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Luspatercept for the treatment of anemia in myelodysplastic syndromes (MDS) and primary myelofibrosis (PMF)

Pierre Fenaux, Jean Jacques Kiladjian and Uwe Platzbecker

Abstract

Anemia of lower risk MDS and PMF generally becomes resistant to available treatments, leading to RBC transfusions, iron overload, shortened survival and poor quality of life. The TGF-β superfamily, including activins and growth differentiation factors (GDF), is aberrantly expressed in lower risk MDS and PMF. Luspatercept (and Sotatercept), ligand traps that particularly inhibit GDF 11, lead to RBC transfusion independence in 10-50% of lower risk MDS resistant to available treatments, and have started to be used in PMF.

  • Submitted November 16, 2018.
  • Accepted December 17, 2018.