Luspatercept: A Review in Transfusion-Dependent Anaemia due to Myelodysplastic Syndromes or β-Thalassaemia

<p><b>Declarations</b></p> <p><b>Funding</b> The preparation of this review was not supported by any external funding.</p> <p><b>Authorship and Conflict of interest</b><b><i> </i></b>Connie Kang and Yahiya Y. Syed are salaried employees of Adis International Ltd/Springer Nature, and declare no relevant conflicts of interest. All authors contributed to the review and are responsible for the article content.</p> <p> </p> <p><b>Ethics approval, Consent to participate, Consent to publish, Availability of data and material, Code availability </b>Not applicable</p><p><br></p><p><br></p><p>Additional information about this Adis Drug Review can be found <a href="https://www.springer.com/gp/adis/products-services/adis-journals-newsletters/adis-drug-reviews">here</a>. <br></p><p><br></p><p><br></p><p>Abstract</p><p></p><p>Luspatercept (Reblozyl^®), a first-in-class erythroid maturation agent, is approved in several countries worldwide for the treatment of adults with transfusion-dependent anaemia due to myelodysplastic syndromes (MDS), who have failed prior erythropoiesis-stimulating therapy, or β-thalassaemia. In pivotal, placebo-controlled, phase III trials, subcutaneous luspatercept significantly reduced red blood cell (RBC) transfusion requirements in patients with MDS or β-thalassaemia. Luspatercept had a generally manageable tolerability profile in clinical trials. Adverse events of special interest include thromboembolic events, hypertension and bone pain. Thus, luspatercept is an emerging treatment option in adults with transfusion-dependent anaemia due to MDS or β-thalassaemia. </p><p></p><p>© Springer Nature Switzerland AG 2021</p><br><p></p><p><br></p>