Vutrisiran demonstrated consistent efficacy in both women and men with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), with no statistically significant differences in treatment effect between sexes, according to a study published in the European Journal of Heart Failure.
Women have historically been underrepresented in ATTR-CM trials, leaving uncertainty about potential sex differences in treatment response.
Among the 654 participants in the HELIOS-B trial (NCT04153149)—comprising 49 women and 605 men—variant ATTR-CM was significantly more common in women (42.9% vs 9.1%). Women had smaller ventricular volumes and lower left ventricular mass, but body-surface-area–indexed wall thickness was higher. They also had better systolic measurements while reporting greater functional limitations and poorer health status, including a Kansas City Cardiomyopathy Questionnaire Overall Summary Score of 66.3 compared with 73.1 in men.