Drugs

All drugs and treatments tracked across rare diseases on Kipine, with evidence-linked profiles and cross-disease comparisons.

55 drugs tracked

Drug5 diseases

Anakinra

IL-1 receptor antagonist

Adult-Onset Still's Disease · ~80-90% · 2nd lineCryopyrin-Associated Periodic Syndromes · Sustained efficacy up to 5 years · 1st lineFamilial Mediterranean Fever · 76.5% complete response · 2nd lineSchnitzler Syndrome · ~94% · 1st lineTNF Receptor-Associated Periodic Syndrome · ~90% (complete ~67%) · 1st line
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Drug5 diseases

Canakinumab

Anti-IL-1beta monoclonal antibody

Adult-Onset Still's Disease · ~67% · 2nd lineCryopyrin-Associated Periodic Syndromes · 78-97% complete response · 1st lineFamilial Mediterranean Fever · 67.5% complete response · 2nd lineSchnitzler Syndrome · High (RCT confirmed) · 2nd lineTNF Receptor-Associated Periodic Syndrome · 45% complete (Phase III); >94% disease control (long-term) · 1st line
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Drug4 diseases

Tocilizumab

Anti-IL-6 receptor monoclonal antibody

Adult-Onset Still's Disease · ~61% (ACR50) · 2nd lineFamilial Mediterranean Fever · Under investigation · Investigational lineSchnitzler Syndrome · Initial response, loss of efficacy · Alternative lineTNF Receptor-Associated Periodic Syndrome · Case reports of efficacy · Alternative line
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Drug3 diseases

Colchicine

Microtubule disruption; reduces leukocyte motility and phagocytosis

Familial Mediterranean Fever · ~95% (attack prevention) · 1st lineHypocomplementemic Urticarial Vasculitis Syndrome · Variable; partial responses reported · 1st lineTNF Receptor-Associated Periodic Syndrome · 12.5% complete response · Alternative line
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Drug3 diseases

Corticosteroids

Broad anti-inflammatory

Cryopyrin-Associated Periodic Syndromes · Partial, temporary · Supportive lineSchnitzler Syndrome · Temporary, partial · Not recommended lineTNF Receptor-Associated Periodic Syndrome · Effective for acute attacks; not for long-term · Alternative line
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Drug3 diseases

Rilonacept

IL-1 decoy receptor (IL-1 Trap)

Cryopyrin-Associated Periodic Syndromes · Significant improvement vs placebo · 2nd lineFamilial Mediterranean Fever · Moderate · 3rd lineSchnitzler Syndrome · Limited data · Alternative line
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Drug3 diseases

Rituximab

Anti-CD20 monoclonal antibody; depletes B cells

Hypocomplementemic Urticarial Vasculitis Syndrome · Promising in refractory cases · 3rd lineSchnitzler Syndrome · Ineffective on inflammation · Not recommended lineWaldenström's Macroglobulinemia · ~25-40% (monotherapy); higher in combinations · 1st line
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Drug2 diseases

Corticosteroids (Prednisone)

Broad immunosuppression via NF-kB inhibition and anti-inflammatory gene transcription

Adult-Onset Still's Disease · ~60% · 1st lineHypocomplementemic Urticarial Vasculitis Syndrome · Initial response in most, frequent relapse on taper · 1st line
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Drug2 diseases

Ibrutinib

BTK inhibitor

Schnitzler Syndrome · Complete in case reports · Alternative lineWaldenström's Macroglobulinemia · 90% ORR; 73% MRR (previously treated) · 1st line
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Drug2 diseases

NSAIDs

Cyclooxygenase inhibition; anti-inflammatory and antipyretic

Adult-Onset Still's Disease · <20% · 1st lineCryopyrin-Associated Periodic Syndromes · Symptomatic only · Supportive line
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Drug2 diseases

Omalizumab

Anti-IgE monoclonal antibody

Chronic Rhinosinusitis with Nasal Polyps · Significant NPS reduction · 1st-line biologic lineHypocomplementemic Urticarial Vasculitis Syndrome · Case reports suggest efficacy for urticarial symptoms · Investigational line
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Drug

Aspirin desensitisation

COX-1 tolerance induction

Chronic Rhinosinusitis with Nasal Polyps · Effective in AERD · AERD-specific line
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Autologous Stem Cell Transplant (ASCT)

