Biomarker & Intervention Discovery

Immune Thrombocytopaenia (ITP)

This page lists 10 druggable gene targets, 57 approved or trial-tested drug candidates and 15 recent clinical trials for Immune Thrombocytopaenia (ITP), with each agent ranked by the strength of its evidence (clinical, mechanistic or correlative). Candidates include existing drugs that could be repurposed. Sources are linked so you can check every claim.

Spontaneous remissionChildhood ITP: spontaneous remission in 70-80% within 6 months; Adult ITP: spontaneous remission in 20-30% at 1 year…
Best-intervention remissionRituximab: 40-60% initial response, 20-25% durable at 5 years; thrombopoietin receptor agonists (TPO-RA: eltrombopag…
Gap sizeModerate — TPO-RAs are highly effective but chronic use required in most; splenectomy (durable cure in 65%) is…
Primary barrierpractice
Full remission profile →
Research Tracker Chronic Disease Interventions Immune Thrombocytopaenia (ITP)

Remission & chronicity

Spontaneous remission
Childhood ITP: spontaneous remission in 70-80% within 6 months; Adult ITP: spontaneous remission in 20-30% at 1 year without treatment; chronic ITP (>12 months) rarely resolves spontaneously
Best-intervention remission
Rituximab: 40-60% initial response, 20-25% durable at 5 years; thrombopoietin receptor agonists (TPO-RA: eltrombopag, romiplostim): 70-90% response rate; splenectomy: 60-70% long-term complete response; fostamatinib: 18% CR in refractory; avatrombopag and lusutrombopag in perioperative settings
Gap size
Moderate — TPO-RAs are highly effective but chronic use required in most; splenectomy (durable cure in 65%) is underused in appropriate patients
Primary barrier
practice
Splenectomy offers durable cure in 65% of ITP patients but is increasingly deferred in favour of chronic TPO-RA therapy which requires lifelong administration; the optimal sequence (corticosteroids → rituximab → TPO-RA → splenectomy) is debated; overtreated mild cases (platelet >30K without bleeding) remain a clinical challenge

Biomarker Targets & Therapeutic Agents

Gene targets queried against DGIdb, Open Targets, ChEMBL, PubMed, and Europe PMC. Agents ranked: Clinical > Mechanistic > Correlative.

BTK 254FCGR2B 12FCGR3A 51FCGRT 120GP1BA 1ITGA2B 52MPL 180MS4A1 28NR3C1 264SYK 216

BTK

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
PRN694 ↓ inhibits Mechanistic DGIdb
RN486 ↓ inhibits Mechanistic DGIdb
PD166285 ? unclear Mechanistic DGIdb
PTD10 ↓ inhibits Mechanistic DGIdb
BRANEBRUTINIB ↓ inhibits Mechanistic DGIdb
ACALABRUTINIB ↓ inhibits Mechanistic DGIdb
COMPOUND 71 [WO2018191577A1] ↓ inhibits Mechanistic DGIdb
COMPOUND 25 [PMID: 31260299] ↓ inhibits Mechanistic DGIdb
COMPOUND 38 [PMID: 24915291] ↓ inhibits Mechanistic DGIdb
IBRUTINIB ↓ inhibits Mechanistic 0.5 nM (IC50) DGIdb
ZM447439 ? unclear Mechanistic DGIdb
BTK INHIBITOR TL-895 ? unclear Mechanistic DGIdb
ZYBT1 ↓ inhibits Mechanistic DGIdb
EVOBRUTINIB ↓ inhibits Mechanistic DGIdb
FENEBRUTINIB ↓ inhibits Mechanistic DGIdb
TOLEBRUTINIB ↓ inhibits Mechanistic DGIdb
RILZABRUTINIB ? unclear Mechanistic DGIdb
CGI1746 ↓ inhibits Mechanistic DGIdb
TAMATINIB ? unclear Mechanistic DGIdb
ZANUBRUTINIB ↓ inhibits Mechanistic DGIdb
ENTRECTINIB ? unclear Mechanistic 158.49 nM (Ki) DGIdb
LUXEPTINIB ↓ inhibits Mechanistic DGIdb
BTK INHIBITOR 16 [PMID: 30122225] ↓ inhibits Mechanistic DGIdb
ZELEBRUDOMIDE ? unclear Mechanistic DGIdb
BIIB068 ↓ inhibits Mechanistic DGIdb
PROTAC 23 [PMID: 38321747] ↓ inhibits Mechanistic DGIdb
NVP-TAE684 ? unclear Mechanistic DGIdb
COMPOUND 36 [PMID: 21958547] ↓ inhibits Mechanistic DGIdb
CINSEBRUTINIB ↓ inhibits Mechanistic DGIdb
COMPOUND 9 [PMID: 26006010] ↓ inhibits Mechanistic DGIdb
DASATINIB ANHYDROUS ? unclear Mechanistic DGIdb
NEMTABRUTINIB ? unclear Mechanistic DGIdb
JNJ-64264681 ↓ inhibits Mechanistic DGIdb
SPEBRUTINIB ↓ inhibits Mechanistic DGIdb
CHEMBL:CHEMBL1997335 ? unclear Mechanistic DGIdb
RITLECITINIB ↓ inhibits Mechanistic DGIdb
BIRELENTINIB ↓ inhibits Mechanistic DGIdb
PF-562271 ? unclear Mechanistic DGIdb
ORELABRUTINIB ↓ inhibits Mechanistic DGIdb
MIDOBRUTINIB ↓ inhibits Mechanistic DGIdb

Showing top 40 of 254 agents ranked by evidence tier.

