Por favor, use este identificador para citar o enlazar este Item:http://hdl.handle.net/20.500.12105/20368
Título
Efficacy and Safety of Aficamten in Symptomatic Nonobstructive Hypertrophic Cardiomyopathy: Results From the REDWOOD-HCM Trial, Cohort 4.
Autor(es)
Masri, Ahmad | Sherrid, Mark V | Abraham, Theodore P | Choudhury, Lubna | Garcia-Pavia, Pablo CNIC | Kramer, Christopher M | Barriales-Villa, Roberto | Owens, Anjali T | Rader, Florian | Nagueh, Sherif F | Olivotto, Iacopo | Saberi, Sara | Tower-Rader, Albree | Wong, Timothy C | Coats, Caroline J | Watkins, Hugh | Fifer, Michael A | Solomon, Scott D | Heitner, Stephen B | Jacoby, Daniel L | Kupfer, Stuart | Malik, Fady I | Meng, Lisa | Sohn, Regina L | Wohltman, Amy | Maron, Martin S
Fecha de publicación
2024-03-15
Cita
J Card Fail. 2024 Mar 15:S1071-9164(24)00082-4.
Idioma
Inglés
Tipo de documento
journal article
Resumen
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.
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