EVOLVİNG PARADİGMS İN THE MANAGEMENT OF PRİMARY IMMUNE THROMBOCYTOPENİA İN ADULTS: FROM CORTİCOSTEROİDS TO TARGETED BİOLOGİC THERAPİES

Résumé fourni par la source

Immune thrombocytopenia (ITP) is an acquired autoimmune disorder characterized by isolated thrombocytopenia (platelet count <100 x 10^9/L) in the absence of another explanatory cause. Although bleeding risk remains central to clinical management, ITP also imposes a substantial broader burden, particularly through fatigue, impaired daily functioning, and reduced health-related quality of life. For decades, treatment relied on corticosteroids as first-line therapy, followed by a limited range of second-line options including thrombopoietin receptor agonists (TPO-RAs), rituximab, splenectomy, and, more recently, fostamatinib. Growing understanding of the immune mechanisms underlying both accelerated platelet destruction and impaired platelet production has expanded the therapeutic landscape to include mechanism-directed agents targeting Bruton tyrosine kinase, BAFF-receptor-dependent B-cell biology, the neonatal Fc receptor, and plasma-cell compartments. This narrative review focuses on primary ITP in adults and summarizes current evidence on epidemiology, pathophysiology, clinical and humanistic burden, established therapies, and emerging targeted treatments. Particular attention is given to distinguishing approved therapies from investigational agents, interpreting heterogeneous trial endpoints with caution, and integrating disease features, safety considerations, and patient preferences into treatment selection. Although recent phase II and III studies support the biological and clinical promise of several targeted agents, the optimal sequencing of these therapies remains to be defined.

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