Version May 2024
| Drug | Mechanism of action | Use in immunodeficiency |
| Abatacept | CTLA-Ig, competitive inhibitor of CD28-B7 thereby preventing secondary activation of T cells. | CTLA-4 haploinsufficiency, LRBA deficiency |
| Adalimumab | Recombinant human monoclonal antibody to TNF-alpha. | CANDLE syndrome, PAPA syndrome, STING-associated vasculopathy with onset in infancy, Blau syndrome, POMP deficiency |
| Alemtuzumab | Antibody to CD52 that is found on lymphocytes, monocytes, macrophages, NK cells, and some granulocytes resulting in lysis of cells. | HLH, autoimmune cytopenias |
| Anakinra | Recombinant IL-1 receptor agonist. | Cryopyrin-associated periodic fever syndromes |
| Baricitinib | Inhibition of JAK enzymes that when inhibited prevent activation of STATs. | STAT1-GOF, CANDLE syndrome |
| Belatacept | Blocks T cell costimulation by binding CD80 and CD86 on APCs. | CTLA-4 haploinsufficiency |
| Canakinumab | Binds IL-1-beta which prevents its interaction with surface receptors. | Deficiency of IL-1 receptor antagonist |
| Corticosteroids | Suppression of migration of polymorphonuclear leukocytes and reversal of increased capillary permeability; suppresses the immune system by reducing activity and volume of the lymphatic system. | CGD (granulomatous stigmata) |
| Cyclosporine | Prevents activation of resting T-lymphocytes by blocking IL-2. Inhibits production of IL-2. | HLH, autoimmune cytopenias |
| Eculizumab | Humanized monoclonal antibody targeting C5. | Complement deficiencies |
| Emapalumab | Monoclonal antibody to IFN-gamma. | HLH |
| Etanercept | Recombinant TNF receptor which prevents TNF from binding to surface receptors. | STING-associated vasculopathy with onset in infancy, CANDLE syndrome, POMP deficiency, PAPA syndrome, Blau syndrome |
| Infliximab | Chimeric monoclonal antibody which binds to TNF-alpha interfering with TNF-alpha activity. | STING-associated vasculopathy with onset in infancy, CANDLE syndrome, POMP deficiency, PAPA syndrome, Blau syndrome |
| Leniolisib | A selective inhibitor of PI3K-delta. | Activated phosphoinositide 3-kinase delta syndrome |
| Mycophenolate mofetil | Inhibition of inosine monophosphate dehydrogenase that inhibits nucleotide synthesis that has cytostatic effects on T and B lymphocytes. | Autoimmune lymphoproliferative disorders |
| Rilonacept | Reduces inflammation by binding to IL-1-beta (some binding of IL-1-alpha and IL-1 receptor antagonist) and preventing interaction with cell surface receptors. | Deficiency of IL-1 receptor antagonist |
| Rituximab | Monoclonal antibody to CD20 which is found on B cells and regulates cell cycle initiation. | Autoimmune cytopenias (commonly in CVID) |
| Ruxolitinib | Selective inhibitor of JAK1 and JAK2. | STAT3-GOF, STAT1-GOF, CANDLE syndrome |
| Sirolimus | Modulates mTOR by binding FK protein, which as a result inhibits IL-2 mediated signal transduction. | NLCR4-GOF, POMP deficiency, CTLA-4 haploinsufficiency, activated phosphoinositide 3-kinase delta syndrome |
| Tadekinig-alfa | IL-18 binding protein as IL-18 is proinflammatory cytokine. | NLCR4-GOF |
| Tocilizumab | IL-6 receptor antagonist. | STAT3-GOF |
| Ustekinumab | Human monoclonal antibody which binds to proinflammatory cytokines IL-12 and IL-23. | Activated phosphoinositide 3-kinase delta syndrome |
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