Activin receptor modulation

Conventional treatment regimen of lower-risk MDS with erythropoiesis-stimulating agents often fails to induce sustained erythroid improvement in these patients that frequently harbor defects in late-stage erythroblasts downstream of erythropoietin action. Thus, alternative treatment options that would achieve sustainable transfusion independence are needed. In this chapter, we will discuss activin receptor ligand traps as novel therapeutic strategies for low-risk MDS subgroups that were recently shown to alleviate anemia by specifically inhibiting aberrant TGF-β signaling and thereby promoting erythroid differentiation.