Thrombopoetin receptor agonist therapy in thrombocytopenia: ITP and beyond.

Eltrombopag is well established in treatment of severe immune thrombocytopenia (ITP) and is increasingly commonplace in second-line management. A role is also suggested for both bridging therapy for surgery, as well as treating thrombocytopenia due to non-immune aetiologies. We present the largest single-centre experience with eltrombopag, with our cohort of 62 patients. Patients with severe ITP (n = 34) had 91·2% response, which was sustained over a median of 18·5 months. In 41·4% of ITP cases (n = 14), complete response (CR- platelet count >100 × 109 /l) was achieved and in 2 cases, therapy was stopped and CR maintained. In our bridging group (n = 15) with a higher baseline platelet count, 93·3% achieved a CR. In the non-ITP group (n = 13), a response was achieved in 76·9%. In all groups, side effects were transient, with the drug discontinued in 2 patients due to minor complications (rash, nausea, diarrhoea). We conclude that eltrombopag is both effective and well tolerated as therapy in severe ITP. It is also advantageous in ITP patients who do not normally require therapy, but need a temporary platelet count boost pre-procedure. Furthermore, there are potentially far wider implications for the use of eltrombopag in counteracting thrombocytopenia beyond ITP, which merit further investigation.