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| KZR-616 | Phase 1 (NCT04039477) withdrawn | |||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.
Blood Res 2022;57:S112~S119 https://doi.org/10.5045/br.2022.2022038
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