Luspatercept
- PMID: 32809470
- Bookshelf ID: NBK560635
Luspatercept
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.
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References
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- 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
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- 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
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- Komrokji RS. Luspatercept in Myelodysplastic Syndromes: Who and When? Hematol Oncol Clin North Am. 2020 Apr;34(2):393-400. - PubMed
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- Markham A. Luspatercept: First Approval. Drugs. 2020 Jan;80(1):85-90. - PubMed
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