Eltrombopag: Wielding a double-edged sword?

Abstract

In this issue of Blood,Winkler et al1 report that extending eltrombopag therapy to at least 24 weeks can improve hematologic responses in patients with refractory severe aplastic anemia (rSAA), a lymphocyte-mediated bone marrow failure syndrome. Genetic analysis of patients from this and the initial rSAA cohorts2,3 warn of cytogenetic evolution in 18% of eltrombopag-treated rSAA patients.