Применение элтромбопага у детей и молодых взрослых с приобретенной апластической анемией

Abstract

The standard immunosuppressive therapy (IST) with antithymocyte globulin (ATG) and cyclosporine in treatment of acquired aplastic anemia (AA) results to hematologic response in two thirds of patients, and approximately one third of the responding patients is relapsed. One third of patients have disease that is refractory to IST, with persistent severe cytopenia and a profound deficit in hematopoietic stem cells. In patients with refractory or recurrent disease, the use of alternative immunosuppressive drugs is possible, but the efficacy of the latter does not high. Recent studies have demonstrated the efficacy of eltrombopag in refractory aplastic anemia and in the first line of therapy in combination with IST. It has been shown eltrombopag not only restored the level of platelets, but was associated with multilineage clinical responses. In this study we analyzed the use of eltrombopag in 15 children and young adults with AA: 11 patients in refractory disease, one patient in relapse and three patients in the first line of therapy in combination with IST. The overall efficacy of treatment was 40% (6/15). The main adverse event was moderate and reversible increase of hepatic transaminases (3/15, 20%). A complete hematologic response was achieved in 3/15 patients (20%), and in 2 patients the response is continued after drug withdrawal. Eltrombopag is effective in patients with refractory AA, and it may be an alternative option in patients who do not have suitable donors for hematopoietic stem cells transplantation (HSCT). The first-line treatment with eltrombopag in combination with standard IST in our small group of patients also showed efficacy (60%) and safety.