Immune Thrombocytopaenia (ITP) — RepurpOS

Disease overview

Alterations

130

Molecular (A)

100

Clinical (B–E)

Direct drugs

108

Via biomarker

445

Natural agents

Natural products

Merged ranked

551

With trial/literature evidence

Sources: Open Targets, HPO, ClinicalTrials.gov, DGIdb, ChEMBL, PubMed Literature, Seeds, Examine.com · Pipeline phase 7 · Updated 2026-07-06

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

Drug-free remission

—

Relapse after remission

—

Chronicity

—

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

Barrier detail: 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

Last SoC change: 2023 — rilzabrutinib (BTK inhibitor) Phase 3 LUNA data; daprodustat + avatrombopag combinations; 2021 fostamatinib (SYK inhibitor) added; 2008 romiplostim/eltrombopag first-line TPO-RAs; splenectomy standard since early 20th century

Remission data layers: disease_db_100 · source-locked manual data · confidence: high

Identifiers: MONDO: MONDO:0008558 · ORPHA: ORPHA:1855

Right to Try Relevance Critical

Eligibility: This disease meets the federal and state right-to-try legal standard (21 U.S.C. § 360bbb-0a and Montana SB 535): it lacks disease-modifying therapy with indication-specific FDA approval. Patients with Immune Thrombocytopenia may be eligible to access investigational therapies outside of clinical trials under right-to-try frameworks.

Investigational Agents

Phase 3: 28, Phase 2: 12

Most Advanced Phase

Phase 3

Highest dev. stage

Gene Targets

130

Actionable

Pipeline Compounds (Top 6)

Avatrombopag, Belimumab, Betamethasone, Betamethasone Sodium Phosphate, Bortezomib, Caffeic Acid

Legal Standard: Right-to-try requires the disease lacks "adequate approved therapy for that specific disease" (not symptom-management drugs). This distinction means a disease with antidepressants available still qualifies if no FDA approval exists for THAT disease indication.

View Right-to-Try Compendium

Alterations

Showing 80 of 130 · Types A (molecular) through E (functional)

