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

Autor: Cappellini MD; Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, University of Milan, Milan, Italy., Taher AT; Department of Internal Medicine, American University of Beirut Medical Center, Beirut, Lebanon., Piga A; Department of Clinical and Biological Sciences, University of Turin, Turin, Italy., Shah F; Department of Haematology, Whittington Health NHS Trust, London, UK., Voskaridou E; Centre of Excellence in Rare Haematological Diseases-Haemoglobinopathies, Laiko General Hospital, Athens, Greece., Viprakasit V; Siriraj Hospital, Mahidol University, Bangkok, Thailand., Porter JB; University College London Hospitals NHS Foundation Trust, London, UK., Hermine O; Necker Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France.; Imagine Institute, INSERM Unité 1163, University of Paris, Paris, France., Neufeld EJ; St. Jude Children's Research Hospital, Memphis, Tennessee, USA., Thompson AA; Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, USA., Tang D; Bristol Myers Squibb, Princeton, New Jersey, USA., Yucel A; Bristol Myers Squibb, Princeton, New Jersey, USA., Lord-Bessen J; Bristol Myers Squibb, Princeton, New Jersey, USA., Yu P; Evidera, Waltham, Massachusetts, USA., Guo S; Evidera, Waltham, Massachusetts, USA., Shetty JK; Bristol Myers Squibb, Princeton, New Jersey, USA., Miteva D; Bristol Myers Squibb, Princeton, New Jersey, USA., Zinger T; Bristol Myers Squibb, Princeton, New Jersey, USA., Backstrom JT; Acceleron Pharma Inc., a Subsidiary of Merck & Co., Inc., Rahway, New Jersey, USA., Oliva EN; Grande Ospedale Metropolitano Bianchi Melacrino Morelli, Reggio Calabria, Italy.
Jazyk: angličtina
Zdroj: European journal of haematology [Eur J Haematol] 2023 Jul; Vol. 111 (1), pp. 113-124. Date of Electronic Publication: 2023 Apr 24.
DOI: 10.1111/ejh.13975
Abstrakt: 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.
(© 2023 The Authors. European Journal of Haematology published by John Wiley & Sons Ltd.)
Databáze: MEDLINE
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