[Efficacy and safety of romiplostim in aplastic anemia refractory or intolerant to immunosuppressive therapy with eltrombopag].

Abstract

Combination of immunosuppressive therapy (IST) and thrombopoietin receptor agonists has revolutionized the treatment of aplastic anemia. In this study, 18 patients with eltrombopag (EPAG)-refractory and intolerant AA, including 16 with severe AA, were switched from EPAG to romiplostim and continued with romiplostim for at least three months. Of the 18 patients (7 refractory and 11 intolerant to EPAG), 13 (72%) achieved a response, with a therapeutic response in at least one lineage within three months, and nine patients (50%) showed a trilineage response. All three patients who started romiplostim over three years after diagnosis failed to respond to romiplostim. In three of the four patients with adequate response to romiplostim, interruption of romiplostim resulted in relapsed thrombocytopenia, and early resumption of romiplostim resulted in recovered blood counts. These results suggest that romiplostim may achieve a response in IST+EPAG refractory/intolerant SAA.