Luspatercept in Myelodysplastic Syndromes: Who and When?

Hematol Oncol Clin North Am. 2020 Apr;34(2):393-400. doi: 10.1016/j.hoc.2019.10.004. Epub 2020 Jan 21.


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.

Publication types

  • Review

MeSH terms

  • Activin Receptors, Type II / metabolism
  • Activin Receptors, Type II / pharmacology
  • Activin Receptors, Type II / therapeutic use*
  • Anemia / diagnosis
  • Anemia / etiology
  • Clinical Trials as Topic
  • Erythropoiesis / drug effects
  • Erythropoiesis / genetics
  • Humans
  • Immunoglobulin Fc Fragments / pharmacology
  • Immunoglobulin Fc Fragments / therapeutic use*
  • Myelodysplastic Syndromes / complications
  • Myelodysplastic Syndromes / drug therapy*
  • Myelodysplastic Syndromes / etiology
  • Myelodysplastic Syndromes / metabolism
  • Recombinant Fusion Proteins / pharmacology
  • Recombinant Fusion Proteins / therapeutic use*
  • Signal Transduction / drug effects
  • Transforming Growth Factor beta / metabolism


  • Immunoglobulin Fc Fragments
  • Recombinant Fusion Proteins
  • Transforming Growth Factor beta
  • luspatercept
  • Activin Receptors, Type II
  • activin receptor type II-B