First-in-class drug brightens outlook for those diagnosed with hypertrophic cardiomyopathy in childhood
A landmark study led by Dr. Andrew Papez and the SCOUT study team examining mavacamten in adolescents with obstructive hypertrophic cardiomyopathy has been published in the New England Journal of Medicine, one of the world’s most prestigious medical journals.
This double-blind, randomized, placebo-controlled trial evaluated mavacamten in 44 adolescents with hypertrophic cardiomyopathy and severe left ventricular outflow tract (LVOT) obstruction, with a mean Valsalva gradient of 80 mmHg. Patients treated with mavacamten experienced a mean 48 mmHg reduction in LVOT gradient, compared with a 0.5 mmHg reduction in the placebo group (p < 0.0001). Significant improvements were also observed in resting and postexercise LVOT gradients (p < 0.0001).
In addition to hemodynamic benefits, mavacamten treatment was associated with improvements in maximal left ventricular wall thickness, average E/e’, and New York Heart Association (NYHA) functional class, reflecting meaningful symptom relief for patients.
The study was selected as a late-breaking clinical trial at the American College of Cardiology Scientific Sessions, underscoring its clinical significance and impact on the field.
Notably, Phoenix Children’s led all participating sites in patient recruitment, enrolling twice as many patients as the next highest enrolling center. This achievement highlights the exceptional efforts of the study team and Dr. Papez’s unwavering commitment to advancing care for children and adolescents with hypertrophic cardiomyopathy.
With anticipated pediatric FDA labeling for mavacamten, the impact of this work will extend well beyond the study—offering new hope and improved outcomes for future patients and families.
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