Effect of Aficamten vs Metoprolol on Patient-Reported Health Status in Obstructive Hypertrophic Cardiomyopathy

作者：Michael E. Nassif, Pablo García‐Pavía, Ahmad Masri, Béla Merkely, María Luisa Peña‐Peña, Roberto Barriales‐Villa, Ozlem Bilen, Melissa Burroughs, Brian Claggett, Juan Pablo Costabel, Edileide de Barros Correia, Anne M. Dybro, Perry Elliott, Neal K. Lakdawala, Gregory D. Lewis, Amy Mann, Martin S. Maron, Zi Michael Miao, Ajith Nair, Steen Hvitfeldt Poulsen, Patricia Réant, P. Christian Schulze, Scott D. Solomon, Andrew Z. Wang, Regina Sohn, Indrias Berhane, Stephen B. Heitner, Daniel Jacoby, Stuart Kupfer, Fady I. Malik, Amy Wohltman, Michael A. Fifer, John A. Spertus · 发表于：Journal of the American College of Cardiology · 年份：2025 · DOI：10.1016/j.jacc.2025.10.059 · 被引用次数：2 · 研究领域：Cardiomyopathy and Myosin Studies、Heart Failure Treatment and Management、Cardiovascular Function and Risk Factors

BACKGROUND: The cardiac myosin inhibitor aficamten was significantly more effective than metoprolol at improving exercise tolerance in MAPLE-HCM (Metoprolol vs Aficamten in Patients with LVOT Obstruction on Exercise Capacity in HCM), a head-to-head, international, double-blind, randomized trial in patients with obstructive hypertrophic cardiomyopathy (oHCM). Given the primary treatment goal to improve patients' health status, defining the incremental benefits of aficamten over metoprolol on patients' symptoms, function, and quality of life is needed. OBJECTIVES: In this study, the authors sought to compare patient-reported health status benefits of aficamten with metoprolol. METHODS: Adults with symptomatic oHCM (Kansas City Cardiomyopathy Questionnaire [KCCQ] Clinical Summary Score [CSS] ≤90; left ventricular outflow tract obstruction ≥30 mm Hg at rest or ≥50 mm Hg with Valsalva) were randomly assigned to 24 weeks of aficamten or metoprolol as monotherapy. Changes in KCCQ Overall Summary Score (OSS) and Seattle Angina Questionnaire Summary Score (SAQ-SS), collected serially throughout the trial, were compared between treatment groups at 24 weeks using linear regression, adjusted for randomization strata and baseline scores. Individual participant experiences were described by comparing categories of clinically meaningful within-participant change: ≤-5 (worse), >-5 to <+5 (no change), +5 to <+10 (small improvement), +10 to <+15 (moderate improvement), +15 to <+20 (large impro...