FCGR2B

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
RECOMBINANT TRANSFORMING GROWTH FACTOR ? unclear Mechanistic DGIdb
MARGETUXIMAB ? unclear Mechanistic DGIdb
INFLIXIMAB ? unclear Mechanistic DGIdb
OBEXELIMAB ? unclear Mechanistic DGIdb
RECOMBINANT INTERLEUKIN-5 ? unclear Mechanistic DGIdb
ETANERCEPT ? unclear Mechanistic DGIdb
RECOMBINANT INTERLEUKIN-2 ? unclear Mechanistic DGIdb
ADALIMUMAB ? unclear Mechanistic DGIdb
MGD-010 ? unclear Mechanistic DGIdb
ANTI-THYMOCYTE GLOBULIN ? unclear Mechanistic DGIdb
TNF-ALPHA ? unclear Mechanistic DGIdb
VALZIFLOCEPT ? unclear Mechanistic DGIdb

FCGR3A

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
PREDNISOLONE ? unclear Mechanistic DGIdb
AFM-13 ? unclear Mechanistic DGIdb
BEMARITUZUMAB ~ modulates Mechanistic DGIdb
RITUXIMAB ? unclear Mechanistic DGIdb
IBOCTADEKIN ? unclear Mechanistic DGIdb
IMMUNOTHERAPEUTIC AGENT ? unclear Mechanistic DGIdb
CIMETIDINE ? unclear Mechanistic DGIdb
PICIBANIL ? unclear Mechanistic DGIdb
TETRADECANOYLPHORBOL ACETATE ? unclear Mechanistic DGIdb
ANGIOGENESIS INHIBITOR ? unclear Mechanistic DGIdb
THALIDOMIDE ? unclear Mechanistic DGIdb
DOXORUBICIN HYDROCHLORIDE ? unclear Mechanistic DGIdb
MUROMONAB-CD3 ? unclear Mechanistic DGIdb
CA19-9 ANTIGEN ? unclear Mechanistic DGIdb
PROTEASE INHIBITOR ? unclear Mechanistic DGIdb
CHONDROITIN SULFATES ? unclear Mechanistic DGIdb
EMFILERMIN ? unclear Mechanistic DGIdb
VINCRISTINE ? unclear Mechanistic DGIdb
RECOMBINANT FLT3 LIGAND ? unclear Mechanistic DGIdb
POLY IC ? unclear Mechanistic DGIdb
FENTANYL CITRATE ? unclear Mechanistic DGIdb
INDOMETHACIN ? unclear Mechanistic DGIdb
TRASTUZUMAB ? unclear Mechanistic DGIdb
IMMUNOSUPPRESSANT ? unclear Mechanistic DGIdb
IMGATUZUMAB ~ modulates Mechanistic DGIdb
MATRIX METALLOPROTEINASE INHIBITOR ? unclear Mechanistic DGIdb
RECOMBINANT INTERLEUKIN-2 ? unclear Mechanistic DGIdb
GMA-161 ? unclear Mechanistic DGIdb
DIMETHYL SULFOXIDE ? unclear Mechanistic DGIdb
RECOMBINANT EOTAXIN ? unclear Mechanistic DGIdb
AFM13 ? unclear Mechanistic DGIdb
UBENIMEX ? unclear Mechanistic DGIdb
LACTULOSE ? unclear Mechanistic DGIdb
PUROMYCIN ? unclear Mechanistic DGIdb
HEPARIN ? unclear Mechanistic DGIdb
CYCLOPHOSPHAMIDE ANHYDROUS ? unclear Mechanistic DGIdb
TOCILIZUMAB ? unclear Mechanistic DGIdb
RECOMBINANT INTERLEUKIN-8 ? unclear Mechanistic DGIdb
ANTI-THYMOCYTE GLOBULIN ? unclear Mechanistic DGIdb
HYDROGEN PEROXIDE ? unclear Mechanistic DGIdb

Showing top 40 of 51 agents ranked by evidence tier.

