Immune thrombocytopenia: a comprehensive review from pathophysiology to promising treatment modalities

Abstract

Introduction: The present understanding of immune thrombocytopenia (ITP) is rapidly growing in tandem with the advent of new treatment modalities. This review article is a bird’s-eye view of ITP from pathogenesis to available therapeutic options, with extended focus on the novel agents. Areas covered: ITP, an acquired autoimmune disorder characterized by isolated thrombocytopenia, can present as a primary disease in the absence of a known etiology of thrombocytopenia, or as a secondary entity associated with other autoimmune disorders, infections, or drug reactions. The pathophysiology of ITP is heterogeneous but generally includes both increased platelet destruction and decreased platelet production. Traditionally, ITP has been treated with immunosuppressive therapy, but the newer agents romiplostim and eltrombopag, which belong to the family of thrombopoietin (TPO) receptor agonists, have proved to be highly efficacious in treating the most refractory ITP cases with a favorable safety profile. Other therapies are also on the rise, including Syk inhibitors and anti-CD40L antibodies, among others. Expert opinion: Although their mechanism of response durability is not yet fully understood, TPO receptor agonists have opened the door to a new era in the treatment of ITP, especially in light of their sustained response rates and minimal associated adverse events. © 2016 Informa UK Limited, trading as Taylor & Francis Group.