The Therapeutic Value of Mavacamten in Hypertrophic Cardiomyopathy With Mid-to-Apical Left Ventricular Obstruction

Lab values

• Obstructive hypertrophic cardiomyopathy (HCM), defined as a maximal left ventricular outflow tract pressure gradient (LVOT-PGmax) ≥30 mmHg at rest and during the Valsalva maneuver on echocardiography;
  • Maximal right ventricular outflow tract pressure gradient (RVOT-PGmax) ≥16 mmHg at rest; ③ Left ventricular ejection fraction (LVEF) \<50% on echocardiography;
  • Uncontrolled primary hypertension;
  • Moderate or severe aortic valve stenosis and/or primary mitral valve disease with severe mitral regurgitation; ⑥ Known infiltrative or storage disorders mimicking the HCM phenotype (e.g., Fabry disease, cardiac amyloidosis); ⑦ Presence of severe infections, hepatic dysfunction, renal impairment, or other serious conditions significantly affecting life expectancy.

Other

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• Patients diagnosed with HCM according to the 2023 Chinese Guidelines for the Diagnosis and Treatment of Adult Hypertrophic Cardiomyopathy, meeting one of the following:
  • Left ventricular wall thickness ≥15 mm at end-diastole in any segment as assessed by echocardiography or cardiac magnetic resonance imaging (CMR);
  • Left ventricular wall thickness ≥13 mm in individuals with a confirmed pathogenic gene mutation or in genetically affected family members;
  • Exclusion of other cardiovascular, systemic, or metabolic disorders that may cause ventricular hypertrophy.
  • Symptomatic non-outflow tract obstructive HCM patients (meeting criterion a and at least one of b or c):
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  • Presence of clinical symptoms such as dyspnea, chest pain, dizziness, palpitations, or syncope, with New York Heart Association (NYHA) functional class II-III;
  • Maximal pressure gradient (PGmax) \>30 mmHg in the mid-ventricle under resting or Valsalva maneuver as assessed by echocardiography;
  • PGmax \>30 mmHg in the apical region under resting or Valsalva maneuver on echocardiography.
  • ③Ability to provide written informed consent (ICF) and any required privacy authorization prior to study enrollment.