FCGRT

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
EFGARTIGIMOD ALFA ↓ inhibits Mechanistic DGIdb
BATOCLIMAB ? unclear Mechanistic DGIdb
DX-2507 ? unclear Mechanistic DGIdb
ZILTIVEKIMAB ? unclear Mechanistic DGIdb
NIPOCALIMAB ↓ inhibits Mechanistic DGIdb
ROZANOLIXIZUMAB ↓ inhibits Mechanistic DGIdb
ORILANOLIMAB ↓ inhibits Mechanistic DGIdb
CHEMBL508564 ↓ inhibits Mechanistic 1400.0 nM (IC50) ChEMBL
CHEMBL508411 ↓ inhibits Mechanistic 36000.0 nM (IC50) ChEMBL
CHEMBL505689 ↓ inhibits Mechanistic 87000.0 nM (IC50) ChEMBL
CHEMBL524509 ↓ inhibits Mechanistic 31000.0 nM (IC50) ChEMBL
CHEMBL503730 ↓ inhibits Mechanistic 34000.0 nM (IC50) ChEMBL
CHEMBL526683 ↓ inhibits Mechanistic 67000.0 nM (IC50) ChEMBL
CHEMBL207341 ↓ inhibits Mechanistic 24000.0 nM (IC50) ChEMBL
CHEMBL451944 ↓ inhibits Mechanistic 27000.0 nM (IC50) ChEMBL
CHEMBL506507 ↓ inhibits Mechanistic 40000.0 nM (IC50) ChEMBL
CHEMBL509774 ↓ inhibits Mechanistic 33000.0 nM (IC50) ChEMBL
PSYCIEGHIDGIYCFNA ↓ inhibits Mechanistic 49000.0 nM (IC50) ChEMBL
CHEMBL503219 ↓ inhibits Mechanistic 64000.0 nM (IC50) ChEMBL
CHEMBL499790 ↓ inhibits Mechanistic 33000.0 nM (IC50) ChEMBL
CHEMBL444514 ↓ inhibits Mechanistic 20000.0 nM (Kd) ChEMBL
CHEMBL525216 ↓ inhibits Mechanistic 34000.0 nM (Kd) ChEMBL
CHEMBL499664 ↓ inhibits Mechanistic 69000.0 nM (IC50) ChEMBL
CHEMBL499402 ↓ inhibits Mechanistic 57000.0 nM (IC50) ChEMBL
CHEMBL506644 ↓ inhibits Mechanistic 48000.0 nM (IC50) ChEMBL
CHEMBL525265 ↓ inhibits Mechanistic 420.0 nM (IC50) ChEMBL
CHEMBL503983 ↓ inhibits Mechanistic 1000.0 nM (IC50) ChEMBL
CHEMBL504423 ↓ inhibits Mechanistic 2100.0 nM (IC50) ChEMBL
CHEMBL525614 ↓ inhibits Mechanistic 1600.0 nM (IC50) ChEMBL
CHEMBL505899 ↓ inhibits Mechanistic 900.0 nM (IC50) ChEMBL
CHEMBL511030 ↓ inhibits Mechanistic 27000.0 nM (IC50) ChEMBL
CHEMBL446284 ↓ inhibits Mechanistic 2000.0 nM (IC50) ChEMBL
CHEMBL510716 ↓ inhibits Mechanistic 1700.0 nM (IC50) ChEMBL
CHEMBL504919 ↓ inhibits Mechanistic 16000.0 nM (IC50) ChEMBL
CHEMBL499766 ↓ inhibits Mechanistic 2000.0 nM (IC50) ChEMBL
CHEMBL524659 ↓ inhibits Mechanistic 2000.0 nM (IC50) ChEMBL
CHEMBL526861 ↓ inhibits Mechanistic 18000.0 nM (IC50) ChEMBL
CHEMBL506647 ↓ inhibits Mechanistic 2000.0 nM (IC50) ChEMBL
CHEMBL504226 ↓ inhibits Mechanistic 20000.0 nM (IC50) ChEMBL
CHEMBL507004 ↓ inhibits Mechanistic 21000.0 nM (IC50) ChEMBL

Showing top 40 of 120 agents ranked by evidence tier.

GP1BA

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
ANFIBATIDE ↓ inhibits Mechanistic DGIdb

