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Echocardiographic Evolution of Symptomatic Patients With Obstructive Hypertrophic Cardiomyopathy treated with Mavacamten: Experience of a Portuguese Terciary Care Center
Session:
SESSÃO DE COMUNICAÇÕES ORAIS 16 – AVANÇOS NO DIAGNÓSTICO E TRATAMENTO DAS MIOCARDIOPATIAS
Speaker:
José Miguel Viegas
Congress:
CPC 2025
Topic:
F. Valvular, Myocardial, Pericardial, Pulmonary, Congenital Heart Disease
Theme:
17. Myocardial Disease
Subtheme:
17.4 Myocardial Disease – Treatment
Session Type:
Comunicações Orais
FP Number:
---
Authors:
José Miguel Viegas; Pedro Garcia Brás; Inês Grácio Almeida; Isabel Cardoso; Miguel Marques Antunes; Rui Cruz Ferreira; Sílvia Aguiar Rosa
Abstract
<p><span style="font-size:11pt"><span style="font-family:Calibri,sans-serif"><strong><span style="font-size:9.0pt">Background: </span></strong><span style="font-size:9.0pt">Mavacamten, a first-in-class cardiac myosin inhibitor, received marketing authorization throughout the European Union in June 2023. The purpose of this study was to evaluate echocardiographic changes over time in patients (pts) with obstructive hypertrophic cardiomyopathy (oHCM) treated in a Portuguese tertiary hospital.</span></span></span></p> <p> </p> <p><span style="font-size:11pt"><span style="font-family:Calibri,sans-serif"><strong><span style="font-size:9.0pt">Methods: </span></strong><span style="font-size:9.0pt">A standardized registry was prospectively performed for all pts who started mavacamten at a single oHCM referral center. Echocardiographic measures of cardiac structure and function were assessed at baseline and subsequently in accordance with the product characteristics. Data were compared using paired t-test.</span></span></span></p> <p> </p> <p><span style="font-size:11pt"><span style="font-family:Calibri,sans-serif"><strong><span style="font-size:9.0pt">Results: </span></strong><span style="font-size:9.0pt">A total of 18 pts, 61% male, mean age </span><span style="font-size:9.0pt">57±16 years. 2 pts had latent exercise-provoked left ventricular outflow tract (LVOT) obstruction. No CYP2C19 poor metabolisers were identified. The mean follow-up period was </span><span style="font-size:9.0pt">18±10 </span><span style="font-size:9.0pt">weeks. At this point, 8 pts (44%) had completed the titration phase and reached the maintenance dose of the drug, of which 1pt on 5mg, 1pt on 15mg, and the remaining on 10mg per day.</span></span></span></p> <p><span style="font-size:11pt"><span style="font-family:Calibri,sans-serif"><span style="font-size:9.0pt">Echocardiographic parameters at baseline and during follow-up are presented in Figure 1. Pts showed rapid and sustained reduction in both resting and Valsalva LVOT gradients (20 and 24% reduction from baseline, respectively, at the 5 mg dose, and 60% reduction at the 10 mg dose). 1pt required a dose reduction per protocol due to a decrease in LVOT gradient to <20 mmHg. There was a decline in LV ejection fraction (LVEF), however, no patient had to discontinue treatment due to an LVEF <50%. A marked improvement in diastolic function was evident early, </span><span style="font-size:9.0pt">even before the complete resolution of LVOT obstruction</span><span style="font-size:9.0pt">, while right ventricular function parameters remained unchanged.</span></span></span></p> <p> </p> <p><span style="font-size:11pt"><span style="font-family:Calibri,sans-serif"><strong><span style="font-size:9.0pt">Conclusion: </span></strong><span style="font-size:9.0pt">Mavacamten significantly improved echocardiographic outcomes in this Portuguese cohort of symptomatic oHCM pts. These findings align with clinical trial data, confirming its efficacy in reducing obstruction and improving myocardial relaxation.</span></span></span></p>
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