Treating Thalassemia Patients with Luspatercept: An Expert Opinion Based on Current Evidence

J Clin Med. 2023 Mar 29;12(7):2584. doi: 10.3390/jcm12072584.

Abstract

Luspatercept has recently been approved for the treatment of beta-thalassemia and its use in clinical practice has been increasing. As it is the first erythroid maturation drug available for this diagnosis, the expertise about its use is still limited. To address this point, and to promote awareness and guide the clinical use of luspatercept in beta-thalassemia, this paper was developed as a consensus by experts from the Italian Society of Thalassemia and Hemoglobinopathies (SITE). After a brief presentation of the core features of luspatercept, a comprehensive set of questions is addressed, covering relevant aspects for the practical management of this new therapeutic option.

Keywords: SITE; erythroid maturation agent; ineffective erythropoiesis; luspatercept; thalassemia.

Grants and funding

Supported by Regione Autonoma Sardegna L.R. 11/90.