5.75, 95% CI: –7.87 to –3.64; p < 0.0001) and septal E/e′ ratio (MD: –3.80, 95% CI: –4.74 to –2.87; p < 0.0001). However, the results did not reveal an association between CMIs and the risk of atrial arrhythmias (risk ratio (RR): 0.98, 95% CI: 0.33 to 2.94; p = 0.9689). Conclusions: CMIs have shown great efficacy in improving left ventricular structure and diastolic function in HCM patients. Additionally, CMIs can reduce left ventricular ejection fraction. However, the impact of CMIs on the risk of atrial arrhythmias remains unclear. The PROSPERO Registration: CRD420251243904, https://www.crd.york.ac.uk/PROSPERO/view/CRD420251243904.

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