Loading...
Although the prognosis for patients with severe aplastic anemia (SAA) has improved in recent years, about 30% of patients remain pancytopenic despite treatment with immunosuppressive agents. Thrombopoietin (TPO) regulates hematopoiesis, and agents that activate the TPO receptor might play a role in the management of patients with refractory SAA. A prior pilot study of eltrombopag, a TPO receptor agonist, showed efficacy in 11 of 25 patients (NEJM JW Oncol Hematol Jul 10 2012).
To further evaluate the effectiveness and safety of eltrombopag, the investigators increased enrollment to 43 patients with median hemoglobin, 7.9 g/dL; median platelet count, 8000 per μL, and median neutrophil count, 570 per μL. Eltrombopag was administered at an init…