Blood Res 2022; 57(S1):
Published online April 30, 2022
https://doi.org/10.5045/br.2022.2022038
© The Korean Society of Hematology
Correspondence to : Dae Sik Kim, M.D., Ph.D.
Division of Hematology-Oncology, Department of Internal Medicine, Korea University Guro Hospital, 148 Gurodong-ro, Guro-gu, Seoul 08308, Korea
E-mail: kay9801@naver.com
This is an Open Access article distributed under the terms of the Creative Commons Attribution Non-Commercial License (http://creativecommons.org/licenses/by-nc/4.0) which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited.
Immune thrombocytopenia (ITP) is isolated thrombocytopenia characterized by autoimmune-mediated disruption of platelet without other etiologies. Treatments for chronic ITP consist of corticosteroids, intravenous immunoglobulins, anti-D immunoglobulin, rituximab, thrombopoietin receptor agonists, immunosuppressants and splenectomy. Although current therapies are effective in over two-thirds of patients, some patients are refractory to therapies or fail to achieve long-term responses. Recently, great advance has been made in identifying various mechanisms involved in ITP pathogenesis, and new treatments targeting these pathways are being developed. Novel agents such as splenic tyrosine kinase inhibitor, Bruton kinase inhibitor, plasma cell targeting therapies, neonatal Fc receptor inhibitor, platelet desialylation inhibitor, and inhibition of the classical complement pathway are expected to be effective for ITP treatment. This review summarizes current strategies and emerging therapies of ITP.
Keywords Immune thrombocytopenia, Platelet, Treatment, Pathogenesis, Thrombopoietin receptor agonist
Blood Res 2022; 57(S1): S112-S119
Published online April 30, 2022 https://doi.org/10.5045/br.2022.2022038
Copyright © The Korean Society of Hematology.
Dae Sik Kim
Division of Hematology-Oncology, Department of Internal Medicine, Korea University Guro Hospital, Seoul, Korea
Correspondence to:Dae Sik Kim, M.D., Ph.D.
Division of Hematology-Oncology, Department of Internal Medicine, Korea University Guro Hospital, 148 Gurodong-ro, Guro-gu, Seoul 08308, Korea
E-mail: kay9801@naver.com
This is an Open Access article distributed under the terms of the Creative Commons Attribution Non-Commercial License (http://creativecommons.org/licenses/by-nc/4.0) which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited.
Immune thrombocytopenia (ITP) is isolated thrombocytopenia characterized by autoimmune-mediated disruption of platelet without other etiologies. Treatments for chronic ITP consist of corticosteroids, intravenous immunoglobulins, anti-D immunoglobulin, rituximab, thrombopoietin receptor agonists, immunosuppressants and splenectomy. Although current therapies are effective in over two-thirds of patients, some patients are refractory to therapies or fail to achieve long-term responses. Recently, great advance has been made in identifying various mechanisms involved in ITP pathogenesis, and new treatments targeting these pathways are being developed. Novel agents such as splenic tyrosine kinase inhibitor, Bruton kinase inhibitor, plasma cell targeting therapies, neonatal Fc receptor inhibitor, platelet desialylation inhibitor, and inhibition of the classical complement pathway are expected to be effective for ITP treatment. This review summarizes current strategies and emerging therapies of ITP.
Keywords: Immune thrombocytopenia, Platelet, Treatment, Pathogenesis, Thrombopoietin receptor agonist
Table 1 . Novel therapies for treatment of immune thrombocytopenia..
Target | Drug class | Mechanism | Agents | Development status |
---|---|---|---|---|
Macrophage | Syk inhibitor | Decrease in ADCP (inhibition of macrophage phagocytosis) | Fostamatinib | Approved (US) |
Macrophage | BTK inhibitor | Decrease in ADCP (inhibition of macrophage phagocytosis) | Rilzabrutinib | Phase 3 (NCT04562766) |
Plasma cells | Proteasome inhibitor | Inhibits plasma cell production of anti-platelet antibody | Bortezomib | Phase 1 (NCT03013114) |
KZR-616 | Phase 1 (NCT04039477) withdrawn | |||
Plasma cells | Anti-CD38 antibody | Inhibits plasma cell production of anti-platelet antibody | Daratumumab | Phase 2 (NCT04703621) |
Mezagitamab | Phase 2 (NCT04278924) | |||
Antiplatelet antibodies | FcRn blocker | Increase clearance of anti-platelet antibody | Efgartigimod | Phase 2 (NCT03102593 |
Rozanolixizumab | Phase 3 (NCT00718692) | |||
Platelet | Neuraminidase inhibitor | Decrease in platelet desialylation thus reducing their destruction in the liver | Oseltamivir | Phase 2 (NCT01965626) |
Classical complement pathway | C1s inhibitor | Decrease in CDC (antibody inhibits C1s activity) | Sutimlimab | Phase 2 (NCT04669600) |
Abbreviations: ADCP, antibody-dependent cellular cytotoxicity; BTK, Bruton tyrosine kinase; CD, cluster of differentiation; CDC, complement-dependent cytotoxicity; FcRn, Neonatal Fc receptor; Syk, spleen tyrosine kinase..
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