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The Following is a summary of “Long-Term effectiveness and safety of Luspatercept for the Treatment of Anaemia in Patients with Transfusion-Pedentent β-Thalassamia (Believe): Final Results From a Phase 3 Randomized Trial,” Published in the March 2025 Issue of Lancet Haematology by Cappellini et al.
Treatment Options for ReduCing Red Blood Cell (RBC) Transfusion Burden in Transfusion-Dependent β-Thalassaemia Are Limited.
RESEARCHERS DRIVED A RETROSPTIVE STUDY ON LONG-TERM DATE FROM THE PHASE 3 BELIEVE TRIAL OF LUSPATERCEPT IN TRANSFUSION-RELEASE β-THalassamia.
They conducted to Phase 3, Randomized, Double-Blind, Placebo-Controlled Study at 65 Sites in 15 Countries. They included adults with transfusion-dependent β-thalassamia or hemoglobin and/β-thalassamia and an eastern cooperative oncology group score of 0–1. PATIES WERE RANDOMLY ASSIGNED (2: 1) TO LUNPATERCEPT (1 · 0–1 · 25 mg/kg) OR PLACEBO EVERY 21 DAYS. After Unblinding, Patients Cour Receive Luspatercept in the Open-Label Extension Phase. They Analyzed the Updated Primary Endpoint and Reported Long-Term Effficy (Intenti-to-Treat) and Safety (Safety Population) Over ~ 3 years.
The Results Showed That 336 Patients Were Randomized to Luspatercept (n = 224) or placebo (n = 112), with a median age of 30 years (IQR 23–40); 195 (58%) Were Female and 141 (42%) evils. AS OF JAN 5, 2021, THE MEDIAN TREATMENT DURATION FOR LUSPATERCEPT WAS 153.6 WEEKS (IQR 81.0–171.0), AND FOLLOW-UP WAS 163.1 Weeks (140.5–176.2). No Comparative Analyses Were Performed after Week 96. Luspatercept Reduced RBC Transfusion Burden, with Mean Decrases of 6.2 RBC Units (SD 5.7) at Weeks 97–144 and 6.4 (4.3) at Weeks 145–192. A 33%or Greater Reduction Occred in 173 (77%) PATIES OVER ANY 12-MEEK INTERVAL AND 116 (52%) OVER ANY 24-SEEK INTERVAL. The Median Duration of this Response Was 586.0 Days (IQR 264.0–1010.0). Among 315 Luspatercept-Treated Patients (Including 92 Who Crossed Over), 3 or Worse Treatment-Emergent Adverse Events (Teaes) Anaemia (9 (3%)), Increaded Liver Iron (7 (2%)), and Bone Pain (7 (2%)); Serious Teaes Occrede in 71 (23%) Patients. In Treatment-Relanted Deaths Occred.
Investigators Affrmed Luspatercept’s long-term effficacy in reducing transfusion burden in transfusion-dependent β-thalassemia with a Manageable Safety Profile.
Source: Thelancet.com/journals/lanhae/article/piis2352-3026(24)00376-4/abstract