Name	Type	Subtype	Direction	Frequency	Evidence	Sources	Links
Abnormal bleeding	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Abnormal vaginal bleeding	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Anti-glutamic acid decarboxylase antibody positivity	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Anti-insulin antibody positivity	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO LOINC
Anti-islet antigen-2 antibody positivity	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Anti-platelet antigen antibody positivity	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Anti-thyroglobulin antibody positivity	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Anti-thyroid peroxidase antibody positivity	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Anti-thyroid-stimulating hormone receptor antibody positivity	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Arterial thrombosis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Bruising susceptibility	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Cerebral hemorrhage	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Epistaxis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Gastrointestinal hemorrhage	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Gingival bleeding	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hematuria	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hemorrhage of the eye	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Liver kidney microsome type 1 antibody positivity	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Petechiae	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Platelet antibody positive	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Purpura	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Thrombocytopenia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
ADRM1 ADRM1 26S proteasome ubiquitin receptor	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
AR androgen receptor	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ATP4A ATPase H+/K+ transporting subunit alpha	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ATP4B ATPase H+/K+ transporting subunit beta	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
BCL2L1 BCL2 like 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
BRD7 bromodomain containing 7	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
BTK Bruton tyrosine kinase	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CD38 CD38 molecule	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CD4 CD4 molecule	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CD52 CD52 molecule	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CD8A CD8 subunit alpha	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CTLA4 cytotoxic T-lymphocyte associated protein 4	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
Consecutive Increased Platelet Counts (≥2 Consecutive PLT ≥ 30,000/µL)	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
DNMT1 DNA methyltransferase 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
DNMT3A DNA methyltransferase 3 alpha	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
Duration of complete response	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
Duration of response	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
Evaluation of platelet response (Complete Response)	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
FCGRT Fc gamma receptor and transporter	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
FOXF1 forkhead box F1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
HMGCR 3-hydroxy-3-methylglutaryl-CoA reductase	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
IFNG interferon gamma	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
IL10 interleukin 10	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
IL17A interleukin 17A	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
IL1B interleukin 1 beta	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
IMPDH1 inosine monophosphate dehydrogenase 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
IMPDH2 inosine monophosphate dehydrogenase 2	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ITGA2B integrin subunit alpha 2b	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
JAK1 Janus kinase 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
JAK2 Janus kinase 2	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
KCNV2 potassium voltage-gated channel modifier subfamily V member 2	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
MIF macrophage migration inhibitory factor	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
MPL MPL proto-oncogene, thrombopoietin receptor	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
MS4A1 membrane spanning 4-domains A1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
NAV2 neuron navigator 2	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
NKD1 NKD inhibitor of Wnt signaling pathway 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
NR3C1 nuclear receptor subfamily 3 group C member 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
PDCD1 programmed cell death 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
PGR progesterone receptor	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
PPIA peptidylprolyl isomerase A	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
PSMA1 proteasome 20S subunit alpha 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
PSMA2 proteasome 20S subunit alpha 2	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
PSMA3 proteasome 20S subunit alpha 3	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
PSMA4 proteasome 20S subunit alpha 4	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
PSMA5 proteasome 20S subunit alpha 5	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
PSMA6 proteasome 20S subunit alpha 6	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
PSMA7 proteasome 20S subunit alpha 7	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
PSMA8 proteasome 20S subunit alpha 8	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
PSMB1 proteasome 20S subunit beta 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
PSMB10 proteasome 20S subunit beta 10	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
PSMB11 proteasome subunit beta 11	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
PSMB2 proteasome 20S subunit beta 2	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
PSMB3 proteasome 20S subunit beta 3	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
PSMB4 proteasome 20S subunit beta 4	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
PSMB5 proteasome 20S subunit beta 5	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
PSMB6 proteasome 20S subunit beta 6	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
PSMB7 proteasome 20S subunit beta 7	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
PSMB8 proteasome 20S subunit beta 8	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets

FDA-Approved Therapies

Phase 4 agents with regulatory approval for this indication

Drug Name	Mechanism	Type
3,3',4',5-TETRACHLOROSALICYLANILIDE		Gene-targeted
ABATACEPT		Gene-targeted
ABIRATERONE ACETATE	inhibitor	Gene-targeted
ACALABRUTINIB	inhibitor; inhibitor	Gene-targeted
ACALABRUTINIB MALEATE	inhibitor	Gene-targeted
ACETAZOLAMIDE	None	Gene-targeted
ACETOPHENAZINE		Gene-targeted
ALCLOFENAC		Gene-targeted
ALCLOMETASONE DIPROPIONATE		Gene-targeted
ALEMTUZUMAB	antibody; antibody; inhibitor	Gene-targeted
AMCINONIDE		Gene-targeted
AMDINOCILLIN		Gene-targeted
AMIODARONE HYDROCHLORIDE		Gene-targeted
AMLEXANOX		Gene-targeted
AMLODIPINE BESYLATE		Gene-targeted
AMPHETAMINE	None	Gene-targeted
ANHYDROUS TACROLIMUS		Gene-targeted
ANTI-THYMOCYTE GLOBULIN		Gene-targeted
APALUTAMIDE	inhibitor; inhibitor	Gene-targeted
APOMORPHINE		Gene-targeted
ARSENIC TRIOXIDE		Gene-targeted
ASTEMIZOLE		Gene-targeted
ATALUREN		Gene-targeted
ATEZOLIZUMAB		Gene-targeted
ATRACURIUM BESYLATE		Gene-targeted
ATROPINE		Gene-targeted
AZACITIDINE	inhibitor	Gene-targeted
AZARIBINE		Gene-targeted
BECLOMETHASONE DIPROPIONATE		Gene-targeted
BENAZEPRIL HYDROCHLORIDE		Gene-targeted
BENORILATE		Gene-targeted
BETAMETHASONE		Gene-targeted
BETAMETHASONE DIPROPIONATE		Gene-targeted
BETAMETHASONE SODIUM PHOSPHATE		Gene-targeted
BETAMETHASONE VALERATE		Gene-targeted
BICALUTAMIDE	inhibitor; inhibitor	Gene-targeted
BICALUTAMIDE	None	Gene-targeted
BIFONAZOLE		Gene-targeted
BISACODYL		Gene-targeted
BITHIONOL		Gene-targeted