High-dose chemotherapy followed by stem cell rescue — eradicates plasma cell clone

POEMS Syndrome · 5-year PFS 75%; neurologic improvement near-universal · 1st line
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Avapritinib (Ayvakit)

Selective type 1 KIT D816V inhibitor

Systemic Mastocytosis · PIONEER: significant symptom reduction vs placebo; AdvSM: 30% molecular response · 1st line
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Bendamustine + Rituximab (BR)

Alkylating agent + anti-CD20 monoclonal antibody

Waldenström's Macroglobulinemia · 91% (PR or better, frontline); 74% (relapsed) · 1st line
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Benralizumab

Anti–IL-5Rα monoclonal antibody (ADCC-mediated eosinophil depletion)

Chronic Rhinosinusitis with Nasal Polyps · Near-complete eosinophil depletion · 1st-line biologic line
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Bevacizumab (anti-VEGF)

Monoclonal antibody targeting VEGF — blocks VEGF-mediated vascular permeability

POEMS Syndrome · Ambiguous — VEGF drops but clinical benefit inconsistent; deaths reported · Not recommended line
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Bisphosphonates / Denosumab

Antiresorptive — inhibit osteoclast-mediated bone resorption

Systemic Mastocytosis · Effective for osteoporosis management · 1st line
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Bortezomib

Proteasome inhibitor (NF-κB pathway)

Schnitzler Syndrome · Single case report · Investigational line
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Bortezomib (BDR regimen)

Proteasome inhibitor (with dexamethasone + rituximab)

Waldenström's Macroglobulinemia · ~80-85% (frontline BDR) · 1st line
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Bortezomib + Dexamethasone (BDex)

Proteasome inhibitor — disrupts NF-kB signaling, induces plasma cell apoptosis

POEMS Syndrome · Neurologic response 88%, hematologic CR 46%, VEGF response 71% · 1st line
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Cladribine (2-CdA)

Purine nucleoside analog — cytoreductive chemotherapy

Systemic Mastocytosis · 50-60% in advanced SM · 2nd line
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Cromolyn sodium

Mast cell membrane stabilizer — reduces degranulation

Systemic Mastocytosis · Moderate for GI symptoms · 2nd line
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Drug

Cyclophosphamide

Alkylating agent; potent immunosuppression, depletes B and T cells

Hypocomplementemic Urticarial Vasculitis Syndrome · Effective for severe organ involvement · 3rd line
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Cyclosporine A

Calcineurin inhibitor; suppresses T-cell activation and cytokine production

Adult-Onset Still's Disease · Variable · Alternative line
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Dapansutrile

Oral NLRP3 inflammasome inhibitor

Cryopyrin-Associated Periodic Syndromes · Phase 2a (no CAPS efficacy data yet) · Experimental line
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Dapsone

Anti-neutrophilic; inhibits myeloperoxidase and neutrophil chemotaxis

Hypocomplementemic Urticarial Vasculitis Syndrome · Effective for cutaneous symptoms in most cases · 2nd line
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Dupilumab

Anti–IL-4Rα monoclonal antibody (blocks IL-4 and IL-13)

Chronic Rhinosinusitis with Nasal Polyps · ~75% polyp reduction · 1st-line biologic line
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Epinephrine autoinjector

Adrenergic agonist — emergency anaphylaxis treatment

Systemic Mastocytosis · Life-saving in anaphylaxis · Alternative line
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Drug

Etanercept

Soluble TNF receptor fusion protein (TNFR2-Fc)

TNF Receptor-Associated Periodic Syndrome · Partial, waning over time · Alternative line
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Drug

Functional endoscopic sinus surgery (FESS)

Surgical polyp removal and sinus drainage

Chronic Rhinosinusitis with Nasal Polyps · Immediate symptom relief · 1st (surgical) line
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H1 Antihistamines (cetirizine, loratadine, levocetirizine)

H1 receptor antagonist — blocks histamine-mediated skin and vascular symptoms

Systemic Mastocytosis · Variable; mainstay of symptomatic therapy · 1st line
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H2 Antihistamines (famotidine)

H2 receptor antagonist — blocks histamine-mediated gastric acid secretion

Systemic Mastocytosis · Effective for GI symptoms · 1st line
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Hydroxychloroquine

