Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Book

Luspatercept

In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2025 Jan.
.
Affiliations
Free Books & Documents
Book

Luspatercept

Bina Patel et al.
Free Books & Documents

Excerpt

Luspatercept is an FDA-approved recombinant fusion protein used to manage anemia in patients with β-thalassemia who require regular blood transfusions. This drug is also indicated for adults with very low-to-intermediate risk myelodysplastic syndromes (MDS) who are naive to erythropoiesis-stimulating agents and may need red blood cell transfusions. Luspatercept binds to transforming growth factor-β ligands, reducing SMAD2 and SMAD3 signaling to promote erythroid maturation.

PubMed Disclaimer

Conflict of interest statement

Disclosure: Bina Patel declares no relevant financial relationships with ineligible companies.

Disclosure: Leila Moosavi declares no relevant financial relationships with ineligible companies.

References

    1. Platzbecker U, Germing U, Götze KS, Kiewe P, Mayer K, Chromik J, Radsak M, Wolff T, Zhang X, Laadem A, Sherman ML, Attie KM, Giagounidis A. Luspatercept for the treatment of anaemia in patients with lower-risk myelodysplastic syndromes (PACE-MDS): a multicentre, open-label phase 2 dose-finding study with long-term extension study. Lancet Oncol. 2017 Oct;18(10):1338-1347. - PubMed
    1. Piga A, Perrotta S, Gamberini MR, Voskaridou E, Melpignano A, Filosa A, Caruso V, Pietrangelo A, Longo F, Tartaglione I, Borgna-Pignatti C, Zhang X, Laadem A, Sherman ML, Attie KM. Luspatercept improves hemoglobin levels and blood transfusion requirements in a study of patients with β-thalassemia. Blood. 2019 Mar 21;133(12):1279-1289. - PMC - PubMed
    1. Komrokji RS. Luspatercept in Myelodysplastic Syndromes: Who and When? Hematol Oncol Clin North Am. 2020 Apr;34(2):393-400. - PubMed
    1. Markham A. Luspatercept: First Approval. Drugs. 2020 Jan;80(1):85-90. - PubMed
    1. Kubasch AS, Platzbecker U. Setting Fire to ESA and EMA Resistance: New Targeted Treatment Options in Lower Risk Myelodysplastic Syndromes. Int J Mol Sci. 2019 Aug 07;20(16) - PMC - PubMed

Publication types

LinkOut - more resources