Fostamatinib in immune thrombocytopenia: real-world experience in European countries

The management of immune thrombocytopenia (ITP) is evolving with the availability of new treatments. Fostamatinib, targeting spleen tyrosine kinase-dependent platelet phagocytosis, offers an alternative approach; it has been approved in Europe and the USA for adult patients with chronic ITP who are refractory to other treatments. In recent years, experience has been gained with fostamatinib in clinical practice. Based on a symposium (Milan, June 2025) and augmented with relevant literature identified in PubMed and key references known to the authors, this article summarizes data from three large European real-world registry cohorts. Studies included heavily pretreated patients with ITP (median 3-6 previous treatments before starting fostamatinib). Real-world evidence offers insights for achieving more durable responses with respect to improving platelet counts, with >40% of patients still receiving fostamatinib after 6 months' therapy, providing indirect evidence of both its efficacy and safety in patients with difficult-to-manage ITP. Despite a lack of second-line effectiveness data, the low thrombosis incidence suggests an earlier place in treatment strategies for patients with a high baseline thrombosis risk. However, the overall findings are limited by the retrospective design of some studies, the heterogeneous nature of the patient populations, and the relatively small number of patients evaluated to date.