Therapeutics

Direct disease associations, biomarker-linked drugs, OSMF narrative-review natural agents, and merged ranking. Only agents with registry trials or published clinical literature are shown by default.

Show agents without direct trial evidence

Drug	Type	Phase	Evidence	Score	Trials & literature	Sources	Links
SIROLIMUS	Small molecule	Approved	Strong	100	4 trials View evidence Registry trials NCT07657793 — Sirolimus in Patients With Systemic Lupus Erythematosus-Associated Immune Thrombocytopenia (RECRUITING) PHASE2, PHASE3 NCT01672151 — Efficacy of Rapamycin Therapy With Chronic Immune Thrombocytopenia (COMPLETED) NCT00392951 — Sirolimus for Autoimmune Disease of Blood Cells (COMPLETED) PHASE1, PHASE2 NCT01971684 — ICON1: Treatment Decisions and Outcomes in Pediatric Refractory ITP (COMPLETED) CT.gov search · Cochrane · PubMed	Open Targets, ChEMBL	ChEMBL Open Targets
TACROLIMUS ANHYDROUS	Small molecule	Approved	Strong	100	2 trials View evidence Registry trials NCT05471050 — The Combination of Tacrolimus and Danazol as the Treatment of Steroid-resistant/Relapse Immune Thrombocytopenia (UNKNOWN) PHASE2 NCT04747080 — The Combination of Tacrolimus and High-dose Dexamethasone as First-line Treatment in Adult Immune Thrombocytopenia (UNKNOWN) PHASE2 CT.gov search · Cochrane · PubMed	Open Targets, ChEMBL	ChEMBL Open Targets
3,3',4',5-TETRACHLOROSALICYLANILIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
ABATACEPT Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
ABIRATERONE ACETATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ACALABRUTINIB Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ACALABRUTINIB MALEATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
ACETAZOLAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ACETOPHENAZINE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ALCLOFENAC Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
ALCLOMETASONE DIPROPIONATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
ALEMTUZUMAB Repurposing	Small molecule	Approved	Strong	95	2 trials View evidence Registry trials NCT00749112 — Alemtuzumab and Rituximab in the Treatment of Refractory Autoimmune Cytopenias (COMPLETED) PHASE2, PHASE3 NCT03784898 — Collection of Blood Samples From Patients With Relapsing MS Who Developed ITP After Receiving Lemtrada (COMPLETED) PHASE4 CT.gov search · Cochrane · PubMed	DGIdb	—
AMCINONIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
AMDINOCILLIN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
AMIODARONE HYDROCHLORIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
AMLEXANOX Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
AMLODIPINE BESYLATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
AMPHETAMINE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ANHYDROUS TACROLIMUS Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ANTI-THYMOCYTE GLOBULIN Repurposing	Small molecule	Approved	Strong	95	1 trials View evidence Registry trials NCT00006055 — Autologous Peripheral Blood Stem Cell Transplantation in Patients With Life Threatening Autoimmune Diseases (UNKNOWN) NA CT.gov search · Cochrane · PubMed	DGIdb	—
APALUTAMIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
APOMORPHINE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ARSENIC TRIOXIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ASTEMIZOLE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ATALUREN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ATEZOLIZUMAB Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
ATRACURIUM BESYLATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ATROPINE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
AZACITIDINE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
AZARIBINE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BECLOMETHASONE DIPROPIONATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
BENAZEPRIL HYDROCHLORIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BENORILATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BETAMETHASONE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
BETAMETHASONE DIPROPIONATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BETAMETHASONE SODIUM PHOSPHATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BETAMETHASONE VALERATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BICALUTAMIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
BICALUTAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
BIFONAZOLE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
BISACODYL Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
BITHIONOL Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
BITHIONOLATE SODIUM Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BLEOMYCIN Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
BORTEZOMIB Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
BORTEZOMIB D-MANNITOL Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BROMOCRIPTINE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
BROMOCRIPTINE MESYLATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BUPRENORPHINE HYDROCHLORIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BUTAMBEN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets

