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Review
. 2020 Apr;34(2):393-400.
doi: 10.1016/j.hoc.2019.10.004. Epub 2020 Jan 21.

Luspatercept in Myelodysplastic Syndromes: Who and When?

Rami S Komrokji  1
Affiliations
Review

Luspatercept in Myelodysplastic Syndromes: Who and When?

Rami S Komrokji. Hematol Oncol Clin North Am. 2020 Apr.

Abstract

Anemia is the most common clinical manifestation of myelodysplastic syndrome (MDS), and most patients become red blood cell transfusion dependent. Defective erythropoiesis includes impaired terminal erythroid maturation. There are limited options for treatments of anemia in lower-risk MDS after failure of erythroid-stimulating agents. Luspatercept is an activin receptor type IIB fusion ligand trap novel agent. Luspatercept showed promising activity for treating anemia in patients with MDS with ring sideroblast subtypes. This article reviews the mechanism of impaired erythropoiesis in MDS. It summarizes clinical data with luspatercept and foresees how to best use this treatment in practice.

Keywords: Anemia; Ineffective erythropoiesis; Luspatercept; Myelodysplastic syndromes.

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Conflict of interest statement

Disclosure Speaker bureaus: Jazz Pharmaceuticals, Novartis Oncology, Alexion Pharmaceuticals. Consultancy and Advisory boards: Celgene, Novartis Oncology, Jazz Pharmaceuticals, Daiichi-Sankyo, Inc (DSI), Agios Pharmaceuticals, Janssen and Janssen, Pfizer.

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