Insights in Blood Disorders
Open AccessPharmacological Management of Immune Thrombocytopenia: Established and Emerging Therapeutic Options
Authors: Emmanuel Andrès, Xavier Jannot, Jean-Edouard Terrade, Noël Lorenzo-Villalba.
Abstract
•Immune thrombocytopenia (ITP) is a heterogeneous, antibody- and T-cell-mediated bleeding disorder driven by accelerated peripheral platelet destruction and inappropriately low, ineffective platelet production; treatment goals have shifted from a fixed platelet-count target to control of bleeding and preservation of quality of life.
•Corticosteroids remain the standard first-line therapy, but should be limited in duration; intravenous immunoglobulin (IVIg) and, where available, intravenous anti-D immunoglobulin, are reserved for rapid platelet elevation in active bleeding or before invasive procedures.
•Thrombopoietin receptor agonists (TPO-RAs: romiplostim, eltrombopag, avatrombopag, and, in Asia, hetrombopag) and rituximab are the best-established second-line options and, per the 2019 American Society of Hematology (ASH) guidelines, are now generally favoured over splenectomy as first steroid-sparing strategies, splenectomy being reserved for patients who fail or are unsuitable for medical second-line therapy.
•Fostamatinib, an oral spleen tyrosine kinase (Syk) inhibitor, and rilzabrutinib, an oral reversible Bruton tyrosine kinase (BTK) inhibitor approved by the FDA in 2025, provide non-TPO-RA, non-immunosuppressive alternatives for multirefractory disease with distinct safety profiles.
•A wave of agents targeting IgG recycling (FcRn antagonists: efgartigimod, rozanolixizumab, nipocalimab), complement activation (sutimlimab, iptacopan), long-lived autoreactive plasma cells and B cells (ianalumab, anti-CD38 and anti- BCMA strategies), and, investigationally, chimeric antigen receptor (CAR) T cells, is being developed for patients with insufficient response to current second-line agents.
•Choice among available options should be individualised, taking into account bleeding phenotype, comorbidities (thrombosis risk, infection risk, pregnancy, renal or hepatic impairment), patient preference for route and frequency of administration, and, where relevant, cost and local marketing authorisation status.
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