Luspatercept for the treatment of anemia in myelodysplastic syndromes and primary myelofibrosis

Blood. 2019 Feb 21;133(8):790-794. doi: 10.1182/blood-2018-11-876888. Epub 2019 Jan 2.


Anemia of lower-risk myelodysplastic syndromes (MDSs) and primary myelofibrosis (PMF) generally becomes resistant to available treatments, leading to red blood cell (RBC) transfusions, iron overload, shortened survival, and poor quality of life. The transforming growth factor-β superfamily, including activins and growth differentiation factors (GDFs), is aberrantly expressed in lower-risk MDSs and PMF. Luspatercept (and sotatercept), ligand traps that particularly inhibit GDF11, lead to RBC transfusion independence in 10% to 50% of lower-risk MDSs resistant to available treatments, and have started to be used in PMF.

Publication types

  • Review

MeSH terms

  • Activin Receptors, Type II
  • Activins / therapeutic use*
  • Anemia / mortality
  • Anemia / therapy*
  • Bone Morphogenetic Proteins / antagonists & inhibitors
  • Disease-Free Survival
  • Erythrocyte Transfusion
  • Growth Differentiation Factors / antagonists & inhibitors
  • Humans
  • Immunoglobulin Fc Fragments / therapeutic use*
  • Iron Overload / etiology
  • Iron Overload / mortality
  • Myelodysplastic Syndromes / mortality
  • Myelodysplastic Syndromes / therapy*
  • Recombinant Fusion Proteins / therapeutic use*
  • Survival Rate


  • Bone Morphogenetic Proteins
  • GDF11 protein, human
  • Growth Differentiation Factors
  • Immunoglobulin Fc Fragments
  • Recombinant Fusion Proteins
  • Activins
  • luspatercept
  • Activin Receptors, Type II