Antimalarial; modulates immune response, inhibits TLR signalling

Hypocomplementemic Urticarial Vasculitis Syndrome · ~57% response rate (UV data) · 1st line
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Ibrutinib + Rituximab

BTK inhibitor + anti-CD20 antibody combination

Waldenström's Macroglobulinemia · 82% 30-month PFS (vs 28% rituximab alone) · 1st line
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Imatinib (Gleevec)

Tyrosine kinase inhibitor (KIT, BCR-ABL, PDGFR)

Systemic Mastocytosis · Effective in non-D816V cases; ineffective in D816V+ SM · 1st line
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Infliximab

Anti-TNF monoclonal antibody

TNF Receptor-Associated Periodic Syndrome · Paradoxical worsening reported · Not recommended line
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Intranasal corticosteroids

Topical anti-inflammatory

Chronic Rhinosinusitis with Nasal Polyps · Modest polyp reduction · 1st line
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Lenalidomide + Dexamethasone

Immunomodulatory drug (IMiD) — inhibits IL-6, TNF-alpha, and VEGF; stimulates T-cell immunity; direct anti-plasma-cell effect via cereblon-mediated degradation of Ikaros/Aiolos

POEMS Syndrome · Hematologic CR 46%, neurologic response 95%, VEGF response 83% · 1st line
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Leukotriene receptor antagonists

CysLT1 receptor blockade (montelukast)

Chronic Rhinosinusitis with Nasal Polyps · Modest in AERD subtype · Adjunctive line
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Melphalan + Dexamethasone (MDex)

Alkylating agent — directly cytotoxic to plasma cells; dexamethasone adds anti-inflammatory and anti-neoplastic effect

POEMS Syndrome · Hematologic response 81% (CR 39%), neurologic response 100% · 1st line
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Mepolizumab

Anti–IL-5 monoclonal antibody

Chronic Rhinosinusitis with Nasal Polyps · Significant NPS reduction · 1st-line biologic line
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Methotrexate

Folate antagonist; anti-inflammatory via adenosine pathway modulation

Adult-Onset Still's Disease · Up to 70% · 2nd line
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Midostaurin (Rydapt)

Multikinase inhibitor (KIT, FLT3, PDGFR, PKC)

Systemic Mastocytosis · 60% ORR in AdvSM; 45% major response · 1st line
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Mycophenolate mofetil

IMPDH inhibitor; suppresses lymphocyte proliferation

Hypocomplementemic Urticarial Vasculitis Syndrome · Effective for maintenance after induction · 2nd line
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Omalizumab (Xolair)

Anti-IgE monoclonal antibody

Systemic Mastocytosis · Variable; effective for refractory anaphylaxis · 3rd line
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Oral corticosteroids (short course)

Systemic anti-inflammatory

Chronic Rhinosinusitis with Nasal Polyps · Rapid polyp shrinkage · Rescue line
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Pirtobrutinib

Non-covalent (reversible) BTK inhibitor

Waldenström's Macroglobulinemia · ~68% ORR (after covalent BTKi); 56% VGPR with venetoclax combo · 2nd line
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Plasmapheresis

Physical removal of IgM from circulation

Waldenström's Macroglobulinemia · Immediate symptomatic relief · Adjunct line
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Drug

Radiation Therapy

Targeted irradiation of solitary or limited (1-3) plasmacytomas

POEMS Syndrome · 97% OS at 4 years · 1st line
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Saline nasal irrigation

Mechanical clearance / mucosal hydration

Chronic Rhinosinusitis with Nasal Polyps · Symptom improvement · Adjunctive line
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Thalidomide + Dexamethasone

Immunomodulatory drug (IMiD) — anti-angiogenic, anti-proliferative, anti-cytokine properties

POEMS Syndrome · VEGF reduction rate 0.39 (J-POST RCT) · 1st line
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Tofacitinib

JAK inhibitor (JAK1/JAK3)

Familial Mediterranean Fever · Promising (case series only) · Investigational line
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Venetoclax

BCL2 inhibitor

Waldenström's Macroglobulinemia · ~70% MRR (relapsed/refractory) · 2nd line
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Zanubrutinib

Second-generation selective BTK inhibitor

Waldenström's Macroglobulinemia · ~95% ORR; 36% VGPR+CR · 1st line
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