Aficamten in Obstructive Hypertrophic Cardiomyopathy

作者：Andrew Wang, Pablo García‐Pavía, Ahmad Masri, Béla Merkely, Michael E. Nassif, 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, Sheila M. Hegde, Ian J. Kulac, Neal K. Lakdawala, Gregory D. Lewis, Amy Mann, Ajith Nair, Steen Hvitfeldt Poulsen, Patricia Réant, P. Christian Schulze, Scott D. Solomon, Regina Sohn, Indrias Berhane, Stephen B. Heitner, Daniel Jacoby, Stuart Kupfer, Fady I. Malik, Amy Wohltman, Michael A. Fifer, Martin S. Maron · 发表于：Journal of the American College of Cardiology · 年份：2025 · DOI：10.1016/j.jacc.2025.10.057 · 被引用次数：4 · 研究领域：Cardiomyopathy and Myosin Studies、Cardiovascular Function and Risk Factors、Heart Failure Treatment and Management

BACKGROUND: For >60 years, beta-blockers have been first-line therapy for symptomatic obstructive hypertrophic cardiomyopathy (oHCM), although limited data support this recommendation. The MAPLE-HCM trial showed greater improvement in exercise capacity and secondary endpoints for aficamten monotherapy vs metoprolol monotherapy. OBJECTIVES: The purpose of this study was to evaluate the effects of aficamten vs metoprolol across multiple clinically relevant and patient-centric measures of disease burden in oHCM. METHODS: ); and ≥10% reduction in left atrial volume index. Clinical response was classified by number of efficacy outcomes achieved: nonresponder (none), limited (1 or 2), positive (3 or 4), or complete (all 5). RESULTS: was 19.9 ± 5.1 mL/kg/min, and mean KCCQ-CSS score was 66 ± 16. At 24 weeks, aficamten treatment was associated with greater benefits in all efficacy outcome measures, with numbers needed to treat ranging from 1.5 to 5.0 relative to metoprolol treatment. Patients treated with aficamten were more likely to be either positive or complete responders compared with patients treated with metoprolol (78% vs 3%, respectively; P < 0.001). CONCLUSIONS: In patients with symptomatic oHCM, aficamten monotherapy led to superior and rapid treatment benefits across several clinically relevant outcome measures of disease burden compared with metoprolol. These observations support the emerging role of aficamten as monotherapy for oHCM. (Phase 3 Trial to Evaluate the Effic...