Abstract：
OBJECTIVE: We aimed to comprehensively assess the safety and efficacy of mavacamten in hypertrophic cardiomyopathy (HCM) patients.
METHODS: A systematic review and meta-analysis was conducted, and efficacy [changes in postexercise left ventricular outflow tract (LVOT) gradient, left ventricular ejection fraction (LVEF), peak oxygen consumption (pVO 2 ), Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ CSS), and the proportion of patients exhibiting an improvement of at least one New York Heart Association (NYHA) functional class from baseline)], safety (total count of treatment-emergent adverse events and SAEs, as well as the proportion of patients experiencing at least one adverse event or SAE), and cardiac biomarkers (NT-proBNP and cTnI) outcomes were evaluated.
RESULTS: We incorporated data from four randomized controlled trials, namely EXPLORER-HCM, VALOR-HCM, MAVERICK-HCM, and EXPLORER-CN. Mavacamten demonstrated significant efficacy in reducing the postexercise LVOT gradient by 49.44 mmHg ( P = 0.0001) and LVEF by 3.84 ( P < 0.0001) and improving pVO 2 by 0.69 ml/kg/min ( P = 0.4547), KCCQ CSS by 8.11 points ( P < 0.0001), and patients with at least one NYHA functional class improvement from baseline by 2.20 times ( P < 0.0001). Importantly, mavacamten increased 1.11-fold adverse events ( P = 0.0184) 4.24-fold reduced LVEF to less than 50% ( P = 0.0233) and 1.06-fold SAEs ( P = 0.8631). Additionally, mavacamten decreased NT-proBNP by 528.62 ng/l ( P < 0.0001) and cTnI by 8.28 ng/l ( P < 0.0001).
CONCLUSIONS: Mavacamten demonstrates both safety and efficacy in patients with HCM, suggesting its potential as a promising therapeutic strategy for this condition. Further research is warranted to confirm these results and explore its long-term effects.
METHODS: A systematic review and meta-analysis was conducted, and efficacy [changes in postexercise left ventricular outflow tract (LVOT) gradient, left ventricular ejection fraction (LVEF), peak oxygen consumption (pVO 2 ), Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ CSS), and the proportion of patients exhibiting an improvement of at least one New York Heart Association (NYHA) functional class from baseline)], safety (total count of treatment-emergent adverse events and SAEs, as well as the proportion of patients experiencing at least one adverse event or SAE), and cardiac biomarkers (NT-proBNP and cTnI) outcomes were evaluated.
RESULTS: We incorporated data from four randomized controlled trials, namely EXPLORER-HCM, VALOR-HCM, MAVERICK-HCM, and EXPLORER-CN. Mavacamten demonstrated significant efficacy in reducing the postexercise LVOT gradient by 49.44 mmHg ( P = 0.0001) and LVEF by 3.84 ( P < 0.0001) and improving pVO 2 by 0.69 ml/kg/min ( P = 0.4547), KCCQ CSS by 8.11 points ( P < 0.0001), and patients with at least one NYHA functional class improvement from baseline by 2.20 times ( P < 0.0001). Importantly, mavacamten increased 1.11-fold adverse events ( P = 0.0184) 4.24-fold reduced LVEF to less than 50% ( P = 0.0233) and 1.06-fold SAEs ( P = 0.8631). Additionally, mavacamten decreased NT-proBNP by 528.62 ng/l ( P < 0.0001) and cTnI by 8.28 ng/l ( P < 0.0001).
CONCLUSIONS: Mavacamten demonstrates both safety and efficacy in patients with HCM, suggesting its potential as a promising therapeutic strategy for this condition. Further research is warranted to confirm these results and explore its long-term effects.
摘要：
目的：我们旨在全面评估mavacamten在肥厚型心肌病（HCM）患者中的安全性和有效性。
方法：进行了系统评价和荟萃分析，和功效[运动后左心室流出道（LVOT）梯度的变化，左心室射血分数(LVEF),峰值耗氧量(pVO2)，堪萨斯城心肌病问卷临床总结评分（KCCQCSS），以及表现出至少一个纽约心脏协会（NYHA）功能等级从基线改善的患者比例）]，安全性(治疗引起的不良事件和SAE的总数，以及经历至少一次不良事件或SAE的患者比例），和心脏生物标志物（NT-proBNP和cTnI）结果进行评估。
结果：我们纳入了四项随机对照试验的数据，即EXPLORER-HCM，VALOR-HCM,MAVERICK-HCM,和EXPLORER-CN.Mavacamten在降低运动后LVOT梯度49.44mmHg（P=0.0001）和LVEF3.84（P<0.0001）以及改善pVO20.69ml/kg/min（P=0.4547）方面具有显着的功效，KCCQCSS下降8.11分（P<0.0001），NYHA功能等级至少比基线改善2.20倍（P<0.0001）。重要的是,mavacamten增加了1.11倍的不良事件（P=0.0184），4.24倍降低LVEF至50％以下（P=0.0233）和1.06倍SAE（P=0.8631）。此外,mavacamten使NT-proBNP降低528.62ng/l(P<0.0001),cTnI降低8.28ng/l(P<0.0001)。
结论：Mavacamten证明了HCM患者的安全性和有效性，表明其作为一种有希望的治疗策略的潜力。需要进一步的研究来证实这些结果并探索其长期影响。
方法：进行了系统评价和荟萃分析，和功效[运动后左心室流出道（LVOT）梯度的变化，左心室射血分数(LVEF),峰值耗氧量(pVO2)，堪萨斯城心肌病问卷临床总结评分（KCCQCSS），以及表现出至少一个纽约心脏协会（NYHA）功能等级从基线改善的患者比例）]，安全性(治疗引起的不良事件和SAE的总数，以及经历至少一次不良事件或SAE的患者比例），和心脏生物标志物（NT-proBNP和cTnI）结果进行评估。
结果：我们纳入了四项随机对照试验的数据，即EXPLORER-HCM，VALOR-HCM,MAVERICK-HCM,和EXPLORER-CN.Mavacamten在降低运动后LVOT梯度49.44mmHg（P=0.0001）和LVEF3.84（P<0.0001）以及改善pVO20.69ml/kg/min（P=0.4547）方面具有显着的功效，KCCQCSS下降8.11分（P<0.0001），NYHA功能等级至少比基线改善2.20倍（P<0.0001）。重要的是,mavacamten增加了1.11倍的不良事件（P=0.0184），4.24倍降低LVEF至50％以下（P=0.0233）和1.06倍SAE（P=0.8631）。此外,mavacamten使NT-proBNP降低528.62ng/l(P<0.0001),cTnI降低8.28ng/l(P<0.0001)。
结论：Mavacamten证明了HCM患者的安全性和有效性，表明其作为一种有希望的治疗策略的潜力。需要进一步的研究来证实这些结果并探索其长期影响。
