This phase II study of 25 patients with refractory aplastic anemia found that treatment with an oral thrombopoietin mimetic, eltrombopag was associated with multilineage clinical responses. Eleven (44%) had a hematologic response in at least one lineage at 12 weeks, with minimal side effects. Of these, nine patients no longer needed platelet transfusion support, three patients became red cell transfusion independent, and nine patients had increased neuthrophil count.

– Lawrence Tim Goodnough