Recent advances in the mechanisms and treatment of immune thrombocytopenia

Primary immune thrombocytopenia is an autoimmune disease associated with a reduced peripheral blood platelet count. The phenotype is variable with some patients suffering no bleeding whilst others have severe bleeding which may be fatal. Variability in clinical behaviour and treatment responses reflects its complex underlying pathophysiology. Historically the management has relied heavily on immune suppression. Recent studies have shown that the older empirical immune suppressants fail to alter the natural history of the disease and are associated with a poor quality of life for patients. Newer treatments, such as the thrombopoietin receptor agonists, have transformed ITP care. They have high efficacy, are well tolerated and improve patients' quality of life. A greater understanding of the underlying pathophysiology of this disorder has helped develop a number of new targeted therapies. These include inhibitors of the neonatal Fc receptor inhibitors, Bruton tyrosine kinase and complement pathway. Here we discuss the mechanisms underlying ITP and the new approach to ITP care. Copyright (C) 2022 The Author(s). Published by Elsevier B.V.

Automated summary

Primary immune thrombocytopenia is an autoimmune disease characterized by a reduced peripheral blood platelet count. Its clinical behavior and treatment responses vary, reflecting its complex underlying pathophysiology. Traditional immune suppression therapy has limited efficacy and affects patients' quality of life. Newer treatments, such as thrombopoietin receptor agonists, have shown high efficacy and improved patient outcomes. Understanding the underlying mechanisms of the disease has led to the development of targeted therapies.