Journal of the American Heart Association · 2026
Long-Term Efficacy and Safety of Mavacamten in Chinese Patients With Obstructive Hypertrophic Cardiomyopathy: Week 78 Results From the EXPLORER-CN Study.
СтатьяРандомизированное исследованиеМногоцентровое исследованиеПоддержка исследования: вне правительства СШАНазвание и аннотация: Перевод в очереди
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Tian Zhuang, Li Xiaoyan, Li Liwen, Zhang Qing, Wang Jian'an, Shi Yunqi, Peng Daoquan, Ma Wei, Yang Ping, Cheng Xiang, Jin Wei, Wang Fang, Xie Yimeng, Pan Beth, Florea Victoria, Zhang Shuyang
Аннотация
BACKGROUND: Long-term efficacy and safety of mavacamten in Chinese patients with obstructive hypertrophic cardiomyopathy are unknown.
METHODS: Patients who completed the 30-week, double-blind, placebo-controlled treatment period in EXPLORER-CN (A Study to Evaluate the Efficacy and Safety of Mavacamten in Chinese Adults With Symptomatic Obstructive HCM), with no active safety concerns, were eligible to enter the long-term extension period to receive 48-week mavacamten treatment. Patients previously on mavacamten continued mavacamten (dose at week 30; mavacamten-mavacamten group); patients previously on placebo received mavacamten (starting dose, 2.5 mg once daily; placebo-mavacamten group). Key efficacy end points included change from baseline in echocardiographic measures, New York Heart Association functional class, 23-item Kansas City Cardiomyopathy Questionnaire Clinical Summary Score, and cardiac biomarkers through week 78. Analyses were descriptive; no between-group hypothesis testing was performed.
RESULTS: Seventy-nine patients (mean age, 51.6 years; 27.8% women) entered the long-term extension period (mavacamten-mavacamten, n=54; placebo-mavacamten, n=25). In the mavacamten-mavacamten group, numerical improvements in Valsalva and resting left ventricular outflow tract peak gradients were maintained through week 78 (mean change from baseline, -73.0 mm Hg [95% CI, -86.4 to -59.5]; and -56.3 mm Hg [95% CI, -67.1 to -45.5], respectively). Numerical improvements were also observed for other echocardiographic parameters, New York Heart Association class, Kansas City Cardiomyopathy Questionnaire Clinical Summary Score, and cardiac biomarkers through week 78. In the placebo-mavacamten group, numerical improvements with mavacamten in these parameters were noted from week 30 to week 78. Long-term mavacamten treatment appeared well tolerated; left ventricular ejection fraction <50% was rare.
CONCLUSIONS: In this exploratory long-term extension study, 78-week mavacamten treatment appeared well-tolerated and was associated with numerical improvements from baseline in echocardiographic parameters, New York Heart Association class, patient-reported health status, and cardiac biomarkers in Chinese patients with obstructive hypertrophic cardiomyopathy.
REGISTRATION: URL: https://www.clinicaltrials.gov; Unique Identifier: NCT05174416.
METHODS: Patients who completed the 30-week, double-blind, placebo-controlled treatment period in EXPLORER-CN (A Study to Evaluate the Efficacy and Safety of Mavacamten in Chinese Adults With Symptomatic Obstructive HCM), with no active safety concerns, were eligible to enter the long-term extension period to receive 48-week mavacamten treatment. Patients previously on mavacamten continued mavacamten (dose at week 30; mavacamten-mavacamten group); patients previously on placebo received mavacamten (starting dose, 2.5 mg once daily; placebo-mavacamten group). Key efficacy end points included change from baseline in echocardiographic measures, New York Heart Association functional class, 23-item Kansas City Cardiomyopathy Questionnaire Clinical Summary Score, and cardiac biomarkers through week 78. Analyses were descriptive; no between-group hypothesis testing was performed.
RESULTS: Seventy-nine patients (mean age, 51.6 years; 27.8% women) entered the long-term extension period (mavacamten-mavacamten, n=54; placebo-mavacamten, n=25). In the mavacamten-mavacamten group, numerical improvements in Valsalva and resting left ventricular outflow tract peak gradients were maintained through week 78 (mean change from baseline, -73.0 mm Hg [95% CI, -86.4 to -59.5]; and -56.3 mm Hg [95% CI, -67.1 to -45.5], respectively). Numerical improvements were also observed for other echocardiographic parameters, New York Heart Association class, Kansas City Cardiomyopathy Questionnaire Clinical Summary Score, and cardiac biomarkers through week 78. In the placebo-mavacamten group, numerical improvements with mavacamten in these parameters were noted from week 30 to week 78. Long-term mavacamten treatment appeared well tolerated; left ventricular ejection fraction <50% was rare.
CONCLUSIONS: In this exploratory long-term extension study, 78-week mavacamten treatment appeared well-tolerated and was associated with numerical improvements from baseline in echocardiographic parameters, New York Heart Association class, patient-reported health status, and cardiac biomarkers in Chinese patients with obstructive hypertrophic cardiomyopathy.
REGISTRATION: URL: https://www.clinicaltrials.gov; Unique Identifier: NCT05174416.