Aficamten and Patient-Reported Outcomes in Real-World nHCM

• Age 18-85 years.

• Confirmed diagnosis of HCM:

  1. Left ventricular wall thickness ≥15 mm at any site on echocardiography or cardiac magnetic resonance.
  2. Left ventricular wall thickness ≥13 mm in individuals testing positive for a pathogenic variant or in genetically affected family members.
  3. Ventricular wall thickening caused by other cardiovascular diseases or systemic or metabolic disorders has been excluded.

• Symptomatic nHCM patients meeting all of the following conditions:

  1. Left ventricular outflow tract gradient (LVOTG) \<30 mmHg at rest and/or with provocation.
  2. Presence of any one of the following symptoms: fatigue (weakness), dyspnea, chest pain, palpitations, or syncope.
  3. New York Heart Association (NYHA) functional class II or III.
• Left ventricular ejection fraction (LVEF) ≥60% as assessed by transthoracic echocardiography.
• Signed and dated written informed consent, and willingness to return for clinical follow-up.

Exclusion Criteria:

• LVOTG ≥30 mmHg at rest and/or with provocation.
• LVEF \<50% on transthoracic echocardiography.
• Symptomatic hypotension or systolic blood pressure (SBP) \<100 mmHg.
• Severe renal impairment, estimated glomerular filtration rate (eGFR) \<30 mL/min/1.73 m², or currently receiving blood purification therapy.
• Planned implantable cardioverter-defibrillator (ICD) implantation or cardiac resynchronization therapy (CRT) within 3 months.
• Women who are pregnant or planning to become pregnant during the trial.
• Currently participating in another investigational device or drug trial.
• Any other clinical condition with an expected survival of less than 2 years.