Background Acquired aplastic anemia results from immune-mediated destruction of bone
marrow. Immunosuppressive therapies are effective, but reduced numbers of residual stem
cells may limit their efficacy. In patients with aplastic anemia that was refractory to
immunosuppression, eltrombopag, a synthetic thrombopoietin-receptor agonist, led to
clinically significant increases in blood counts in almost half the patients. We combined
standard immunosuppressive therapy with eltrombopag in previously untreated patients with …

Immunosuppressive treatment (IST) with horse antithymocyte globulin (hATG) and
cyclosporine (CsA) for severe aplastic anemia (SAA) has a 60-65% rate of overall
hematologic response (OR) and approximately 10% complete response (CR). These rates
have remained stable over the last three decades despite efforts to improve upon this
regimen (Scheinberg Blood 2012). Eltrombopag (EPAG), an oral thrombopoietin receptor
agonist has activity in refractory SAA producing hematologic responses, the majority …