Health-related quality of life in patients with β-thalassemia: Data from the phase 3 BELIEVE trial of luspatercept

Background: Patients with transfusion-dependent (TD) ss-thalassemia require longterm red blood cell transfusions (RBCTs) that lead to iron overload, impacting healthrelated quality of life (HRQoL). Methods: The impact of luspatercept, a first- in-class erythroid maturation agent, versus placebo on HRQoL of patients with TD ss-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 nonresponders. 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.