Compared with best supportive care (BSC) and placebo, BSC with luspatercept appears to reduce the need for transfusion while maintaining patient health-related quality of life (HRQoL) among patients with beta-thalassemia, according to research published in the European Journal of Haematology.

Red blood cell transfusions help to overcome short-term problems related to anemia in patients with beta-thalassemia, but may lead to morbidity in the long term, including iron overload.

Luspatercept — a first-in-class agent that enhances late-stage erythropoiesis — was previously approved by the US Food and Drug Administration for patients with beta-thalassemia requiring regular RBCTs based on data from the phase 3 BELIEVE study ( Identifier: NCT02604433). For this analysis of data from BELIEVE, researchers reported their HRQoL findings.

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Patients enrolled to BELIEVE 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.

Researchers assessed HRQoL at baseline and every 12 weeks using the 36-item Short Form Health Survey (SF-36) and the Transfusion-dependent Quality of Life questionnaire (TranQol).

Data from the SF-36 and TranQol evaluations were analyzed through week 48. By this point, mean scores on both the SF-36 and the TranQol had not shown a meaningful change from baseline.

More patients in the luspatercept who reached a clinical response — defined as at least a 50% reduction in RBCT burden over 24 weeks — had, however, an improvement in the SF-36 Physical Function section (27.1% vs 11.5% with placebo; P =.019).

“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. “Improvements in HRQoL, particularly Physical Functioning, may be observed in some patients with significant and durable reduction in transfusion burden and/or those who achieve transfusion independence.”

Disclosures: This research was supported by Celgene. Please see the original reference for a full list of disclosures.


Cappellini MD, Taher AT, Piga A, et al. Health-related quality of life in patients with β-thalassemia: Data from the phase 3 BELIEVE trial of luspatercept. Eur J Haematol. Published online April 24, 2023. doi:10.1111/ejh.13975