Eltrombopag (EPAG), an oral thrombopoietin receptor agonist (TPO-RA) initially developed for chronic immune thrombocytopenia, was first proven by the National Institutes of Health (NIH) to be effective in severe aplastic anemia (SAA) patients refractory to IST (7), presuming its efficacy in stimulating residual HSPCs. The gene discussed is TPO; the disease is severe aplastic anemia.