Health-related quality of life in patients with β-thalassemia: Data from the phase 3 BELIEVE trial of luspatercept
1
Issued Date
2023-01-01
Resource Type
ISSN
09024441
eISSN
16000609
Scopus ID
2-s2.0-85153617192
Journal Title
European Journal of Haematology
Rights Holder(s)
SCOPUS
Bibliographic Citation
European Journal of Haematology (2023)
Suggested Citation
Cappellini M.D., Taher A.T., Piga A., Shah F., Voskaridou E., Viprakasit V., Porter J.B., Hermine O., Neufeld E.J., Thompson A.A., Tang D., Yucel A., Lord-Bessen J., Yu P., Guo S., Shetty J.K., Miteva D., Zinger T., Backstrom J.T., Oliva E.N. Health-related quality of life in patients with β-thalassemia: Data from the phase 3 BELIEVE trial of luspatercept. European Journal of Haematology (2023). doi:10.1111/ejh.13975 Retrieved from: https://repository.li.mahidol.ac.th/handle/123456789/82500
Title
Health-related quality of life in patients with β-thalassemia: Data from the phase 3 BELIEVE trial of luspatercept
Author's Affiliation
Siriraj Hospital
Whittington Health NHS Trust
Université Paris Cité
Evidera, USA
American University of Beirut Medical Center
Università degli Studi di Milano
Hôpital Necker Enfants Malades
University College London Hospitals NHS Foundation Trust
Laikon General Hospital
St. Jude Children's Research Hospital
Ann & Robert H. Lurie Children's Hospital of Chicago
Università degli Studi di Torino
Merck & Co., Inc.
Bristol-Myers Squibb
Grande Ospedale Metropolitano Bianchi-Melacrino-Morelli
Whittington Health NHS Trust
Université Paris Cité
Evidera, USA
American University of Beirut Medical Center
Università degli Studi di Milano
Hôpital Necker Enfants Malades
University College London Hospitals NHS Foundation Trust
Laikon General Hospital
St. Jude Children's Research Hospital
Ann & Robert H. Lurie Children's Hospital of Chicago
Università degli Studi di Torino
Merck & Co., Inc.
Bristol-Myers Squibb
Grande Ospedale Metropolitano Bianchi-Melacrino-Morelli
Other Contributor(s)
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.