Sustained remission of chronic immune thrombocytopenia after discontinuation of treatment with thrombopoietin-receptor agonists in adults

Thrombopoietin-receptor agonists (TPO-RAs) are highly effective in immune thrombocytopenia (ITP). Recently, reports of sustained remission after TPO-RA discontinuation in adult ITP have been reported. We aimed to describe the subset of patients in whom TPO-RA therapy may induce a durable response. We retrospectively analyzed all of our adult ITP patients treated with TPO-RAs, and focused on patients with discontinued TPO-RA therapy due to treatment response. Forty-six relapsed or refractory ITP patients were treated with TPO-RAs. In 11 of these cases, TPO-RA therapy (seven romiplostim; four eltrombopag) was stopped after achieving treatment response. No side effects of TPO-RAs were observed. These patients were pretreated with 1–3 therapy lines plus splenectomy in six patients prior to TPO-RA administration. None of these patients experienced disease relapse after therapy discontinuation over a median follow-up of 33 months (16–54). Substantial proportion of ITP patients receiving TPO-RAs can maintain a durable response after treatment discontinuation. Sustained ITP remission probably does not depend on previous treatment, splenectomy, or disease duration.