Clinical efficacy of luspatercept in patients with β-thalassemia in China
Objective:To summarize the clinical efficacy and safety of luspatercept,the first erythroid matura-tion agent,in Chinese β-thalassemia patients in real-world study.Methods:A retrospective analysis was carried out in patients diagnosed with β-thalassemia from May 2022 to January 2023.Luspatercept was prescribed accord-ing to drug instruction.The efficacy and safety data of patients were collected.Results:A total of 6 patients withβ-thalassemia were included.The hemoglobin level increased in two non-transfusion dependent thalassemia pa-tients by 1.8 g/dL and 2.3 g/dL,respectively.Two among the four transfusion dependent thalassemia patients had decreased need for blood transfusion by 40.0%and 80.7%,respectively.The hemoglobin level before trans-fusion were increased to 10.1 g/dL and 10.3 g/dL.Conclusion:Luspatercept can improve the hemoglobin level in non-transfusion dependent β-thalassaemia patients,and reduce the need for blood transfusion in transfusion de-pendent patients,and has a good efficacy in Chinese population.More accumulated data is needed to improve the prognosis of Chinese patients with β-thalassemia.