Home > Haematology > EHA 2021 > Haemoglobinopathies > Luspatercept improved anaemia in patients with non-transfusion-dependent β-thalassaemia

Luspatercept improved anaemia in patients with non-transfusion-dependent β-thalassaemia

Presented By
Prof. Ali Taher, American University of Beirut Medical Center, Lebanon
Conference
EHA 2021
Trial
Phase 2, BEYOND
Results from the double-blind, multicentre, phase 2 BEYOND study demonstrated that luspatercept is safe and efficacious in patients with non-transfusion-dependent β-thalassaemia (NTDT). A durable increase in haemoglobin levels was achieved, which was associated with increased quality of life scores. β-thalassemia is an inherited haemoglobinopathy characterised by impaired haemoglobin (Hb) production, chronic anaemia, and iron overload that affects survival and quality of life. Tailored red blood cell transfusions and novel therapies target key pathophysiologic mechanisms in transfusion-dependent β-thalassaemia (TDT) and NTDT. Patients with NTDT do not require lifelong regular transfusion for survival; however, they may require occasional transfusions during pregnancy, surgery, or infection [1]. The Hb level significantly correlates with morbidity-free survival in NTDT: an Hb increase of <8 g/dL to >10 g/dL in steps of 1 g/dL dramatically decr...


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