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

Abstract Background 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.