Birtamimab plus standard of care in light-chain amyloidosis: the phase 3 randomized placebo-controlled VITAL trial
Author(s): Morie A. Gertz1; Adam D. Cohen2; Raymond L. Comenzo3; Efstathios Kastritis4; Heather J. Landau5; Edward N. Libby6,7; Michaela Liedtke8; Vaishali Sanchorawala9; Stefan Schönland10; Ashutosh Wechalekar11; Jeffrey A. Zonder12; Giovanni Palladini13,14; Jackie Walling15; Spencer Guthrie15; Christie Nie15; Carol Karp15; Yuying Jin15; Gene G. Kinney15; Giampaolo Merlini13,14
Source: Blood (2023) 142 (14): 1208–1218
Dr. Maen Hussein's Thoughts
Birtamimab is an investigational humanized monoclonal antibody designed to neutralize toxic LC aggregates and deplete insoluble organ-deposited amyloid via macrophage-induced phagocytosis. Per this study, it was effective in Mayo stage 4 amyloidosis.
It is a rare disease, but at least now it has a possible option.
KEY POINTS
The VITAL study of birtamimab in all stages of newly diagnosed AL amyloidosis was discontinued early per futility analysis. Birtamimab improved post hoc ACM in patients with Mayo stage IV AL amyloidosis with cardiac involvement, who are at high risk of early death.VISUAL ABSTRACT
