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Translating mechanisms into therapeutic strategies for immune thrombocytopenia (ITP): Lessons from clinical trials

免疫性血小板减少症 美罗华 埃尔特罗姆博帕格 医学 临床试验 重症监护医学 人口 罗米普洛斯蒂姆 免疫学 血小板生成素 伊库利珠单抗 免疫系统 血小板 抗体 补体系统 内科学 干细胞 遗传学 环境卫生 造血 生物
作者
Mahda Delshad,Zeinab Davoodi‐Moghaddam,Atieh Pourbagheri‐Sigaroodi,Mohammad Faranoush,Hassan Abolghasemi,Davood Bashash
出处
期刊:Thrombosis Research [Elsevier BV]
卷期号:235: 125-147
标识
DOI:10.1016/j.thromres.2024.02.005
摘要

Abstract

Immune thrombocytopenia (ITP) is an autoimmune disorder that causes a significant reduction in peripheral blood platelet count. Fortunately, due to an increased understanding of ITP, there have been significant improvements in the diagnosis and treatment of these patients. Over the past decade, there have been a variety of proven therapeutic options available for ITP patients, including intravenous immunoglobulins (IVIG), Rituximab, corticosteroids, and thrombopoietin receptor agonists (TPO-RAs). Although the effectiveness of current therapies in treating more than two-thirds of patients, still some patients do not respond well to conventional therapies or fail to achieve long-term remission. Recently, a significant advancement has been made in identifying various mechanisms involved in the pathogenesis of ITP, leading to the development of novel treatments targeting these pathways. It seems that new agents that target plasma cells, Bruton tyrosine kinase, FcRn, platelet desialylation, splenic tyrosine kinase, and classical complement pathways are opening new ways to treat ITP. In this study, we reviewed the pathophysiology of ITP and summarized updates in this population's management and treatment options. We also took a closer look at the 315 ongoing trials to investigate their progress status and compare the effectiveness of interventions. May our comprehensive view of ongoing clinical trials serve as a guiding beacon, illuminating the path towards future trials of different drugs in the treatment of ITP patients.
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