Evaluating fostamatinib disodium as a treatment option for immune thrombocytopenia in adult patients.

Introduction: Immune thrombocytopenia (ITP) is an autoimmune bleeding disorder characterized by increased platelet destruction and decreased platelet production, leading to thrombocytopenia with or without bleeding manifestations. The majority of patients experiencing treatment need will eventually need secondary treatment following first-line therapy with steroids. In 2018, the oral spleen tyrosine kinase inhibitor fostamatinib received US Food and Drug Administration approval for ITP patients with an insufficient response to a previous treatment.

Areas Covered: This review outlines pharmacological characteristics of fostamatinib and provides an overview of its efficacy and safety results in phase II and III trials, followed by the expert opinion of the authors.

Expert Opinion: Increasing knowledge on the role of different players and mechanisms in the pathophysiology of autoimmune disorders, in general, and of ITP, in particular, has led to the development of several new treatment options, as illustrated by the introduction of fostamatinib in the treatment of ITP. However, lacking direct comparison with other recent treatment options (in particular, thrombopoietin receptor agonists), its use should be evaluated critically taking into account the unique toxicity and potential drug-drug interaction profile.