Introduction: Some studies have indicated that the use of luspatercept may be a novel and efficient treatment for β-thalassemia patients. In this article, we aimed to review the current evidence related to luspatercept prescription and its clinical effectiveness in patients with β-thalassemia.
Methods: PubMed, Web of Science, Scopus, Trip and CENTRAL were searched up to June 2020. The inclusion criteria were English-language articles that studied the effects of luspatercept on improving anemia severity in patients with β-thalassemia in a clinical setting.
Results: The search strategy yielded 273 potentially relevant articles. After searching the databases, scanning of titles, abstracts and full texts for relevancy was performed to identify suitable articles. A total of 77 articles were confirmed for full text analysis. The estimated number of patients needed to treat (NNT) for luspatercept treatment, using data derived from conducted clinical trials, according to a reduction in transfusion need of ≥ 33% or ≥ 50 from baseline, during week 13-24/week 37-48/any 12- and 24-week intervals as outcomes, was 3-5 in patients with β-thalassemia.
Conclusion: Based on the conducted studies, the effectiveness of luspatercept on transfusion burden and hemoglobin levels was outstanding in β-thalassemia patients. Further large and well-designed clinical studies are needed to identify any unforeseen complications subsequent to use of luspatercept, particularly when used with other treatments with potentially serious adverse effects such as anti-osteoporotic and iron chelator agents.
Keywords: Luspatercept; Reblozyl; Red cell transfusion; β-Thalassemia.