Table 3.
Target therapies in autoimmune hemolytic anemia (AIHA).
| Drug | Mechanism | Setting | Route of administration | Efficacy |
|---|---|---|---|---|
| B-cell directed monoclonal antibodies | ||||
| Rituximab | Anti-CD20 | wAIHA/CAD | IV | 70–80%/50–60% |
| Rituximab | Anti-CD20 | wAIHA/CAD | SC | 100% |
| R-Fludarabine | Anti-CD20 + purine analog | CAD | IV | 76% |
| R-CTX-Dex | Anti-CD20 + alkylator | WAIHA | IV | 97% |
| R-Bendamustine | Anti -CD20 + alkylator | CAD | IV | 71% |
| Ofatumumab | Anti-CD20 | Secondary AIHA | IV | Case report |
| Alemtuzumab | Anti-CD52 | Secondary AIHA | SC | Case reports |
| Daratumumab | Anti-CD38 | Secondary AIHA | IV | Case reports |
| B-cell receptor inhibitors | ||||
| Ibrutinib | BTKi | Secondary AIHA | Oral | Case reports |
| Parsaclisib | PI3Ki | Primary wAIHA/CAD | Oral | Not available |
| Venetoclax | Bcl2 | Secondary AIHA | Oral | Case reports |
| Proteasome inhibitor | ||||
| Bortezomib | Proteasome inhibitor | CAD/Secondary AIHA | IV | Case reports |
| Bortezomib | Proteasome inhibitor | CAD | IV | 31.6% |
| Complement inhibitors | ||||
| Eculizumab | C5i | CAD/Mixed AIHA | IV | Case reports |
| Sutimlimab | Anti-C1s MoAb | CAD | IV | 50% |
| APL-2 | C3/C3bi | CAD/wAIHA | SC | 50/40% |
| T-cell directed therapies | ||||
| Soluble IL-2 | T-reg stimulation | wAIHA | SC | Not available |
| Sirolimus | mTORi | Evans'/Secondary AIHA | Oral | 80% |
| Mycophenolate Mofetil | Purine synthesis inhibitor | wAIHA/CAD/Secondary AIHA/Evans' | Oral | 81–100% |
| IgG mediated phagocytosis inhibitors | ||||
| Fostamatinib | Syki | wAIHA | Oral | 44% |
| SYNT001 | FcRn MoAb | wAIHA | IV | Not available |
| M281 | FcRn MoAb | wAIHA | IV | Not available |
CTX, cyclophosphamide; BTKi, Bruton tyrosine kinase inhibitor; Bcl2, B-cell lymphoma 2; δPI3Ki, phosphatidylinositol-4,5-bisphosphate 3-kinase delta type inhibitor; MoAb, monoclonal antibody; mTORi, mammalian Target Of Rapamycin inhibitor; Syki, Spleen tyrosine kinase; FcRn, neonatal crystallizable fragment receptor.