Immune Thrombocytopenia: Advances in Pathogenesis and Targeted Therapy

Abstract

Immune thrombocytopenia (ITP), an acquired autoimmune disease characterized by accelerated platelet destruction and impaired platelet production, is clinically heterogeneous and associated with a high risk of bleeding. With the advancement of multi-omics technologies, the medical community’s understanding of the pathogenesis of ITP has expanded from a single autoantibody-mediated hypothesis to a multidimensional framework encompassing lymphocyte network dysregulation, megakaryocyte maturation defects, and immune tolerance defects in the bone marrow microenvironment. Innovations in diagnostic approaches, coupled with the emergence of targeted therapies such as TPO receptor agonists, FcRn inhibitors, and multi-targeted kinase inhibitors, are contributing to the development of precision treatment strategies for ITP. Integrated Traditional Chinese and Western Medicine (ITCM) has has shown potential clinical value for its role in comprehensive disease-course intervention, restoration of immune homeostasis, and improvement of the bone marrow hematopoietic microenvironment. This article systematically reviews the core pathological mechanisms and cutting-edge targeted intervention strategies in the field of ITP, evaluates the clinical efficacy of integrated traditional Chinese and Western medicine approaches, and prospectively explores future development pathways guided by molecular subtyping, with the aim of providing high-value academic references for basic translational research and personalized clinical diagnosis and treatment.