Long-Term Efficacy and Safety of Mavacamten in Chinese Patients With Obstructive Hypertrophic Cardiomyopathy: Week 78 Results From the EXPLORER-CN Study
Abstract
Background: Long-term efficacy and safety of mavacamten in Chinese patients with obstructive hypertrophic cardiomyopathy are unknown.
Methods: Patients who completed the 30-week, double-blind, placebo-controlled treatment period in EXPLORER-CN (A Study to Evaluate the Efficacy and Safety of Mavacamten in Chinese Adults With Symptomatic Obstructive HCM), with no active safety concerns, were eligible to enter the long-term extension period to receive 48-week mavacamten treatment. Patients previously on mavacamten continued mavacamten (dose at week 30; mavacamten-mavacamten group); patients previously on placebo received mavacamten (starting dose, 2.5 mg once daily; placebo-mavacamten group). Key efficacy end points included change from baseline in echocardiographic measures, New York Heart Association functional class, 23-item Kansas City Cardiomyopathy Questionnaire Clinical Summary Score, and cardiac biomarkers through week 78. Analyses were descriptive; no between-group hypothesis testing was performed.
Results: Seventy-nine patients (mean age, 51.6 years; 27.8% women) entered the long-term extension period (mavacamten-mavacamten, n=54; placebo-mavacamten, n=25). In the mavacamten-mavacamten group, numerical improvements in Valsalva and resting left ventricular outflow tract peak gradients were maintained through week 78 (mean change from baseline, -73.0 mm Hg [95% CI, -86.4 to -59.5]; and -56.3 mm Hg [95% CI, -67.1 to -45.5], respectively). Numerical improvements were also…
How strong is the evidence?
Evidence Hub reads this as a randomized trial, done in people. Its evidence level is 2 of 5 (High · randomized trial). Randomized trials of a reasonable size are the strongest single-study design. Reported size: 54 participants or records.
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