ITGA2B

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
EPTIFIBATIDE ↓ inhibits Mechanistic DGIdb
GANTOFIBAN ? unclear Mechanistic DGIdb
ASPIRIN ? unclear Mechanistic DGIdb
TIROFIBAN ? unclear Mechanistic DGIdb
ALTEPLASE ? unclear Mechanistic DGIdb
THROMBIN ALFA ? unclear Mechanistic DGIdb
TIROFIBAN HYDROCHLORIDE ↓ inhibits Mechanistic DGIdb
TADOCIZUMAB ↓ inhibits Mechanistic DGIdb
ANGIOGENESIS INHIBITOR ? unclear Mechanistic DGIdb
ZALUNFIBAN ↓ inhibits Mechanistic DGIdb
ABCIXIMAB ↓ inhibits Mechanistic DGIdb
ENOXAPARIN ? unclear Mechanistic DGIdb
CHEMBL1314113 ~ modulates Mechanistic ChEMBL
CHEMBL1514487 ~ modulates Mechanistic ChEMBL
CHEMBL1354212 ~ modulates Mechanistic ChEMBL
CHEMBL1511944 ~ modulates Mechanistic ChEMBL
CHEMBL1512960 ~ modulates Mechanistic ChEMBL
CHEMBL1234739 ~ modulates Mechanistic ChEMBL
CHEMBL1515059 ~ modulates Mechanistic ChEMBL
CHEMBL1589758 ~ modulates Mechanistic ChEMBL
CHEMBL1393970 ~ modulates Mechanistic ChEMBL
CHEMBL1314095 ~ modulates Mechanistic ChEMBL
CHEMBL1515323 ~ modulates Mechanistic ChEMBL
CHEMBL1395650 ~ modulates Mechanistic ChEMBL
CHEMBL1589764 ~ modulates Mechanistic ChEMBL
CHEMBL1314120 ~ modulates Mechanistic ChEMBL
CHEMBL1472460 ~ modulates Mechanistic ChEMBL
CHEMBL1354401 ~ modulates Mechanistic ChEMBL
CHEMBL1398179 ~ modulates Mechanistic ChEMBL
CHEMBL1354140 ~ modulates Mechanistic ChEMBL
CHEMBL1473296 ~ modulates Mechanistic ChEMBL
CHEMBL1314464 ~ modulates Mechanistic ChEMBL
CHEMBL4108850 ↓ inhibits Mechanistic 101.0 nM (IC50) ChEMBL
CHEMBL4112949 ↓ inhibits Mechanistic 29.0 nM (IC50) ChEMBL
CHEMBL4113538 ↓ inhibits Mechanistic 20.0 nM (IC50) ChEMBL
CHEMBL4111249 ↓ inhibits Mechanistic 84.0 nM (IC50) ChEMBL
CHEMBL4110076 ↓ inhibits Mechanistic 16.0 nM (IC50) ChEMBL
CHEMBL4107531 ↓ inhibits Mechanistic 16.0 nM (IC50) ChEMBL
CHEMBL4114919 ↓ inhibits Mechanistic 11.0 nM (IC50) ChEMBL
CHEMBL4115273 ↓ inhibits Mechanistic 14.0 nM (IC50) ChEMBL

Showing top 40 of 52 agents ranked by evidence tier.

MPL

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
HETROMBOPAG OLAMINE ↑ activates Mechanistic DGIdb
HETROMBOPAG ↑ activates Mechanistic DGIdb
AVATROMBOPAG ↑ activates Mechanistic DGIdb
ELTROMBOPAG ↑ activates Mechanistic 38.0 nM (EC50) DGIdb
ROMIPLOSTIM ↑ activates Mechanistic DGIdb
THROMBOPOIETIN ↑ activates Mechanistic DGIdb
AVATROMBOPAG MALEATE ↑ activates Mechanistic DGIdb
ELTROMBOPAG OLAMINE ↑ activates Mechanistic DGIdb
RAFUTROMBOPAG ↑ activates Mechanistic DGIdb
ELTROMBOPAG CHOLINE ↑ activates Mechanistic DGIdb
LUSUTROMBOPAG ↑ activates Mechanistic DGIdb
TOTROMBOPAG ? unclear Mechanistic DGIdb
CHEMBL3144682 ↑ activates Mechanistic 400.0 nM (EC50) ChEMBL
CHEMBL125996 ↑ activates Mechanistic 2000.0 nM (EC50) ChEMBL
CHEMBL3144783 ↑ activates Mechanistic 70.0 nM (EC50) ChEMBL
CHEMBL122504 ↑ activates Mechanistic 2000.0 nM (EC50) ChEMBL
CHEMBL3144667 ↑ activates Mechanistic 10000.0 nM (EC50) ChEMBL
CHEMBL3144785 ↑ activates Mechanistic 2000.0 nM (EC50) ChEMBL
CHEMBL3144672 ↑ activates Mechanistic 200.0 nM (EC50) ChEMBL
CHEMBL3144775 ↑ activates Mechanistic 20.0 nM (EC50) ChEMBL
CHEMBL340040 ↑ activates Mechanistic 3000.0 nM (EC50) ChEMBL
CHEMBL3144788 ↑ activates Mechanistic 3000.0 nM (EC50) ChEMBL
CHEMBL3144786 ↑ activates Mechanistic 700.0 nM (EC50) ChEMBL
CHEMBL3144789 ↑ activates Mechanistic 2000.0 nM (EC50) ChEMBL
CHEMBL124367 ↑ activates Mechanistic 200.0 nM (EC50) ChEMBL
CHEMBL3144776 ↑ activates Mechanistic 500.0 nM (EC50) ChEMBL
CHEMBL3144677 ↑ activates Mechanistic 1000.0 nM (EC50) ChEMBL
CHEMBL121918 ↑ activates Mechanistic 600.0 nM (EC50) ChEMBL
CHEMBL3144778 ↑ activates Mechanistic 70.0 nM (EC50) ChEMBL
CHEMBL3144665 ↑ activates Mechanistic 10000.0 nM (EC50) ChEMBL
CHEMBL3144780 ↑ activates Mechanistic 2000.0 nM (EC50) ChEMBL
CHEMBL3144666 ↑ activates Mechanistic 100.0 nM (EC50) ChEMBL
CHEMBL3144790 ↑ activates Mechanistic 900.0 nM (EC50) ChEMBL
CHEMBL3144676 ↑ activates Mechanistic 7000.0 nM (EC50) ChEMBL
CHEMBL3144836 ↑ activates Mechanistic 8000.0 nM (EC50) ChEMBL
CHEMBL3144675 ↑ activates Mechanistic 5000.0 nM (EC50) ChEMBL
CHEMBL3144787 ↑ activates Mechanistic 2000.0 nM (EC50) ChEMBL
CHEMBL3144670 ↑ activates Mechanistic 300.0 nM (EC50) ChEMBL
CHEMBL3144781 ↑ activates Mechanistic 300.0 nM (EC50) ChEMBL
CHEMBL3144668 ↑ activates Mechanistic 5000.0 nM (EC50) ChEMBL

