Mavacamten for Improving Cardiac Function and Clinical Outcomes in Adult Patients With Obstructive Hypertrophic Cardiomyopathy: A Retrospective Cohort Study
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 222
- 试验地点
- 1
- 主要终点
- Change in Resting LVOT Gradient at Week 30
研究概览
简要总结
This retrospective cohort study aims to evaluate the clinical efficacy and safety of mavacamten in adult patients with obstructive hypertrophic cardiomyopathy (oHCM). A total of 222 patients were included and categorized based on treatments received in routine clinical practice into a mavacamten group and a standard therapy group.
The primary outcome is the change in resting left ventricular outflow tract (LVOT) gradient at Week 30. Secondary outcomes include changes in Valsalva LVOT gradient, New York Heart Association (NYHA) functional class, cardiac biomarkers, and echocardiographic parameters. Safety outcomes include adverse events and left ventricular systolic dysfunction.
This study provides real-world evidence on the effectiveness and safety of mavacamten in Chinese patients with oHCM.
详细描述
Hypertrophic cardiomyopathy (HCM) is a genetic cardiac disorder characterized by left ventricular hypertrophy, with approximately two-thirds of patients presenting with obstructive physiology. Obstructive hypertrophic cardiomyopathy (oHCM) is associated with significant morbidity due to left ventricular outflow tract (LVOT) obstruction.
Mavacamten, a first-in-class cardiac myosin inhibitor, directly targets the underlying pathophysiology of oHCM by reducing excessive myosin-actin cross-bridge formation. Although previous randomized clinical trials, such as EXPLORER-CN, have demonstrated its efficacy, real-world data in Chinese populations remain limited.
This study is a retrospective cohort study conducted at Fuwai Central China Cardiovascular Hospital. Patients were not assigned to interventions. Instead, they were categorized based on treatments received in routine clinical practice. Eligible adult patients with oHCM who initiated mavacamten therapy were included in the treatment group, while patients receiving standard pharmacological therapy served as the control group.
Clinical data were collected from January 2024 to May 2025, with final database lock completed in June 2025. Baseline characteristics, echocardiographic parameters, and laboratory biomarkers were obtained from medical records.
The primary endpoint was the change in resting LVOT peak gradient at Week 30 compared with baseline. Secondary endpoints included changes in Valsalva LVOT gradient, NYHA functional class improvement, NT-proBNP, high-sensitivity cardiac troponin I (hs-cTnI), left ventricular ejection fraction (LVEF), and tricuspid annular plane systolic excursion (TAPSE). Safety endpoints included adverse events, serious adverse events, treatment discontinuation, and reduction of LVEF below 50%.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age ≥18 years
- •Diagnosis of hypertrophic cardiomyopathy
- •NYHA functional class II-III
- •First-time treatment with mavacamten
排除标准
- •Known hypersensitivity to mavacamten or its components
- •Left ventricular ejection fraction (LVEF) <55%
- •Septal reduction therapy within 6 months prior to enrollment
- •Presence of other conditions requiring long-term anticoagulation
- •Pregnancy or breastfeeding
- •Any condition deemed unsuitable by the investigator
研究组 & 干预措施
Mavacamten Group
Adult patients with obstructive hypertrophic cardiomyopathy (oHCM) who received mavacamten as part of routine clinical practice. Patients were not assigned to interventions but were categorized based on treatment received.
干预措施: mavacamten (Drug)
Standard Therapy Group
Adult patients with obstructive hypertrophic cardiomyopathy (oHCM) who received standard pharmacological therapy, including beta-blockers or non-dihydropyridine calcium channel blockers, in routine clinical practice.
结局指标
主要结局
Change in Resting LVOT Gradient at Week 30
时间窗: Baseline to Week 30
Change from baseline in resting left ventricular outflow tract (LVOT) peak gradient measured by echocardiography.
Change in Resting Left Ventricular Outflow Tract (LVOT) Peak Gradient at Week 30
时间窗: Baseline to Week 30
Change from baseline in resting left ventricular outflow tract (LVOT) peak gradient measured by Doppler echocardiography (unit: mmHg). Higher values indicate greater obstruction.
次要结局
- Change in Valsalva LVOT Gradient at Week 30(Baseline to Week 30)
- Improvement in NYHA Functional Class at Week 30(Baseline to Week 30)
- Change in NT-proBNP at Week 30(Baseline to Week 30)
- Change in High-Sensitivity Cardiac Troponin I at Week 30(Baseline to Week 30)
- Change in Left Ventricular Ejection Fraction at Week 30(Baseline to Week 30)
- Change in Tricuspid Annular Plane Systolic Excursion at Week 30(Baseline to Week 30)
- Incidence of Adverse Events(Up to Week 30)
- Change in Valsalva-Induced Left Ventricular Outflow Tract (LVOT) Peak Gradient at Week 30(Baseline to Week 30)
- Proportion of Participants with Improvement in New York Heart Association (NYHA) Functional Classification at Week 30(Baseline to Week 30)
- Change in N-terminal pro-B-type Natriuretic Peptide (NT-proBNP) Levels at Week 30(Baseline to Week 30)
- Change in High-Sensitivity Cardiac Troponin I (hs-cTnI) Levels at Week 30(Baseline to Week 30)
- Change in Left Ventricular Ejection Fraction (LVEF) at Week 30(Baseline to Week 30)
- Change in Tricuspid Annular Plane Systolic Excursion (TAPSE) at Week 30(Baseline to Week 30)
- Incidence of Serious Adverse Events(Up to Week 30)
- Incidence of Treatment Discontinuation Due to Adverse Events(Up to Week 30)
- Incidence of Left Ventricular Ejection Fraction Reduction Below 50%(Up to Week 30)
研究者
Yanan Shi
Cardiologist
Chinese Academy of Medical Sciences, Fuwai Hospital