{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,22]],"date-time":"2026-10-22T19:25:02Z","timestamp":1792697102781,"version":"4.5.0"},"reference-count":40,"publisher":"American Society of Hematology","issue":"23","content-domain":{"domain":["ashpublications.org"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2025,12,9]]},"abstract":"<jats:sec>\n                    <jats:title>Abstract<\/jats:title>\n                    <jats:p>Chronic anemia due to non\u2013transfusion-dependent \u03b2-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 (\u223c2.2 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 \u22651.0 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 &amp;gt;1 risk factor. These results show that luspatercept led to a sustained increase in hemoglobin levels in patients with NTDT for up to \u223c4.6 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.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1182\/bloodadvances.2025016554","type":"journal-article","created":{"date-parts":[[2025,8,28]],"date-time":"2025-08-28T17:20:16Z","timestamp":1756401616000},"page":"6108-6119","update-policy":"https:\/\/doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":13,"title":["Efficacy and safety of luspatercept in non\u2013transfusion-dependent \u03b2-thalassemia: long-term results from the BEYOND study"],"prefix":"10.1182","volume":"9","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-8515-2238","authenticated-orcid":false,"given":"Ali T.","family":"Taher","sequence":"first","affiliation":[{"name":"1Department of Internal Medicine, American University of Beirut Medical Center, Beirut, Lebanon"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3162-1849","authenticated-orcid":false,"given":"Vip","family":"Viprakasit","sequence":"additional","affiliation":[{"name":"2Department of Pediatrics and Thalassemia Center, Faculty of Medicine, Siriraj Research Hospital, Mahidol University, Bangkok, Thailand"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5178-0655","authenticated-orcid":false,"given":"Antonis","family":"Kattamis","sequence":"additional","affiliation":[{"name":"3First Department of Pediatrics, National and Kapodistrian University of Athens, Athens, Greece"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6149-6845","authenticated-orcid":false,"given":"Silverio","family":"Perrotta","sequence":"additional","affiliation":[{"name":"4Dipartimento della Donna, del Bambino e di Chirurgia Generale e Specialistica, Universit\u00e0 della Campania, Luigi Vanvitelli, Caserta, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7361-3308","authenticated-orcid":false,"given":"Paolo","family":"Ricchi","sequence":"additional","affiliation":[{"name":"5Unit\u00e0 Operativa Semplice Dipartimentale, Malattie Rare del Globulo Rosso, Azienda Ospedaliera di Rilievo Nazionale A. Cardarelli, Naples, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John B.","family":"Porter","sequence":"additional","affiliation":[{"name":"6Department of Haematology, University College London, University College London Hospitals, London, United Kingdom"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9878-6029","authenticated-orcid":false,"given":"Thomas D.","family":"Coates","sequence":"additional","affiliation":[{"name":"7Hematology Section, Cancer and Blood Disease Institute, Children\u2019s Hospital Los Angeles, Los Angeles, CA"},{"name":"8Department of Pediatrics, University of Southern California Keck School of Medicine, Los Angeles, CA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gian Luca","family":"Forni","sequence":"additional","affiliation":[{"name":"9Centro della Microcitemia e Anemie Congenite e del Dismetabolismo del Ferro, Ospedale Galliera, Genoa, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3935-903X","authenticated-orcid":false,"given":"Khaled M.","family":"Musallam","sequence":"additional","affiliation":[{"name":"10Center for Research on Rare Blood Disorders and Thalassemia & Sickle Cell Center, Burjeel Medical City, Abu Dhabi, United Arab Emirates"},{"name":"11Department of Public Health and Epidemiology, Khalifa University, Abu Dhabi, United Arab Emirates"},{"name":"12Division of Hematology\/Oncology, Department of Pediatrics, Weill Cornell Medicine, New York, NY"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Oriana","family":"Esposito","sequence":"additional","affiliation":[{"name":"13Celgene International S\u00e0rl, a Bristol-Myers Squibb Company, Boudry, Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Richard","family":"Pilot","sequence":"additional","affiliation":[{"name":"14Bristol Myers Squibb, Princeton, 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