Hasana Uswa, Korpal Mayank, Haider Faseeh, Umer Mohammad, Lwin Nang Phyu Thant, Iqbal Sohaba, Kumar Rajanikant, Ahsan Maria, Hassan Ebaad, Ullah Waheed, Khalid Saif, Gupta Akhil, Alsubari Asma'a Munasar Ali, Ehsan Muhammad, Ahmad Adeel
Fatima Jinnah Medical University, Lahore, Pakistan.
Government Medical College, Amritsar, India.
Ann Med Surg (Lond). 2025 Apr 29;87(6):3810-3818. doi: 10.1097/MS9.0000000000003326. eCollection 2025 Jun.
BACKGROUND/OBJECTIVE: HCM is a structural disorder of the myocardium that leads to sudden cardiac death in young adults. We synthesized an updated understanding of the role of Cardiac Myosin Inhibitors (CMIs) in HCM by pooling data from RCTs.
We identified six published RCTs, involving 826 participants. Data were extracted pertaining to study characteristics; primary outcomes of interest-(1) change from baseline in resting left ventricular outflow tract (LVOT) peak gradient, (2) change from baseline in Valsalva LVOT peak gradient, and (3) improvement of ≥1 NYHA class-and secondary outcomes. These were pooled using Review Manager 5.4, employing a random-effects model, and reported as odds ratios (ORs) or mean differences (MDs).
We found statistically significant between-group difference favoring CMIs in change from baseline in LVOT peak gradient: at rest (MD -39.33; -53.01 to -25.64), post-Valsalva (MD -48.99; -53.96 to -44.03), and post-exercise (MD -37.11; -44.34 to -29.87); ≥1 NYHA class improvement (OR 4.10; 2.79-6.02), change from baseline in peak oxygen uptake (MD -37.11; -44.34 to -29.87), LVOT gradient ≤30 mm hg (RR 14.89; 7.47-29.67), participants eligible for septal reduction therapy (RR 0.26; 0.18-0.36), and change from baseline in KCCQ-CSS score (MD 8.54; 5.36-11.71). Subgrouping by intervention type (mavacamten vs. aficamten) revealed non-significant results for all primary outcomes.
CMIs can contribute to improving key efficacy outcomes for patients with HCM while reducing incidence of SRT.
背景/目的：肥厚型心肌病（HCM）是一种心肌结构紊乱疾病，可导致年轻成年人心脏性猝死。我们通过汇总随机对照试验（RCT）的数据，综合了解了心肌肌球蛋白抑制剂（CMIs）在HCM中的作用。
我们确定了6项已发表的RCT，涉及826名参与者。提取了与研究特征相关的数据；感兴趣的主要结局——（1）静息左心室流出道（LVOT）峰值梯度相对于基线的变化，（2）瓦尔萨尔瓦动作时LVOT峰值梯度相对于基线的变化，以及（3）纽约心脏协会（NYHA）心功能分级改善≥1级——以及次要结局。使用RevMan 5.4进行汇总，采用随机效应模型，并报告为比值比（OR）或均值差（MD）。
我们发现，在LVOT峰值梯度相对于基线的变化方面，CMIs组间差异具有统计学意义：静息时（MD -39.33；-53.01至-25.64）、瓦尔萨尔瓦动作后（MD -48.99；-53.96至-44.03）和运动后（MD -37.11；-44.34至-29.87）；NYHA心功能分级改善≥1级（OR 4.10；2.79 - 6.02）、峰值摄氧量相对于基线的变化（MD -37.11；-44.34至-29.87）、LVOT梯度≤30 mmHg（RR 14.89；7.4个7至29.67）、符合室间隔减容治疗条件的参与者（RR 0.26；0.18至0.36）以及堪萨斯城心肌病问卷临床总结（KCCQ-CSS）评分相对于基线的变化（MD 8.54；5.36至11.71）。按干预类型（mavacamten与aficamten）进行亚组分析，所有主要结局均显示无显著结果。
CMIs有助于改善HCM患者的关键疗效结局，同时降低室间隔减容治疗的发生率。