Showing top 40 of 180 agents ranked by evidence tier.

MS4A1

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
RITUXIMAB ~ modulates Mechanistic DGIdb
UBLITUXIMAB ~ modulates Mechanistic DGIdb
VELTUZUMAB ? unclear Mechanistic DGIdb
OFATUMUMAB ~ modulates Mechanistic DGIdb
OBINUTUZUMAB ~ modulates Mechanistic DGIdb
OCARATUZUMAB ~ modulates Mechanistic DGIdb
TOSITUMOMAB ~ modulates Mechanistic DGIdb
TISAGENLECLEUCEL ? unclear Mechanistic DGIdb
IODINE-131-TOSITUMOMAB ? unclear Mechanistic DGIdb
EPCORITAMAB ? unclear Mechanistic DGIdb
GLOFITAMAB ~ modulates Mechanistic DGIdb
TRU-015 ~ modulates Mechanistic DGIdb
ODRONEXTAMAB ? unclear Mechanistic DGIdb
OCRELIZUMAB ~ modulates Mechanistic DGIdb
ANTI-CD20 CAR T CELLS PREPARATION ? unclear Mechanistic DGIdb
DI-LEU16-IL2 ? unclear Mechanistic DGIdb
MONOCLONAL ANTIBODY 1F5 ? unclear Mechanistic DGIdb
MT-3724 ~ modulates Mechanistic DGIdb
MOSUNETUZUMAB ? unclear Mechanistic DGIdb
PLAMOTAMAB ? unclear Mechanistic DGIdb
AUTOLOGOUS ANTI-CD20 CAR TRANSDUCED CD4/CD8 ENRICHED T-CELLS MB-CART20.1 ? unclear Mechanistic DGIdb
FBT-A05 ~ modulates Mechanistic DGIdb
YTTRIUM Y 90 IBRITUMOMAB TIUXETAN ~ modulates Mechanistic DGIdb
BSAB (CD89-CD20) ? unclear Mechanistic DGIdb
LFB-R603 ~ modulates Mechanistic DGIdb
DIVOZILIMAB ? unclear Mechanistic DGIdb
IBRITUMOMAB TIUXETAN ? unclear Mechanistic DGIdb
TOSITUMOMAB 131I ~ modulates Mechanistic DGIdb

NR3C1

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
METHYLPREDNISOLONE ↑ activates Mechanistic DGIdb
FOSDAGROCORAT ↑ activates Mechanistic DGIdb
ZK216348 ↑ activates Mechanistic DGIdb
PHENYLMERCURIC ACETATE ? unclear Mechanistic DGIdb
TNF-ALPHA ? unclear Mechanistic DGIdb
HYDROCORTAMATE HYDROCHLORIDE ↑ activates Mechanistic DGIdb
TRIAMCINOLONE ↑ activates Mechanistic DGIdb
BUDESONIDE ↑ activates Mechanistic DGIdb
ANTICONVULSANT AGENT ? unclear Mechanistic DGIdb
FLUTICASONE FUROATE ↑ activates Mechanistic DGIdb
METHYLPREDNISOLONE ACETATE ↑ activates Mechanistic DGIdb
DAGROCORAT ↑ activates Mechanistic DGIdb
MIFEPRISTONE ↓ inhibits Mechanistic 0.24 nM (Ki) DGIdb
TESTOSTERONE PROPIONATE ? unclear Mechanistic DGIdb
FLURANDRENOLIDE ↑ activates Mechanistic DGIdb
CHEMBL:CHEMBL1915271 ? unclear Mechanistic DGIdb
CHRYSIN ? unclear Mechanistic DGIdb
FLUOXYMESTERONE ? unclear Mechanistic DGIdb
LOTEPREDNOL ↑ activates Mechanistic DGIdb
DIFLUPREDNATE ↑ activates Mechanistic DGIdb
CORT 108297 ↓ inhibits Mechanistic DGIdb
PROGESTERONE ? unclear Mechanistic 30.5 nM (Ki) DGIdb
BETAMETHASONE SODIUM PHOSPHATE ↑ activates Mechanistic DGIdb
ANTIDEPRESSANT AGENT ? unclear Mechanistic DGIdb
HYDROCORTISONE BUTYRATE ↑ activates Mechanistic DGIdb
VELSECORAT ↑ activates Mechanistic DGIdb
FLUMETHASONE PIVALATE ↑ activates Mechanistic DGIdb
AMCINONIDE ↑ activates Mechanistic DGIdb
CYPROTERONE ACETATE ↓ inhibits Mechanistic DGIdb
C108297 ↑ activates Mechanistic DGIdb
COMPOUND 17 [PMID: 29741897] ↑ activates Mechanistic DGIdb
FLUOCINONIDE ↑ activates Mechanistic DGIdb
MAPRACORAT ↑ activates Mechanistic DGIdb
CLOBETASOL PROPIONATE ↑ activates Mechanistic DGIdb
CHEMBL:CHEMBL210411 ? unclear Mechanistic DGIdb
FLUOCINOLONE ACETONIDE ↑ activates Mechanistic DGIdb
BETAMETHASONE ↑ activates Mechanistic DGIdb
BECLOMETHASONE DIPROPIONATE ↑ activates Mechanistic DGIdb
FLUNISOLIDE ANHYDROUS ↑ activates Mechanistic DGIdb
CORTISONE ACETATE ↑ activates Mechanistic DGIdb

