ATTRibute-CM
Efficacy and Safety of Acoramidis (AG10) in Subjects With Transthyretin Amyloid Cardiomyopathy
Doente / População Intervenção / Exposição Comparação Desfecho
In 632 patients with transthyretin amyloid cardiomyopathy, acoramidis improved a hierarchical composite of death, CV hospitalisation, NT-proBNP and 6-minute walk distance compared with placebo.
N
632
Desenho
Double-blind placebo-controlled RCT, phase 3
Desfecho
Hierarchical composite (all-cause death, CV hospitalisation, NT-proBNP, 6-minute walk) at 30 months
Relevância
3
ResultadoAcoramidis superior on the hierarchical primary endpoint (win ratio 1.8, 95% CI 1.4-2.2; P<0.001), driven largely by mortality and CV hospitalisation.
Gillmore JD, et al. N Engl J Med. 2024;390(2):132-142. 10.1056/NEJMoa2305434
Discussão e crítica
Win-ratio composite harder to interpret clinically than event rates, and high background tafamidis use complicates comparison. Expanded disease-modifying options for ATTR-CM as a second-generation TTR stabiliser.