European Hematology Association - Above and BEYOND: Luspatercept is Efficacious and Well-Tolerated in Patients with Non-Transfusion-Dependent β-Thalassemia

THE HAGUE, Netherlands, June 11, 2021 /PRNewswire/ -- The non-transfusion-dependent (NTD) form of β-thalassemia, an inherited blood disorder with impaired production of hemoglobin, is characterized by chronic anemia of mild-moderate severity and iron overload. Despite their transfusion-independence, patients with NTD β-thalassemia can still develop serious morbidities in various organ systems and have reduced quality of life. There are currently no approved therapies for the management of anemia in NTD β-thalassemia. Luspatercept is a drug that is approved for the treatment of anemia in transfusion-dependent β-thalassemia and may also be beneficial for patients with NTD β-thalassemia. The BEYOND study enrolled 145 adults with NTD β-thalassemia in a randomized phase 2 clinical trial and compared the efficacy and safety of luspatercept with placebo. All patients continued to receive best supportive care throughout the study and were followed up to Week 24.

Treatment with luspatercept significantly improved anemia compared with placebo. Patients showed increased hemoglobin and almost 90% of luspatercept-treated patients remained transfusion-free through Week 24. Consequently, patients in the intervention arm reported a higher quality of life score, indicating a reduction in clinical symptoms such as tiredness and weakness. The improvement in quality of life significantly correlated with hemoglobin increase. Importantly, the incidence of treatment-emergent adverse events was comparable between the luspatercept and placebo groups and no thromboembolic or thrombophlebitis events occurred in the trial. In conclusion, luspatercept is efficacious and safe for treating patients with NTD β-thalassemia.

The results of this study will be presented by Professor Ali Taher in the Presidential Symposium.

Presenter:          Professor Ali T. Taher

Affiliation:          American University of Beirut Medical Center,  Beirut, Lebanon 

Abstract:              #S101  THE BEYOND STUDY: RESULTS OF A PHASE 2, DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED MULTICENTER STUDY OF LUSPATERCEPT IN ADULT PATIENTS WITH NON-TRANSFUSION DEPENDENT BETA-THALASSEMIA

About the EHA Annual Congress: Every June, EHA organizes its Annual Congress in a major European city. This year, due to the persisting COVID19 pandemic, EHA organizes a virtual Congress for the second time. The Congress is aimed at health professionals working in or interested in the field of hematology.

Website: www.ehaweb.org

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