Real-world evidence on clinical outcomes in immune thrombocytopenia treated with thrombopoietin receptor agonists

Abstract

Aim: Eltrombopag and romiplostim are US FDA approved for treatment of immune thrombocytopenia in patients with insufficient response to other treatments. Clinical or real-world data comparing outcomes of the two drugs are limited. Methods: This retrospective cross-sectional study sought information on bleeding-related episodes (BREs), adverse events (AEs) and other outcomes of eltrombopag or romiplostim treatment in immune thrombocytopenia. Results: Patients receiving eltrombopag experienced significantly reduced BREs, severe BREs, rescue medication use and platelet transfusions. Diarrhea and headache were significantly less frequent in patients receiving eltrombopag; other AEs occurred equally in both groups. Conclusion: There may be a potential advantage for the use of eltrombopag versus romiplostim in the practice settings studied, based on rates of BREs and AEs and rescue medication utilization.