{"id":"85c10f9e3b14","type":"article","url":"https://hartvaat.nl/2023/01/03/aficamten-bij-obstructieve-hypertrofische-cardiomyopathie-fase-2-resultaten/","title":"Aficamten bij obstructieve hypertrofische cardiomyopathie: fase 2 resultaten","title_en":"Phase 2 Study of Aficamten in Patients With Obstructive Hypertrophic Cardiomyopathy.","category":"hartfalen","category_label":"Hartfalen","professions":["cardioloog"],"tags":["acuut-hartfalen","aficamten","aritmogene-cardiomyopathie","gedilateerde-cardiomyopathie","hypertrofische-cardiomyopathie","iaso-dcm","immunoadsorptie","laminopathie","mavacamten"],"journal":"Journal of the American College of Cardiology","doi":"10.1016/j.jacc.2022.10.020","source_url":"https://doi.org/10.1016/j.jacc.2022.10.020","authors":["Martin S Maron","Ahmad Masri","Lubna Choudhury","Iacopo Olivotto","Sara Saberi","Andrew Wang","Pablo Garcia-Pavia","Neal K Lakdawala","Sherif F Nagueh","Florian Rader","Albree Tower-Rader","Aslan T Turer","Caroline Coats","Michael A Fifer","Anjali Owens","Scott D Solomon","Hugh Watkins","Roberto Barriales-Villa","Christopher M Kramer","Timothy C Wong","Sharon L Paige","Stephen B Heitner","Stuart Kupfer","Fady I Malik","Lisa Meng","Amy Wohltman","Theodore Abraham"],"significance":8,"published":"2023-01-03","source_date":"2023-01-03","image":"","kennis":["https://hartvaat.nl/kennis/hartfalen/hypertrofische-cardiomyopathie/"],"congress":"","summary_en":"This phase 2 study of aficamten, a next-generation cardiac myosin inhibitor, showed dose-dependent reduction of the LVOT gradient and symptom improvement in patients with obstructive hypertrophic cardiomyopathy. The results advanced aficamten toward phase 3 development as an alternative to mavacamten.","created":"2026-07-03T10:30:09Z","updated":"2026-07-03T18:38:59Z","licence":"Citeer vrij, met bronvermelding en een link naar hartvaat.nl (de url van het record). Samenvattingen zijn redactioneel werk van HartVaat; de oorspronkelijke publicaties blijven van hun uitgevers (doi). Geen medisch advies.","body_markdown":"Fase 2 studie van aficamten, een volgende-generatie cardiale myosineremmer, toonde dosisafhankelijke verlaging van de LVOT-gradiënt en verbetering van symptomen bij obstructieve HCM. Aficamten is een veelbelovend alternatief voor mavacamten met een breder therapeutisch venster.","abstract_original":"BACKGROUND: Left ventricular outflow tract (LVOT) obstruction is a major determinant of heart failure symptoms in obstructive hypertrophic cardiomyopathy (oHCM). Aficamten, a next-in-class cardiac myosin inhibitor, may lower gradients and improve symptoms in these patients. OBJECTIVES: This study aims to evaluate the safety and efficacy of aficamten in patients with oHCM. METHODS: Patients with oHCM and LVOT gradients ≥30 mm Hg at rest or ≥50 mm Hg with Valsalva were randomized 2:1 to receive aficamten (n = 28) or placebo (n = 13) in 2 dose-finding cohorts. Doses were titrated based on gradients and ejection fraction (EF). Safety and changes in gradient, EF, New York Heart Association functional class, and cardiac biomarkers were assessed over a 10-week treatment period and after a 2-week washout. RESULTS: From baseline to 10 weeks, aficamten reduced gradients at rest (mean difference: -40 ± 27 mm Hg, and -43 ± 37 mm Hg in Cohorts 1 and 2, P = 0.0003 and P = 0.0004 vs placebo, respectively) and with Valsalva (-36 ± 27 mm Hg and -53 ± 44 mm Hg, P = 0.001 and <0.0001 vs placebo, respectively). There were modest reductions in EF (-6% ± 7.5% and -12% ± 5.9%, P = 0.007 and P < 0.0001 vs placebo, respectively). Symptomatic improvement in ≥1 New York Heart Association functional class was observed in 31% on placebo, and 43% and 64% on aficamten in Cohorts 1 and 2, respectively (nonsignificant). With aficamten, N-terminal pro-B-type natriuretic peptide was reduced (62% relative to placebo, P = 0.0002). There were no treatment interruptions and adverse events were similar between treatment arms. CONCLUSIONS: Aficamten resulted in substantial reductions in LVOT gradients with most patients experiencing improvement in biomarkers and symptoms. These results highlight the potential of sarcomere-targeted therapy for treatment of oHCM."}