Cardiac Myosin Inhibitors for Obstructive Hypertrophic Cardiomyopathy: A Meta-analysis of Randomized...

Area of Science:

• Cardiology
• Pharmacology

Background:

• Cardiac myosin inhibitors (CMI) are a novel, non-invasive therapy for hypertrophic cardiomyopathy.
• The efficacy and safety of CMI in obstructive hypertrophic cardiomyopathy (oHCM) require further investigation, particularly regarding secondary endpoints in randomized controlled trials (RCTs).

Purpose of the Study:

• To systematically evaluate the efficacy and safety of CMI in patients with oHCM by analyzing data from RCTs.
• To assess the impact of CMI on clinical and hemodynamic parameters, as well as potential adverse events.

Main Methods:

• Systematic literature search of PubMed, Embase, Web of Science, and Clinicaltrials.gov for RCTs comparing CMI with placebo in oHCM patients.
• Random-effects model applied to evaluate efficacy and safety outcomes, including primary and secondary endpoints.

Main Results:

• Five RCTs with 767 patients were included; 402 received CMI.
• CMI significantly improved New York Heart Association (NYHA) functional class (RR 2.33) and reduced resting and provoked left ventricular outflow tract (LVOT) gradients.
• CMI increased the risk of left ventricular ejection fraction <50% (RR 4.80), affecting 8% of patients, but a class effect was observed, with benefits outweighing risks.

Conclusions:

• Cardiac myosin inhibitors (mavacamten, aficamten) represent a class effect that improves clinical and hemodynamic endpoints in oHCM patients.
• Despite an increased incidence of reduced left ventricular ejection fraction, the overall benefit-risk profile favors CMI use in oHCM.