Showing top 40 of 264 agents ranked by evidence tier.

SYK

UniProt Ensembl DGIdb Open Targets
Therapeutic Agent Effect Direction Evidence Tier Potency Source
COMPOUND 23 [PMID: 17600705] ↓ inhibits Mechanistic DGIdb
FOSTAMATINIB DISODIUM ↓ inhibits Mechanistic DGIdb
MIVAVOTINIB CITRATE ? unclear Mechanistic DGIdb
APITOLISIB ↓ inhibits Mechanistic DGIdb
FOSTAMATINIB ↓ inhibits Mechanistic DGIdb
R-343 ↓ inhibits Mechanistic DGIdb
ENTOSPLETINIB ↓ inhibits Mechanistic DGIdb
GUSACITINIB ↓ inhibits Mechanistic DGIdb
R-348 ↓ inhibits Mechanistic DGIdb
CHEMBL:CHEMBL379975 ? unclear Mechanistic DGIdb
GSK2646264 ↓ inhibits Mechanistic DGIdb
CYC-116 ? unclear Mechanistic DGIdb
TAMATINIB ? unclear Mechanistic DGIdb
DASATINIB ANHYDROUS ? unclear Mechanistic DGIdb
SOVLEPLENIB ↓ inhibits Mechanistic DGIdb
R343 ? unclear Mechanistic DGIdb
LANRAPLENIB ↓ inhibits Mechanistic DGIdb
PD166285 ? unclear Mechanistic DGIdb
CHEMBL:CHEMBL535331 ? unclear Mechanistic DGIdb
ADAVOSERTIB ? unclear Mechanistic DGIdb
NVP-TAE684 ? unclear Mechanistic DGIdb
BAY 61-3606 ↓ inhibits Mechanistic DGIdb
TAS05567 ↓ inhibits Mechanistic DGIdb
IMATINIB ? unclear Mechanistic DGIdb
SYK INHIBITOR ↓ inhibits Mechanistic DGIdb
R-333 ↓ inhibits Mechanistic DGIdb
ERLOTINIB ? unclear Mechanistic DGIdb
PRT-2607 ↓ inhibits Mechanistic DGIdb
R-112 ↓ inhibits Mechanistic 300.0 nM (IC50) DGIdb
CENISERTIB ? unclear Mechanistic DGIdb
CERDULATINIB ↓ inhibits Mechanistic DGIdb
SYK INHIBITOR III ↓ inhibits Mechanistic DGIdb
GSK143 ↓ inhibits Mechanistic DGIdb
PACLITAXEL PROTEIN-BOUND ? unclear Mechanistic DGIdb
SP-600125 ? unclear Mechanistic DGIdb
GW441756X ? unclear Mechanistic DGIdb
CEVIDOPLENIB DIMESYLATE ? unclear Mechanistic DGIdb
P505-15 ↓ inhibits Mechanistic DGIdb
COMPOUND 7 [PMID: 22464456] ↓ inhibits Mechanistic DGIdb
SYK INHIBITOR II ↓ inhibits Mechanistic DGIdb

Showing top 40 of 216 agents ranked by evidence tier.

Clinical Trials

Drug, biologic and supplement trials (treatment or prevention) from ClinicalTrials.gov whose main condition is Immune Thrombocytopaenia (ITP): recruiting, active or completed, most recently updated first. Data retrieved 2026-10-28.

A Study to Assess the Tolerability of Ianalumab (VAY736) With Investigator's Choice Thrombopoietin Receptor Agonist (IC TPO-RA) in Patients With Primary Immune Thrombocytopenia (ITP) Recruiting
NCT07421167 Phase: 2 Started: 2026-08-04

The purpose of this study is to investigate the tolerability of ianalumab (9 mg/kg) with investigator's choice thrombopoietin receptor agonist (IC TPO-RA) in participants diagnosed with primary immune thrombocytopenia (ITP) who have been treated with at least one but no more than four prior treatmen...

