Details

Autor(en) / Beteiligte

• Fenaux, Pierre
• Kiladjian, Jean Jacques
• Platzbecker, Uwe

Titel

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

Ist Teil von

• Blood, 2019-02, Vol.133 (8), p.790-794

Ort / Verlag

United States: Elsevier Inc

Erscheinungsjahr

2019

Quelle

MEDLINE

Beschreibungen/Notizen

• 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. [Display omitted]