Eltrombopag (TPO-R agonism) and selective TET2-mutant clone suppression

Current understanding

Eltrombopag, a small-molecule thrombopoietin receptor (MPL) agonist, is hypothesized to both stimulate residual normal hematopoiesis and preferentially suppress the growth of TET2-mutant clones in low-risk MDS and CMML. The mutation-selective cytoreductive effect is a novel proposed mechanism currently supported only by early-phase clinical investigation (trial-early evidence), with the molecular basis for TET2-clone selectivity not yet established.

Evidence log

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