Mavacamten for treatment of symptomatic obstructive hypertrophic cardiomyopathy (EXPLORER-HCM): a randomised, double-blind, placebo-controlled, phase 3 trial.

Olivotto, Iacopo; Oreziak, Artur; Barriales-Villa, Roberto; Abraham, Theodore P; Masri, Ahmad; Garcia-Pavia, Pablo; Saberi, Sara; Lakdawala, Neal NK; Wheeler, Matthew MT; Owens, Anjali; Kubanek, Milos; Wojakowski, Wojciech; Jensen, Morten K; Gimeno-Blanes, Juan; Afshar, Kia; Myers, Jonathan; Hegde, Sheila SM; Solomon, Scott D; Sehnert, Amy AJ; Zhang, David; Li, Wanying; Bhattacharya, Mondira; Edelberg, Jay JM; Waldman, Cynthia Burstein; Lester, Steven J; Wang, Andrew; Ho, Carolyn Y; Jacoby, Daniel; Bondue, Antoine

doi:doi/10.1016/S0140-6736(20)31792-X

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Article révisé par les pairs

Résumé :

Cardiac muscle hypercontractility is a key pathophysiological abnormality in hypertrophic cardiomyopathy, and a major determinant of dynamic left ventricular outflow tract (LVOT) obstruction. Available pharmacological options for hypertrophic cardiomyopathy are inadequate or poorly tolerated and are not disease-specific. We aimed to assess the efficacy and safety of mavacamten, a first-in-class cardiac myosin inhibitor, in symptomatic obstructive hypertrophic cardiomyopathy.

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