Diane McDowell, MD
Results from the pivotal phase III BELIEVE trial, which supported the FDA approval of luspatercept-aamt (Reblozyl) for the treatment of anemia in adult patients with beta thalassemia who require regular red blood cell (RBC) transfusions, have been published in the New England Journal of Medicine
The most common grade ≥3 AEs occurring with luspatercept were anemia (3.1% vs 0%), increased liver iron concentration (2.7% vs 0.9%) and hyperuricemia (2.7% vs 0%). Serious AEs occurred in 15.2% versus 5.5% of the luspatercept versus placebo arms, respectively. There was 1 death due to an unconfirmed case of acute myeloid leukemia.
Cappellini MD, Viprakasit V, Taher AT, et al. A phase 3 trial of luspatercept in patients with transfusion-dependent β-thalassemia. N Engl J Med. 2020;382(13):1219-1231. doi: 10.1056/NEJMoa1910182.
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