Luspatercept for β-thalassemia: beyond red blood cell transfusions.

Abstract

INTRODUCTION:

Red blood cell transfusions and iron chelation therapy are the cornerstone of treatment for β-thalassemia, with allogeneic hematopoietic stem cell transplantation and gene therapy offering further disease-management options for eligible patients. With up to 90% of severe cases of β-thalassemia occurring in resource-constrained countries, and estimates indicating that 22,500 deaths occur annually as a direct consequence of undertransfusion, provision of adequate treatment remains a major issue.

AREAS COVERED:

In this review, we provide an overview of luspatercept, a first-in-class erythroid maturation agent, and present the available clinical data related to the treatment of β-thalassemia.

EXPERT OPINION:

The recent approval of luspatercept offers a new, long-term therapeutic option for adult patients with transfusion-dependent β-thalassemia to reduce red blood cell transfusion burden, anemia, and iron overload.

• Authors: Ali T Taher, Maria Domenica Cappellini
• Journal: Expert opinion on biological therapy (Expert Opin Biol Ther) Vol. 21 Issue 11 Pg. 1363-1371 (11 2021) ISSN: 1744-7682 [Electronic] England
• PMID: 34404288 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't, Review)

Chemical References

• Immunoglobulin Fc Fragments
• Recombinant Fusion Proteins
• luspatercept
• Activin Receptors, Type II

Topics

• Activin Receptors, Type II
• Erythrocyte Transfusion
• Humans
• Immunoglobulin Fc Fragments
• Recombinant Fusion Proteins
• beta-Thalassemia (therapy)