Cappellini M.D.Taher A.T.Piga A.Shah F.Voskaridou E.Viprakasit V.Porter J.B.Hermine O.Neufeld E.J.Thompson A.A.Tang D.Yucel A.Lord-Bessen J.Yu P.Guo S.Shetty J.K.Miteva D.Zinger T.Backstrom J.T.Oliva E.N.Mahidol University2023-05-192023-05-192023-01-01European Journal of Haematology (2023)09024441https://repository.li.mahidol.ac.th/handle/20.500.14594/82500Background: Patients with transfusion-dependent (TD) β-thalassemia require long-term red blood cell transfusions (RBCTs) that lead to iron overload, impacting health-related quality of life (HRQoL). Methods: The impact of luspatercept, a first-in-class erythroid maturation agent, versus placebo on HRQoL of patients with TD β-thalassemia was evaluated in the phase 3 BELIEVE trial. HRQoL was assessed at baseline and every 12 weeks using the 36-item Short Form Health Survey (SF-36) and Transfusion-dependent Quality of Life questionnaire (TranQol). Mean change in HRQoL was evaluated from baseline to week 48 for patients receiving luspatercept + best supportive care (BSC) and placebo + BSC and between luspatercept responders and non-responders. Results: Through week 48, for both groups, mean scores on SF-36 and TranQol domains were stable over time and did not have a clinically meaningful change. At week 48, more patients who achieved clinical response (≥50% reduction in RBCT burden over 24 weeks) in the luspatercept + BSC group had improvement in SF-36 Physical Function compared with placebo + BSC (27.1% vs. 11.5%; p =.019). Conclusions: Luspatercept + BSC reduced transfusion burden while maintaining patients' HRQoL. HRQoL domain improvements from baseline through 48 weeks were also enhanced for luspatercept responders.MedicineArticleSCOPUS10.1111/ejh.139752-s2.0-8515361719216000609