VALOR-HCM
A Study to Evaluate Mavacamten in Adults With Symptomatic Obstructive HCM Eligible for Septal Reduction Therapy
Doente / População Intervenção / Exposição Comparação Desfecho
In 112 patients with obstructive HCM referred for septal reduction therapy, mavacamten reduced the need to proceed with or remain eligible for septal reduction therapy compared with placebo.
N
112
Desenho
Double-blind placebo-controlled RCT, phase 3
Desfecho
Composite at week 16: decision to proceed with septal reduction therapy, or remaining SRT-eligible
Relevância
3
Resultado17.9% with mavacamten vs 76.8% with placebo met the composite (difference 58.9%, 95% CI 44.0-73.9; P<0.001).
Desai MY, et al. J Am Coll Cardiol. 2022;80(2):95-108. 10.1016/j.jacc.2022.04.048
Discussão e crítica
Small, short-term surrogate endpoint (SRT eligibility/referral rather than actual procedures or hard outcomes). Demonstrated that mavacamten can defer or avoid invasive septal reduction in obstructive HCM.