2026-05-17T05:52:36Zhttps://repisalud.isciii.es/rest/oai/request

oai:repisalud.isciii.es:20.500.12105/203682024-09-27T08:58:44Zcom_20.500.12105_19604com_20.500.12105_2051col_20.500.12105_19605

00925njm 22002777a 4500 dc Masri, Ahmad author Sherrid, Mark V author Abraham, Theodore P author Choudhury, Lubna author Garcia-Pavia, Pablo author Kramer, Christopher M author Barriales-Villa, Roberto author Owens, Anjali T author Rader, Florian author Nagueh, Sherif F author Olivotto, Iacopo author Saberi, Sara author Tower-Rader, Albree author Wong, Timothy C author Coats, Caroline J author Watkins, Hugh author Fifer, Michael A author Solomon, Scott D author Heitner, Stephen B author Jacoby, Daniel L author Kupfer, Stuart author Malik, Fady I author Meng, Lisa author Sohn, Regina L author Wohltman, Amy author Maron, Martin S author 2024-03-15 BACKGROUND This open-label phase 2 trial evaluated the safety and efficacy of aficamten in patients with nonobstructive hypertrophic cardiomyopathy (nHCM). METHODS Patients with symptomatic nHCM (left ventricular outflow tract obstruction gradient ≤ 30 mmHg, left ventricular ejection fraction [LVEF] ≥ 60%, N-terminal pro-B-type natriuretic peptide [NT-proBNP] > 300 pg/mL) received aficamten 5-15 mg once daily (doses adjusted according to echocardiographic LVEF) for 10 weeks. RESULTS We enrolled 41 patients (mean ± SD age 56 ± 16 years; 59% female). At Week 10, 22 (55%) patients experienced an improvement of ≥ 1 New York Heart Association class; 11 (29%) became asymptomatic. Clinically relevant improvements in Kansas City Cardiomyopathy Questionnaire Clinical Summary Scores occurred in 22 (55%) patients. Symptom relief was paralleled by reductions in NT-proBNP levels (56%; P < 0.001) and high-sensitivity cardiac troponin I (22%; P < 0.005). Modest reductions in LVEF (mean ± SD) of -5.4% ± 10 to 64.6% ± 9.1 were observed. Three (8%) patients had asymptomatic reduction in LVEF < 50% (range: 41%-48%), all returning to normal after 2 weeks of washout. One patient with prior history of aborted sudden cardiac death experienced a fatal arrhythmia during the study. CONCLUSIONS Aficamten administration for symptomatic nHCM was generally safe and was associated with improvements in heart failure symptoms and cardiac biomarkers. TRIAL REGISTRATION ClinicalTrials.gov Identifier: NCT04219826. J Card Fail. 2024 Mar 15:S1071-9164(24)00082-4. 10.1016/j.cardfail.2024.02.020 1532-8414 Journal of cardiac failure 38493832 http://hdl.handle.net/20.500.12105/20368 Efficacy and Safety of Aficamten in Symptomatic Nonobstructive Hypertrophic Cardiomyopathy: Results From the REDWOOD-HCM Trial, Cohort 4.