玛伐凯泰治疗梗阻性肥厚型心肌病的疗效与安全性OA

Objective To evaluate the efficacy and safety of mavacamten in patients with obstructive hypertrophic cardiomyopathy(oHCM).Methods Patients with oHCM who received mavacamten treatment at Sun Yat-sen Memorial Hospital,Sun Yat-sen University,between October 2024 and October 2025 were retrospectively enrolled.All patients initiated treatment at a dose of 2.5 mg once daily and underwent follow-up assessments at weeks 4,8,12,16,and 24,with dose adjustments based on serial echocardiographic findings.Changes in resting and Valsalva-provoked left ventricu-lar outflow tract gradients(LVOTGs)from baseline were evaluated.Changes in New York Heart Association(NYHA)functional class,left ventricular ejection fraction(LVEF),maximal left ventricular wall thickness,and quality of life as-sessed using the 12-item Kansas City Cardiomyopathy Questionnaire(KCCQ-12)were also analyzed.Safety endpoints,including adverse events,were recorded throughout the follow-up period.Results After treatment,both resting and Valsalva-provoked LVOTGs decreased significantly from baseline.Resting LVOTG decreased from 57.3±39.8 mmHg at baseline to 17.3±15.0 mmHg at week 24(P＜0.01),while Valsalva-provoked LVOTG decreased from 66.2±33.5 mmHg to 32.9±23.7 mmHg(P＜0.05).Cardiac function and quality of life were also improved.By week 24,53 pa-tients(79.1％)had an improvement of at least one NYHA functional class,and no worsening of heart failure symptoms was observed during follow-up.The KCCQ-12 score was significantly higher at week 12 than at baseline(P＜0.001).LVEF remained generally stable throughout follow-up(P＞0.05).Maximal left ventricular wall thickness at each follow-up time point differed significantly from baseline(P＜0.05).During follow-up,2 patients died,3 were rehospitalized because of worsening heart failure,and 1 developed new-onset atrial fibrillation.Overall,mavacamten demonstrated a favorable safety profile.Conclusion Mavacamten demonstrated favorable efficacy and safety in Chinese patients with ob-structive hypertrophic cardiomyopathy,with significant reductions in resting and Valsalva-provoked LVOTGs and im-provements in functional status and quality of life.These findings support the potential value of mavacamten as a targeted therapeutic option for patients with refractory obstructive hypertrophic cardiomyopathy.