Efficacy and safety of luspatercept in non–transfusion-dependent β-thalassemia: long-term results from the BEYOND study
1
Issued Date
2025-12-09
Resource Type
ISSN
24739529
eISSN
24739537
Scopus ID
2-s2.0-105023308842
Pubmed ID
40875596
Journal Title
Blood Advances
Volume
9
Issue
23
Start Page
6108
End Page
6119
Rights Holder(s)
SCOPUS
Bibliographic Citation
Blood Advances Vol.9 No.23 (2025) , 6108-6119
Suggested Citation
Taher A.T., Viprakasit V., Kattamis A., Perrotta S., Ricchi P., Porter J.B., Coates T.D., Forni G.L., Musallam K.M., Esposito O., Pilot R., Kuo W.L., Lai Y., Reverte M., Wei R., Bueno L.M., Cappellini M.D. Efficacy and safety of luspatercept in non–transfusion-dependent β-thalassemia: long-term results from the BEYOND study. Blood Advances Vol.9 No.23 (2025) , 6108-6119. 6119. doi:10.1182/bloodadvances.2025016554 Retrieved from: https://repository.li.mahidol.ac.th/handle/123456789/113401
Title
Efficacy and safety of luspatercept in non–transfusion-dependent β-thalassemia: long-term results from the BEYOND study
Author's Affiliation
Weill Cornell Medicine
National and Kapodistrian University of Athens
Keck School of Medicine of USC
Mahidol University
Università degli Studi della Campania Luigi Vanvitelli
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
American University of Beirut
Khalifa University of Science and Technology
Children's Hospital Los Angeles
Bristol Myers Squibb
UCL Cancer Institute
Azienda Ospedaliera Di Rilievo Nazionale Antonio Cardarelli
E.O. Ospedali Galliera
Burjeel Medical City
National and Kapodistrian University of Athens
Keck School of Medicine of USC
Mahidol University
Università degli Studi della Campania Luigi Vanvitelli
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
American University of Beirut
Khalifa University of Science and Technology
Children's Hospital Los Angeles
Bristol Myers Squibb
UCL Cancer Institute
Azienda Ospedaliera Di Rilievo Nazionale Antonio Cardarelli
E.O. Ospedali Galliera
Burjeel Medical City
Corresponding Author(s)
Other Contributor(s)
Abstract
Chronic anemia due to non–transfusion-dependent β-thalassemia (NTDT) can result in clinical morbidities, particularly with inadequate management. Luspatercept was previously shown to improve hemoglobin levels in patients with NTDT in the phase 2, randomized, double-blind, placebo-controlled BEYOND trial (ClinicalTrials.gov identifier: NCT03342404). Here, we report long-term efficacy and safety results from the final analysis of BEYOND spanning an additional 26 months (∼2.2 years) of follow-up. Median treatment duration was 202.8 weeks for luspatercept and 61.1 weeks for placebo. Overall, 94.8% and 22.4% of patients in the luspatercept and placebo arms, respectively, achieved a mean hemoglobin increase from baseline ≥1.0 g/dL during any 12-week interval, with mean durations of response of 1136.0 and 203.3 days, respectively. Patient-reported tiredness and weakness showed sustained improvement with luspatercept treatment. The most common treatment-emergent adverse events in the luspatercept group were headache (45.8% vs 20.4% with placebo), bone pain (43.8% vs 6.1%), back pain (39.6% vs 12.2%), and arthralgia (38.5% vs 16.3%). Treatment-emergent extramedullary hematopoiesis events were reported in 12 (9.0%) and 2 (4.1%) patients receiving luspatercept and placebo, respectively, although differences in treatment exposures prevented informative comparisons. Of the 4 patients receiving luspatercept who reported thromboembolic events, all had >1 risk factor. These results show that luspatercept led to a sustained increase in hemoglobin levels in patients with NTDT for up to ∼4.6 years of treatment, with a consistent safety profile and no new safety findings. Luspatercept is a valuable treatment option for patients with NTDT, addressing the need for effective long-term treatment of anemia. This trial was registered at www.clinicaltrials.gov as #NCT03342404.