Mavacamten in Symptomatic Patients Resistant to Previous Advanced Therapy for Obstructive Hypertrophic Cardiomyopathy

Daniele Massera1, Elizabeth Adlestein1, Sumar Frejat1

  • 1Hypertrophic Cardiomyopathy Program, Leon H. Charney Division of Cardiology NYU Langone Health New York NY USA.

Insights

Mavacamten effectively treats obstructive hypertrophic cardiomyopathy (oHCM) in patients resistant to prior therapies. This study shows mavacamten improves symptoms and reduces left ventricular outflow obstruction in this challenging patient group.

Area of Science:

  • Cardiology
  • Pharmacology
  • Medical Therapeutics

Background:

  • The clinical utility of mavacamten in obstructive hypertrophic cardiomyopathy (oHCM) patients with prior advanced therapy is not well-established.
  • oHCM is a condition characterized by left ventricular outflow obstruction (LVOTO).

Purpose of the Study:

  • To evaluate the efficacy and safety of mavacamten in patients with oHCM and LVOTO who have previously undergone advanced treatments.
  • To assess clinical and echocardiographic outcomes in patients treated with mavacamten.

Main Methods:

  • Retrospective analysis of 115 patients with oHCM treated with mavacamten for at least 8 weeks.
  • Patients were categorized based on prior treatment: disopyramide, septal myectomy, alcohol septal ablation, dual-chamber pacing, or post-aortic valve replacement LVOTO.

Main Results:

  • Mavacamten treatment led to significant improvement in New York Heart Association class (P<0.0001).
  • Maximal LVOTO gradients decreased substantially in both previously treated and untreated groups (from ~100 mmHg to ~12 mmHg).
  • Left ventricular systolic dysfunction occurred in 10% of patients, with no significant difference between groups.

Conclusions:

  • Mavacamten demonstrates safety and efficacy for symptomatic LVOTO in oHCM patients refractory to advanced therapies.
  • It is effective in patients who develop LVOTO after aortic valve replacement.
Abstract

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