Sustained response off therapy after fostamatinib: A chronic refractory ITP case report

• DOI: 10.1016/j.heliyon.2023.e13462
• Publication Date: 2023-02-04T02:10:25Z
• Authors: Giuseppe Auteri; Mattia Biondo; Camilla Mazzoni; Marta Venturi; Andrea Davide Rom...; Simona Paglia; Michele Cavo; Nicola Vianelli; Francesca Palandri
• Keywords: Discontinuation; Refractory (planetary science); Eltrombopag; Romiplostim

Fostamatinib is a SYK-inhibitor drug recently approved by the FDA and EMA for treating chronic immune thrombocytopenia. This induces response in about 40% of patients has good toxicity profile. It known that discontinuing thrombopoietin receptor agonists (TRAs) with maintenance sustained off therapy possible. On fostamatinib, we do not yet have such information. In this case report, describe story woman multirefractory thrombocytopenia (steroids, splenectomy, rituximab, both available TRAs). After 16 years from diagnosis, she started fostamatinib within clinical trial achieved complete response. Grade 1-2 headache diarrhea occurred during first months therapy. These adverse events were resolved dose reduction fostamatinib. Despite reduction, platelet count remained steadily above 80 × 109/L. 4 years, was gradually reduced finally discontinued no drop count. which discontinuation resulted