Novel therapeutics for immune thrombocytopenia: an evolving treatment landscape

Immune thrombocytopenia (ITP) is an autoimmune-bleeding disorder; its management is shifting from empiricimmunosuppression and splenectomy to targeted, pathway-specific drugs that raise platelet counts with fewer long-term toxicities.

This review critically appraises evidence behind six mechanistic drug classes poised to reshape ITP care: thrombopoietin receptor agonists, spleen tyrosine kinase inhibitors, reversible Brutontyrosine kinase inhibitors, neonatal Fc-receptor antagonists, proximal complementblockers, and plasma-cell or BAFF-directed therapies. We interrogated PubMed, ClinicalTrials.gov, and hematology-conference abstracts (January 2010-May2025), retrieving synthesizing phase 2-3 trials and key observational studies. Throughout, we contrast these agents with steroids, intravenous immunoglobulin, and rituximab, highlighting shared immunomodulatory nodes and unique points of divergence that may inform rational sequencing or combination.

Mechanism-focused agents already enable steroid-sparing outpatient regimens and personalized care, yet durable remission and predictive biomarkers remain elusive. FcRn and reversible BTK inhibitors are closest to regulatory approval; complement blockade delivers24-hour platelet rescue, while plasma-cell or BAFF inhibition may consolidate sustained disease control. Research priorities include biomarker-guided pathway selection, optimal positioning with thrombopoietin receptor agonists, long-termpharmacovigilance, and cost-effectiveness analyses to ensure equitable global access.