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

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, University of Milan, Milan, Italy. Department of Internal Medicine, American University of Beirut Medical Center, Beirut, Lebanon. Department of Clinical and Biological Sciences, University of Turin, Turin, Italy. Department of Haematology, Whittington Health NHS Trust, London, UK. Centre of Excellence in Rare Haematological Diseases-Haemoglobinopathies, Laiko General Hospital, Athens, Greece. Siriraj Hospital, Mahidol University, Bangkok, Thailand. University College London Hospitals NHS Foundation Trust, London, UK. Necker Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France. Imagine Institute, INSERM Unité 1163, University of Paris, Paris, France. St. Jude Children's Research Hospital, Memphis, Tennessee, USA. Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, USA. Bristol Myers Squibb, Princeton, New Jersey, USA. Evidera, Waltham, Massachusetts, USA. Acceleron Pharma Inc., a Subsidiary of Merck & Co., Inc., Rahway, New Jersey, USA. Grande Ospedale Metropolitano Bianchi Melacrino Morelli, Reggio Calabria, Italy.

European journal of haematology. 2023
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.
Study details
Language : eng
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