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

Cappellini, MD; Taher, AT; Piga, A; Shah, F; Voskaridou, E; Viprakasit, V; Porter, JB; ... Oliva, EN; + view all (2023) Health-related quality of life in patients with β-thalassemia: Data from the phase 3 BELIEVE trial of luspatercept. European Journal of Haematology , 111 (1) pp. 113-124. 10.1111/ejh.13975. Green open access

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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.

Type: Article
Title: Health-related quality of life in patients with β-thalassemia: Data from the phase 3 BELIEVE trial of luspatercept
Open access status: An open access version is available from UCL Discovery
DOI: 10.1111/ejh.13975
Publisher version: https://doi.org/10.1111/ejh.13975
Language: English
Additional information: © 2023 The Authors. European Journal of Haematology published by John Wiley & Sons Ltd. This is an open access article under the terms of the Creative Commons Attribution-NonCommercial-NoDerivs License (http://creativecommons.org/licenses/by-nc-nd/4.0/).
Keywords: beta-thalassemia, iron overload, quality of life
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute > Research Department of Haematology
URI: https://discovery.ucl.ac.uk/id/eprint/10169805
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