Cardiology and therapy · 2026
Mavacamten in Chinese Patients with Obstructive Hypertrophic Cardiomyopathy: Patient-Reported Health Status Analysis up to 78 Weeks in the EXPLORER-CN Study.
СтатьяНазвание и аннотация: Перевод в очереди
Обработка статьи · этапы, ошибки и управление
Загрузка состояния…
Ошибки и ограничения
История · было → стало
Состояние обновляется каждые 5 секунд, пока блок открыт. Процент показывается только там, где известен объём работы. Пауза останавливает продолжение этапа; текущий запрос к источнику может завершиться.
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, Memaj Arteid, Xie Yimeng, Pan Beth, Florea Victoria, Zheng Jing, Zhong Yue, Zhang Shuyang
Аннотация
INTRODUCTION: Improvement of symptoms, physical function, and quality of life is one treatment goal for patients with obstructive hypertrophic cardiomyopathy (HCM). Mavacamten improved left-ventricular outflow tract gradients in Chinese patients with obstructive HCM in the EXPLORER-CN trial. We report here a detailed analysis of patient-reported health status per the 23-item Kansas City Cardiomyopathy Questionnaire (KCCQ-23) in Chinese patients after 78 weeks of mavacamten.
METHODS: Patients who completed the double-blind, placebo-controlled (DBPC) period of EXPLORER-CN with no active safety concerns could enter a long-term extension (LTE) period to receive mavacamten for 48 weeks at either the 30-week dose (mavacamten-mavacamten group) or a once-daily starting dose of 2.5 mg (subsequently adjusted via pharmacodynamics-based dose titration: placebo-mavacamten group). Health status endpoints included KCCQ-23 clinical summary score (CSS), overall symptom score (OSS), total symptom score (TSS), physical limitations score, and quality-of-life scores through week 78. Week 78 endpoint analyses were descriptive.
RESULTS: In the DBPC period, 54 patients received mavacamten and 27 received placebo; of these, 54 and 25, respectively, entered the LTE period. KCCQ-23 CSS, OSS, and TSS improved with mavacamten and worsened with placebo during the DBPC period (mean change baseline to week 30: 5.7, 6.4, and 8.1, respectively [mavacamten-mavacamten group] and - 5.4, - 4.3, and - 4.8, respectively [placebo-mavacamten group]). CSS, OSS, and TSS continued to improve in the mavacamten-mavacamten group during the LTE period (mean change baseline to week 78: 7.1, 8.2, and 10.0, respectively). Scores improved in the placebo-mavacamten group after switching to mavacamten (mean change week 30 to week 78: 7.3, 9.5, and 5.7, respectively). Similar improvements in KCCQ-23 physical limitations and quality-of-life scores with mavacamten were observed throughout both study periods.
CONCLUSIONS: Long-term mavacamten treatment for up to 78 weeks led to sustained improvements in patient-reported health status, supporting long-term treatment with mavacamten for Chinese patients with symptomatic obstructive HCM.
TRIAL REGISTRATION: ClinicalTrials.gov identifier NCT05174416.
METHODS: Patients who completed the double-blind, placebo-controlled (DBPC) period of EXPLORER-CN with no active safety concerns could enter a long-term extension (LTE) period to receive mavacamten for 48 weeks at either the 30-week dose (mavacamten-mavacamten group) or a once-daily starting dose of 2.5 mg (subsequently adjusted via pharmacodynamics-based dose titration: placebo-mavacamten group). Health status endpoints included KCCQ-23 clinical summary score (CSS), overall symptom score (OSS), total symptom score (TSS), physical limitations score, and quality-of-life scores through week 78. Week 78 endpoint analyses were descriptive.
RESULTS: In the DBPC period, 54 patients received mavacamten and 27 received placebo; of these, 54 and 25, respectively, entered the LTE period. KCCQ-23 CSS, OSS, and TSS improved with mavacamten and worsened with placebo during the DBPC period (mean change baseline to week 30: 5.7, 6.4, and 8.1, respectively [mavacamten-mavacamten group] and - 5.4, - 4.3, and - 4.8, respectively [placebo-mavacamten group]). CSS, OSS, and TSS continued to improve in the mavacamten-mavacamten group during the LTE period (mean change baseline to week 78: 7.1, 8.2, and 10.0, respectively). Scores improved in the placebo-mavacamten group after switching to mavacamten (mean change week 30 to week 78: 7.3, 9.5, and 5.7, respectively). Similar improvements in KCCQ-23 physical limitations and quality-of-life scores with mavacamten were observed throughout both study periods.
CONCLUSIONS: Long-term mavacamten treatment for up to 78 weeks led to sustained improvements in patient-reported health status, supporting long-term treatment with mavacamten for Chinese patients with symptomatic obstructive HCM.
TRIAL REGISTRATION: ClinicalTrials.gov identifier NCT05174416.