Eltrombopag Inhibits TET Dioxygenase to Contribute to Hematopoietic Stem Cell Expansion in Aplastic Anemia

TET inhibition in TET2 mutant malignant myeloid cells prevented neoplastic clonal evolution, in vitro and in vivo. This new mechanism of action may offer a restorative therapeutic index and provide a scientific rationale to treat selected patients with TET2-mutant or TET deficiency associated myeloid malignancies.