{"id":"f1449bb02ce2","type":"article","url":"https://hartvaat.nl/2026/03/05/aficamten-veilig-en-effectief-bij-langdurige-behandeling-van-obstructieve-hcm/","title":"Aficamten veilig en effectief bij langdurige behandeling van obstructieve HCM","title_en":"Aficamten in symptomatic obstructive hypertrophic cardiomyopathy: the FOREST-HCM long-term study.","category":"hartfalen","category_label":"Hartfalen","professions":["cardioloog"],"tags":["aficamten","cardiomyopathie-gerichte-therapie","gedilateerde-cardiomyopathie","hypertrofische-cardiomyopathie","laminopathie","mavacamten"],"journal":"European heart journal","doi":"10.1093/eurheartj/ehaf1085","source_url":"https://doi.org/10.1093/eurheartj/ehaf1085","authors":["Albree Tower-Rader","Ahmad Masri","Michael E Nassif","Theodore P Abraham","Roberto Barriales-Villa","Lubna Choudhury","Robert M Cooper","Perry M Elliott","Martin S Maron","Iacopo Olivotto","Artur Oreziak","Anjali Tiku Owens","Scott D Solomon","Stephen B Heitner","Daniel L Jacoby","Stuart Kupfer","Xueli Liu","Fady I Malik","Chiara Melloni","Tyrell J Simkins","Jenny Wei","Sara Saberi"],"significance":7,"published":"2026-03-21","source_date":"2026-03-05","image":"","kennis":["https://hartvaat.nl/kennis/hartfalen/hypertrofische-cardiomyopathie/"],"congress":"","summary_en":"The FOREST-HCM long-term study confirmed that sustained treatment with aficamten, a selective cardiac myosin inhibitor, is safe and effective for reducing LVOT obstruction and improving symptoms in hypertrophic cardiomyopathy.","created":"2026-07-03T10:32:36Z","updated":"2026-07-03T13:31:33Z","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":"De FOREST-HCM-studie in het EHJ toont aan dat langdurige behandeling met aficamten, een selectieve cardiale myosineremmer, veilig en effectief is bij patiënten met symptomatische obstructieve hypertrofische cardiomyopathie. Na eerdere korte-termijndata bevestigt dit de duurzaamheid van het klinisch voordeel.","abstract_original":"BACKGROUND AND AIMS: Aficamten is a next-in-class, oral selective cardiac myosin inhibitor that ameliorates hypercontractility in hypertrophic cardiomyopathy (HCM). This study assessed the safety and efficacy of extended aficamten treatment in symptomatic obstructive HCM (oHCM). METHODS: Patients completing a parent aficamten study were eligible to enrol in FOREST-HCM (NCT04848506), an open-label study evaluating long-term aficamten treatment. RESULTS: Patients with oHCM (N = 296; mean age ±SD 61 ± 12.3 years, 44.3% female) enrolled between May 2021 and August 2024. Cumulative exposure was 352 patient-years; median follow-up 51.6 (IQR 41.5, 70.8) weeks. At Weeks 12 and 96, aficamten reduced Valsalva left ventricular outflow tract gradient by 56 ± 43 and 62 ± 33 mmHg from baseline (both P < 0.0001), with minimal reduction in left ventricular ejection fraction (LVEF) (-3% ± 6% and -5% ± 5%); 69% and 93% of participants had at least one NYHA class improvement; Kansas City Cardiomyopathy Questionnaire-Clinical Summary Score improved by 15 ± 16 and 16 ± 16 points. Treatment-emergent serious adverse events (TESAEs) occurred in 36 (12.2%) patients; no deaths, heart failure, or events considered related to aficamten were reported. One (0.3%) patient terminated therapy due to a TESAE (ischemic colitis). LVEF<50% occurred in 10 (3.4%) patients [exposure-adjusted incidence rate (EAIR): 2.9 per 100 patient-years] with 2 having non-serious mild/moderate dyspnoea. No treatment interruptions for LVEF<50%, and no events of LVEF<40% occurred. New-onset atrial fibrillation occurred in seven (2.4%) patients (EAIR 2.0 per 100 patient-years). CONCLUSIONS: Extended aficamten treatment in patients with symptomatic oHCM yielded early and sustained hemodynamic and clinical responses with low incidences of new-onset atrial fibrillation and LVEF<50%."}