An alternative intermittent eltrombopag dosing protocol for the treatment of chronic immune thrombocytopenia

Abstract

Eltrombopag, an oral thrombopoietin receptor agonist, is dosed daily to treat chronic immune thrombocytopenia (ITP). As it has a half-life of 26–35 h in ITP patients and requires patents to abide by strict dietary restrictions that may impair quality of life and reduce compliance, we developed an alternative intermittent (AI) eltrombopag dosing protocol for ITP, with dosing less frequent than once daily. Ten patients were treated with AI dosing for a median of 94 (range: 29–156) weeks, with most patients treated with 2–4 weekly doses for extended durations. During AI dosing, 95%, 84% and 71% of all platelet counts were ≥20 × 109 l–1, ≥50 × 109 l–1 and ≥100 × 109 l–1, respectively. Five patients required rescue treatment for thrombocytopenia and World Health Organization grade 1 mucocutaneous bleeding, and there were no thromboembolic events. In summary, intermittently dosed eltrombopag was efficacious in treating chronic ITP in a small cohort, with rates of platelet response and rescue treatment comparable with rates in studies evaluating daily dosing.