Recent advances in the management of immune thrombocytopenic purpura (ITP): A comprehensive review

Abstract

Autoimmune disorders place a substantial burden on the healthcare system all over the world affecting almost 3% to 8% of the population. Immune thrombocytopenic purpura (ITP), also known as idiopathic thrombocytopenic purpura, is a blood disorder in which the body immune system destroys platelets, leading to low platelet counts in the blood (peripheral blood platelet count < 150 × 109/L). Although the pathophysiology of ITP is not fully understood, it is believed to result from a complex interplay between hereditary and environmental variables. Certain factors, such as a low platelet count, history of bleeding, and certain comorbidities can increase the risk of severe bleeding in patients with ITP. Corticosteroids, intravenous immunoglobulin (IVIG), immunosuppressants, rituximab, and thrombopoietin receptor agonists (TPO-RAs) are some of the advanced treatments for ITP. Although these therapies may be successful, they also carry the risk of negative effects. Recently, significant advancements have been made in the understanding and treatment of ITP. There is still much to learn about the disease, and new, more effective treatments are needed. This comprehensive review offers a comprehensive assessment of recent advancements in ITP management, with a focus on active research projects, novel therapeutic targets, new treatment modalities, and areas of uncertainty and unmet needs. According to research, it is crucial to develop individualized treatment plans for ITP patients based on their age, platelet count, risk of bleeding, and comorbidities. The article also looks at how future developments in gene editing, bispecific antibody therapies, and cellular therapy may completely change the treatment of ITP.

Figure 1.

ITP immune effector mechanism: In ITP, bone marrow-based antigen-presenting cells (APCs) process and deliver platelet autoantigens to autoreactive T cells. This starts a chain of events that result in the creation of platelet-specific autoantibodies and the activation of cytotoxic T cells. In peripheral circulation, platelets can be destroyed by both autoantibodies and cytotoxic T lymphocytes. The complement cascade may also assault autoantibody-opsonized platelets. Autoantibodies and cytotoxic T lymphocytes can also prevent megakaryocytes in the bone marrow from producing platelets.^[5] Source: Provan D, Semple JW. Recent advances in the mechanism and treatment of immune thrombocytopenia. EBioMedicine, 2022;76:103820. CC BY-NC-ND 4.0.

Figure 2.

T cell and B cell mechanisms in pathogenesis of ITP: Both T cells and B cells play crucial roles in the development of ITP. Autoantibodies produced by B cells have the potential to impair platelet function and formation. In addition, platelets can be directly destroyed by cytotoxic T lymphocytes. Regulating the autoimmune response in ITP patients is frequently insufficient due to an imbalance in the numbers of regulatory T cells (Tregs)^[22] Source: Swinkels M, Rijkers M, Voorberg J, Vidarsson G, Leebeek FWG, Jansen AJG. Emerging Concepts in Immune Thrombocytopenia. Front Immunol, 2018 Apr 30;9:880. CC BY-NC-ND 4.0.