Health-related Quality of Life in Patients with β-Thalassemia: Data from the Phase 3 BELIEVE Trial of Luspatercept

The effects of anemia and fatigue, together with the effects of treatment regimens (i.e., RBCTs and iron chelation therapy) can have a considerable impact on the health-related quality of life (HRQoL) of patients with β-thalassemia. Adults with TD β-thalassemia have been found to have worse physical, emotional, and social functioning HRQoL scores compared with the general population.
Patients report a high burden from their transfusion regimen related to lack of blood availability, fear of adverse events, and negative impact on their employment.
Increases in life expectancy observed over the past few decades have highlighted the need for improvements in HRQoL as working-age adults face particular challenges related to employment, higher education, and mental health issues. As such, there is a need to reduce RBCT burden while maintaining or improving HRQoL for these patients.
Luspatercept – marketed under the name ‘Reblozyl – is a first-in-class erythroid maturation agent that enhances late-stage erythropoiesis. Luspatercept has been approved by the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) for the treatment of anemia due to β-thalassemia in adult patients who require regular RBCTs.
Data from BELIEVE study, published in the European Journal of Haematology, showed the effectiveness of luspatercept in transfusion-dependent thalassemia (TDT) patients seem to underline the benefits of the addition of luspatercept in the best supportive care (BSC) of TDT patients.
It has been shown that adding luspatercept in the BSC not only has reduced the transfusion’s burden but also kept at the same levels several parameters related to HRQoL. It has to be underlined that in the group of patients who received luspatercept as adjunctive treatment to BSC, those who responded to luspatercept had better profile among all indicators’ measurements.
Patients enrolled to the BELIEVE phase 3 study were randomly assigned to receive BCS plus placebo (104 patients) or BSC plus luspatercept (212 patients). In the luspatercept and placebo groups, the mean ages were 32.1 and 31.6 years, respectively, 41% and 45.2% of patients were male sex, and 49.5% and 50% of patients were receiving more than 6 transfusions every 12-week period.
More patients in the luspatercept who reached a clinical response — defined as at least a 50% reduction in RBCT burden over 24 weeks.
“The addition of luspatercept to BSC reduced transfusion burden while maintaining patients’ HRQoL over time throughout the study period,” the authors wrote in their report.
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