A Study Investigating Intravenous Human Normal Immunoglobulin 10% in Adults With Chronic Immune Thrombocytopenia (ITP) Recruiting
NCT07059000 Phase: 3 Started: 2025-08-13

The purpose of this study is to evaluate the efficacy and safety of KIg 10 (Intravenous Immunoglobulin 10%) in adult patients with chronic primary ITP

Study of PN20 in Adult Patients With Primary Immune Thrombocytopenia (ITP) Completed
NCT06880081 Phase: 1 Started: 2025-03-07

The main aim of this clinical trial is to assess the safety and tolerability of PN20 in adult patients with primary immune thrombocytopenia (ITP). The main questions it aims to answer are: * Is PN20 safe in these patients? * Could these patients potentially benefit from PN20 treatment? Participants ...

A Study of Ianalumab (VAY736) in Patients With Primary Immune Thrombocytopenia (ITP) Previously Treated With at Least Two Lines of Therapies Active Not Recruiting
NCT05885555 Phase: 2 Started: 2023-08-16

The purpose of this study is to evaluate the therapeutic efficacy, safety and tolerability of ianalumab in adult patients with primary ITP previously treated with at least one corticosteroid and one TPO-RA.

A Phase 3 Study to Evaluate the Safety and Efficacy of Efgartigimod PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia Active Not Recruiting
NCT04812925 Phase: 3 Started: 2021-11-17

The main aim of the study is to evaluate the safety and efficacy of efgartigimod PH20 SC (subcutaneous) in adult patients with primary immune thrombocytopenia (ITP). Participants who completed the 24-week trial period of the ARGX- 113-2004 trial are given the opportunity to enter into this open-labe...

A Study of Efgartigimod IV in Participants From 12 Years to Less Than 18 Years of Age With Chronic Immune Thrombocytopenia (ITP) Recruiting
NCT07194850 Phase: 2/3 Started: 2025-10-20

The main purpose of this study is to confirm the correct dose of efgartigimod IV for treating patients aged 12 to younger than 18 years with chronic immune thrombocytopenia (ITP). The study consists of a double-blinded treatment period (DBTP) in which the participants will be randomized in a 2:1 rat...

Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP) Completed
NCT04562766 Phase: 3 Started: 2020-12-14

This was a randomized, double-blind study of rilzabrutinib in patients with persistent or chronic ITP, with an average platelet count of \<30,000/μL (and no single platelet count \>35,000/μL) on two counts at least 5 days apart in the 14 days before treatment begins. Patients received rilzabrutinib ...

A Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia Recruiting
NCT06722235 Phase: 3 Started: 2025-02-27

Primary immune thrombocytopenia (ITP) is a condition where the immune system mistakenly destroys platelets, which are cells that help stop bleeding. This leads to a low number of platelets, making it easier to bruise or bleed. The main aim of this study is to learn whether mezagitamab, when given ju...

A Study of Romiplostim Plus Predniso(lo)ne vs. Predniso(lo)ne Alone for the Treatment of Previously Untreated Primary Immune Thrombocytopenia (ITP) Recruiting
NCT07700238 Phase: 3 Started: 2026-08-21

This Phase 3 study is designed to evaluate the efficacy and safety of romiplostim in combination with predniso(lo)ne compared with predniso(lo)ne alone in adults with previously untreated Primary Immune Thrombocytopenia (ITP).

Safety, Tolerability, and Efficacy of NVG-2089 in Participants With Immune Thrombocytopenia Recruiting
NCT07095127 Phase: 2 Started: 2025-09-30

The purpose of the study is to evaluate the safety of NVG-2089 and to evaluate how well patients respond to this investigational treatment. NVG-2089 is a new drug that is being developed for treating patients with ITP. NVG-2089 is designed to mimic the effects of a protein called IVIg. NVG-2089 is d...

Study to Assess the Safety and Tolerability of Tafasitamab in Adult Participants With Primary Autoimmune Blood Cell Disorders Recruiting
NCT07104565 Phase: 2 Started: 2025-12-29

This study will evaluate the safety and efficacy of tafasitamab in adult participants with primary autoimmune blood cell disorders.

A Follow-up Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia Recruiting
NCT06948318 Phase: 3 Started: 2025-08-14

Primary immune thrombocytopenia (ITP) is a condition where the immune system mistakenly destroys platelets, which are cells that help stop bleeding. This leads to a lower number of platelets, making it easier to bruise or bleed. The main aim of this study is to check how safe mezagitamab is and how ...

SG301-SC Injection Safety Study in Subjects With Primary ITP Patients Recruiting
NCT07710248 Phase: 2 Started: 2026-06-11

This is a multicenter, randomized, double-blind, placebo-controlled Phase II clinical study. The primary objective is to evaluate the safety and efficacy of SG301 SC Injection in participants with ITP, while the secondary objectives are to assess the pharmacokinetic, pharmacodynamic and immunogenici...

