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Sotatercept with long-term extension for the treatment of anaemia in patients with lower-risk myelodysplastic syndromes: a phase 2, dose-ranging trial

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Sotatercept with long-term extension for the treatment of anaemia in patients with lower-risk myelodysplastic syndromes: a phase 2, dose-ranging trial

The Lancet Haematology

Published: 10 January 2018

DOI: https://doi.org/10.1016/S2352-3026(18)30002-4

Rami Komrokji, Guillermo Garcia-Manero, MD, Lionel Ades, MD, Thomas Prebet, MD, David P Steensma, MD, Joseph G Jurcic, MD, Mikkael A Sekeres, MD, Jesus Berdeja, MD, Michael R Savona, MD, Prof Odile Beyne-Rauzy, MD, Aspasia Stamatoullas, MD, Amy E DeZern, MD, Jacques Delaunay, MD, Gautam Borthakur, MD, Robert Rifkin, MD, Thomas E Boyd, MD, Abderrhamane Laadem, MD, Bond Vo, PharmD, Jennie Zhang, PhD, Marie Puccio-Pick, MS, Kenneth M Attie, MD, Prof Pierre Fenaux, MD, Alan F List, MD

Background: Myelodysplastic syndromes are characterised by ineffective erythropoiesis leading to anaemia. Sotatercept (ACE-011) is a novel activin receptor type IIA fusion protein that acts as a ligand trap to neutralise negative regulators of late-stage erythropoiesis. The aim of the study was to establish a safe and effective dose of sotatercept for the treatment of anaemia in patients with lower-risk myelodysplastic syndromes.

 

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