The first drug for β-thalassemia—luspatercept
ZHANG Shan-shan
XU Wen-feng
WANG Jing
XU Shuo
JIN Peng-fei
Abstract：Luspatercept is the world's first and currently only red blood cell maturation agent which increases hemoglobin levels by promoting advanced red blood cell maturation.It's the first FDA-approved drug for beta-thalassemia.In January 2022,China's NMPA approved it for the treatment of adult beta-thalassemia patients who require regular red blood cell infusion and red blood cell infusion ≤ 15 units/24 weeks.Clinical studies have shown that luspatercept can effectively reduce blood transfusion burden in patients with beta-thalassemia with controllable adverse reactions and good tolerance.The mechanism,pharmacokinetics,clinical evaluation and safety are reviewed in this article so as to provide reference for clinical medication.
Keywords：luspatercepterythrocyte ripening agentbeta thalassemia
Publication Date：2024-06-15
Online Publishing Date：2026-08-14(First online date of this platform, not the publication date of the document)
Pages：4( 1098-1101 )