The following article features coverage from the American Society of Hematology 2021 meeting. Click here to read more of Hematology Advisor‘s conference coverage.

For patients with nontransfusion-dependent b-thalassemia (NTDT), results of a phase 2 trial suggest that luspatercept may be associated with improved quality of life (QOL) and reduced red blood cell (RBC) transfusion burden. The study results were presented in a poster at the 2021 American Society of Hematology (ASH) Annual Meeting by Antonis Kattamis, MD, PhD, of the University of Athens in Greece, and colleagues.

The BEYOND trial ( Identifier: NCT03342404) evaluated the use of luspatercept in patients with NTDT. In a double-blind, placebo-controlled phase of the study, patients were randomized 2:1 to either a luspatercept arm or a placebo arm, and they were stratified according to baseline hemoglobin levels and NTDT Patient-Reported Outcomes (NTDT-PRO) Tiredness/Weakness (T/W) domain scores at baseline. The placebo-controlled phase for each patient lasted a minimum of 48 weeks. The presented analysis focused on QOL outcomes and RBC transfusion burden during this phase of the study, with QOL investigated through multiple instruments.

A total of 96 patients were in the luspatercept arm and 49 patients were in the placebo arm. At baseline, QOL scores reportedly were comparable across treatment arms. Differences in QOL between arms appeared to show a tendency to widen over time. During weeks 37 through 48, least-squares mean (LSM) changes in scores in the NTDT-PRO T/W domain were -0.78 with luspatercept and 0.01 with the placebo (P =.0510). LSM changes in scores in the NTDT-PRO shortness-of-breath domain were -0.59 with luspatercept and 0.47 with the placebo during the same period (P =.0047). Decreases in scores with these NTDT-PRO instruments indicate improvement.

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With the 36-Item Short Form Health Survey (SF-36) version 2 Mental Component Summary, the LSM difference at week 48 was 2.70 in favor of luspatercept (P =.0469). On the Patient Global Impression of Severity instrument, over weeks 1 through 48, patients in the luspatercept group also showed significant improvements relative to the placebo group. Patients in the luspatercept arm more often indicated greater overall improvements in symptoms using the Patient Global Impression of Change scale. They also tended to demonstrate trends toward improvements over time with other QOL instruments.

Overall, 82.3% of patients in the luspatercept arm were free of RBC transfusions from weeks 1 through 48, compared with 44.9% in the placebo arm (P <.0001).

“In conclusion, luspatercept treatment improved quality of life and RBC transfusion burden compared with placebo in patients with NTDT in the BEYOND trial,” Dr Kattamis said in a presentation accompanying his research team’s poster.

Disclosures: Some authors have declared affiliations with or received grant support from the pharmaceutical industry. Please refer to the original study for a full list of disclosures.

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Kattamis A, Viprakasit V, Cappellini MD, et al. Luspatercept improves quality of life and reduces red blood cell transfusion burden in patients with non-transfusion-dependent β-thalassemia in the BEYOND trial. Presented at ASH 2021; December 11-14, 2021. Abstract 3081.