Background

In a single institution retrospective study from King's College Hospital, data was presented on Eltrombopag use as a single agent, over a 5 year period in aplastic anaemia (AA).

Findings

In the real world setting, Eltrombopag use is safe and well tolerated. Responses are seen in nearly a quarter of patients, and a response is more likely in people with less severe disease and predicts for a better survival amongst patients with aplastic anaemia.

In this context, we eagerly await data from the RACE clinical trial, conducted across various centres around Europe, where nearly 130 patients have been recruited into this study. It compares the addition of Eltrombopag to standard immunosuppressive treatment (IST) in patients with aplastic anaemia. More importantly, the derivatives from this study will also provide information about the genetic, molecular and immunologic aspects of the disease which help stage, prognosticate and define response to treatment in aplastic anaemia.