Upfront eltrombopag monotherapy induces stable hematologic remission in pediatric patients with nonsevere idiopathic aplastic anemia.

Aplastic anemia (AA) is characterized by multilineage cytopenias and bone marrow hypocellularity. Severe AA can be treated with immunosuppressive therapy (IST) and/or allogeneic hematopoietic stem cell transplantation. The thrombopoietin agonist eltrombopag has been shown to induce hematopoietic recovery and transfusion independence in adults with refractory and relapsed AA. Recently, upfront eltrombopag therapy in patients with AA in combination with IST has shown efficacy. Data for its use without concurrent IST in pediatric patients with AA remain sparse. Here we report two pediatric patients with AA not meeting severe criteria who achieved hematologic response with upfront eltrombopag monotherapy.