Multi-target Drugs Sequential Combination Therapy in Adults Patients With Newly Diagnosed Primary Immune Thrombocytopenia Recruiting
NCT07788456 Phase: 4 Started: 2026-06-22

This prospective, single-arm, multicenter, exploratory study will enroll 38 adults patients with newly diagnosed primary immune thrombocytopenia (ITP). Patients will receive standard-dose corticosteroids plus a thrombopoietin receptor agonist (TPO-RA) as initial therapy During the core treatment pha...

A Study of Efficacy and Safety of Ianalumab Versus Placebo in Addition to Eltrombopag in Primary Immune Thrombocytopenia Patients Who Failed Steroids Active Not Recruiting
NCT05653219 Phase: 3 Started: 2023-02-02

The purpose of this study is to evaluate the effect of two different doses of ianalumab added to eltrombopag to prolong Time to Treatment Failure (TTF) in adults with primary ITP who failed previous first-line treatment with steroids.

Agents Found By Disease-Level Search

Searched directly from “Immune Thrombocytopaenia (ITP)” via ClinicalTrials.gov interventions and Open Targets' disease→drug data — independent of the 10-gene target panel above. This surfaces agents whose mechanism doesn't route through a curated gene (combination therapies, standard-of-care drugs, targets outside the panel).

57 agents shown (approved or with trial evidence) — 45 not found by the gene-target search above.

Eltrombopag

· 9 trials · source: ClinicalTrials.gov
NCT05653219 NCT07093606 NCT03939637

Romiplostim

· 7 trials · source: ClinicalTrials.gov
NCT02279173 NCT01516619 NCT07700238

Rituximab

· 5 trials · source: ClinicalTrials.gov
NCT07362238 NCT00372892 NCT01525836

Dexamethasone

· 5 trials · source: ClinicalTrials.gov
NCT01965626 NCT07065968 NCT02556814

IanalumabNOT in gene-target list

· 4 trials · source: ClinicalTrials.gov
NCT05653219 NCT07039422 NCT05885555

Hydroxychloroquine Oral TabletNOT in gene-target list

· 3 trials · source: ClinicalTrials.gov
NCT06479291 NCT06479317 NCT06479304

romiplostim (AMG 531)NOT in gene-target list

Approved · 2 trials · source: ClinicalTrials.gov
NCT00102336 NCT00283439

Hetrombopag Olamine

· 2 trials · source: ClinicalTrials.gov
NCT03222843 NCT06478537

Predniso(lo)ne

· 2 trials · source: ClinicalTrials.gov
NCT07700238 NCT00451594

Fostamatinib disodium

· 2 trials · source: ClinicalTrials.gov
NCT02076399 NCT02077192

ChidamideNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT03838354 NCT03838354

cyclosporine

· 2 trials · source: ClinicalTrials.gov
NCT00031980 NCT02281370

rilzabrutinib

· 1 trial · source: ClinicalTrials.gov
NCT07007962

Interleukin-2NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00774358

thrombopoietin receptor agonist (TPO-RA)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07421167

efgartigimod alfa (ARGX-113)NOT in gene-target list

Approved · 1 trial · source: ClinicalTrials.gov
NCT03102593

Kedrion IVIG 10%NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07059000

rhTPONOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02391272

TQB3473 TabletsNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06900920

CD4-IgGNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00002250

Eltrombopag TabletsNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02201290

Eltrombopag PfOSNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02201290

herombopag olamine tabletsNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06838949

IgPro10NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01390649

Eltrombopag Olamine

· 1 trial · source: ClinicalTrials.gov
NCT01610180

Neofordex®NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04968899

Intravenous immunoglobulinsNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04968899

Avatrombopag Oral TabletNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04638829

DaratumumabNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07362238

sirolimusNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00392951

Rituxan, Cyclophosphamide, Vincristine, PrednisoneNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00774202

Higher Dose of RituximabNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00774202

eltrombopag combining rituximabNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04518475

All-trans retinoic acidNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01667263

DanazolNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01667263

Mycophenolate Mofetil (MMF) TreatmentNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06288932

fostamatinib (R788)NOT in gene-target list

Approved · 1 trial · source: ClinicalTrials.gov
NCT04132050

OseltamivirNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01965626

Aspirin

· 1 trial · source: ClinicalTrials.gov
NCT06478537

CM336 InjectionNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06799611

Second Line ITP agentsNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01971684

TeriflunomideNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07065968

Obinutuzumab Injection [Gazyva]NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06094881

TPO-RANOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04890041

BCMA-CD19 CAR-T therapyNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06794008

Eltrombopag oral tabletsNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00828750

TPONOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04089267

Sym001NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00718692

Caffeic acid tabletsNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02556814

CM313 injectionNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06594146

Fostamatinib

· 1 trial · source: ClinicalTrials.gov
NCT06071520

Rituximab combined with Daratumumab（Anti-CD38 Monoclonal Antibody）NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07362199

QT-019CNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07315087

PovetaciceptNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05757570

totrombopag (LGD-4665)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00621894

SteroidsNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03939637

IVIGNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03939637

Generated by the Open Source Medicine Foundation Biomarker Pipeline — 2026-10-08. Data sources: DGIdb, Open Targets, ChEMBL, PubMed, ClinicalTrials.gov. Not medical advice.