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.
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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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