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Impact of Mavacamten on Disease-Related Symptoms in Patients With Obstructive Hypertrophic Cardiomyopathy: HCMSQ Outcomes in EXPLORER-HCM
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DOI：10.1016/j.cardfail.2026.03.036.png)
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Abstract
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• This study is the first comprehensive report of symptom burden as measured by the Hypertrophic Cardiomyopathy Symptom Questionnaire (HCMSQ), a validated disease-specific patient-reported outcome instrument, among patients with obstructive hypertrophic cardiomyopathy (HCM) who were treated with mavacamten in a phase 3 clinical trial (EXPLORER-HCM). • Treatment with mavacamten was associated with improvements versus placebo from baseline to week 30 across all HCMSQ domain scores (shortness of breath [SoB], tiredness, and cardiovascular symptoms) and the total score. • Both baseline HCMSQ SoB domain scores and improvements at week 30 were associated with improvements in left ventricular outflow tract obstruction and some measures of diastolic function.
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