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

Alexis A Thompson, Jay T Backstrom, Tatiana Zinger, Dimana Miteva, Jeevan K Shetty, Shien Guo, Peiwen Yu, Jennifer Lord-Bessen, Aylin Yucel, Derek Tang, Ali T Taher, Olivier Hermine, John B Porter, Ersi Voskaridou, Farrukh Shah, Antonio Piga, Maria Domenica Cappellini, Vip Viprakasit, Esther Natalie Olíva, Ellis J Neufeld

Journal: European journal of haematology 2023;111(1):113-124

PMID: 37095595

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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.

Address: 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.

DOI: 10.1111/ejh.13975

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MeSH Terms: Humans, Activin Receptors, Type II, beta-Thalassemia, Immunoglobulin Fc Fragments, Quality of Life