Internal medicine

Myelodysplastic syndromes

Aplastic anemia

Immunology

Anemia

Ineffective erythropoiesis

Erythropoiesis

Bone marrow

Myelofibrosis

Medicine

Blood

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.

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