Reduced Need for Red Blood Cell Transfusions in Patients Treated With Luspatercept for Beta-Thalassemia
The drug’s ability to boost late-stage erythropoiesis makes it a potential treatment option for patients with beta-thalassemia.
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Luspatercept may reduce the need for red blood cell (RBC) transfusions in patients with beta-thalassemia, according to an abstract presented at the 60th American Society of Hematology (ASH) Annual Meeting in San Diego, California.1
Luspatercept has been investigated as a possible treatment for beta-thalassemia due to its ability to boost late-stage erythropoiesis. In a phase 3 clinical trial (ClinicalTrials.gov Identifier: NCT02604433), its efficacy and safety were assessed in adult patients with beta-thalassemia or hemoglobin E/beta-thalassemia who required frequent blood transfusions.
Patients were given either luspatercept (224 patients) or placebo (112 patients) triweekly for at least 48 weeks. Luspatercept was started at a dosage of 1.0 mg/kg and raised to 1.25 mg/kg. All patients continued to undergo RBC transfusions and iron chelation therapy as needed to uphold a baseline hemoglobin level.
The primary study endpoint was a 33% or greater reduction in transfusions in weeks 13 to 24 compared with the baseline transfusion burden during the first 12 weeks. Other comparisons over time were also examined.
The primary endpoint was met by 21.4% of patients receiving luspatercept and 4.5% of patients given placebo (odds ratio 5.79, P <.0001). Patients in the luspatercept arm also showed significant reductions in RBC transfusions compared with the placebo arm regarding other comparisons over time.
Overall, for any consecutive 12-week period, 70.5% of patients in the luspatercept arm showed at least a 33% reduction in transfusions, while 29.5% of patients in the placebo arm showed the same (P <.0001).
No fatalities were reported among patients receiving luspatercept in this study, and, according to the authors, the occurrence of adverse events was comparable to reported data from the prior phase 2 analysis.2
The researchers concluded that luspatercept significantly reduced the need for RBC transfusions and was well-tolerated.
Disclosures: Multiple authors declare affiliations with the pharmaceutical industry. For a full list of disclosures, please refer to the original study.
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1. Cappellini MD, Viprakasit V, Taher A, et al. The Believe Trial: results of a phase 3, randomized, double-blind, placebo-controlled study of luspatercept in adult beta-thalassemia patients who require regular red blood cell (RBC) transfusions. Oral presentation at: 60th ASH Annual Meeting and Exposition; December 1-4, 2018; San Diego, CA.
2. Piga AG, Tartaglione I, Gamberini R, et al. Luspatercept increases hemoglobin, decreases transfusion burden and improves iron overload in adults with beta-thalassemia. Oral presentation at: 58th Annual Meeting and Exposition; December 9-12, 2017; Atlanta, GA.