Drug	Type	Phase	Evidence	Score	Sources	Links
AVATROMBOPAG	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
BELIMUMAB	Biologic	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
BETAMETHASONE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
BETAMETHASONE SODIUM PHOSPHATE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
BORTEZOMIB	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
CAFFEIC ACID	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
CYCLOSPORINE	Biologic	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
DAPSONE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
DECITABINE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
EFGARTIGIMOD ALFA	Biologic	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
HETROMBOPAG OLAMINE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
HYDROCORTISONE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
IANALUMAB	Biologic	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
METHYLPREDNISOLONE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
METHYLPREDNISOLONE ACETATE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
MEZAGITAMAB	Biologic	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
MYCOPHENOLATE MOFETIL	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
ORELABRUTINIB	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
OSELTAMIVIR	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
PREDNISOLONE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
RAFUTROMBOPAG	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
RECOMBINANT HUMAN THROMBOPOIETIN	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
RITUXIMAB	Biologic	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
ROZANOLIXIZUMAB	Biologic	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
SOVLEPLENIB	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
TRETINOIN	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
TRIAMCINOLONE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
TRIAMCINOLONE ACETONIDE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
ACETYLCYSTEINE	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
ALEMTUZUMAB	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
ATORVASTATIN	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
BARICITINIB	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
BATOCLIMAB	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
BERBERINE	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
BLISIBIMOD	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
BRIOBACEPT	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
CYCLOPHOSPHAMIDE	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
DACLIZUMAB	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
DANAZOL	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
DARATUMUMAB	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets

Drug	Type	Phase	Evidence	Score	Trials & literature	Sources	Links
3,3',4',5-TETRACHLOROSALICYLANILIDE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
ABATACEPT Repurposing via CTLA4	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
ABIRATERONE ACETATE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	—
ACALABRUTINIB Repurposing via BTK	Small molecule	Approved	Strong	95	—	DGIdb	—
ACALABRUTINIB MALEATE Repurposing via BTK	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
ACETAZOLAMIDE Repurposing via ADRM1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ACETOPHENAZINE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	—
ALCLOFENAC Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
ALCLOMETASONE DIPROPIONATE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
ALEMTUZUMAB Repurposing via CD52	Small molecule	Approved	Strong	95	2 trials View evidence Registry trials NCT00749112 — Alemtuzumab and Rituximab in the Treatment of Refractory Autoimmune Cytopenias (COMPLETED) PHASE2, PHASE3 NCT03784898 — Collection of Blood Samples From Patients With Relapsing MS Who Developed ITP After Receiving Lemtrada (COMPLETED) PHASE4 CT.gov search · Cochrane · PubMed	DGIdb	—
AMCINONIDE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	—
AMDINOCILLIN Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	—
AMIODARONE HYDROCHLORIDE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
AMLEXANOX Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	—
AMLODIPINE BESYLATE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
AMPHETAMINE Repurposing via ADRM1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ANHYDROUS TACROLIMUS Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	—
ANTI-THYMOCYTE GLOBULIN Repurposing via CTLA4	Small molecule	Approved	Strong	95	1 trials View evidence Registry trials NCT00006055 — Autologous Peripheral Blood Stem Cell Transplantation in Patients With Life Threatening Autoimmune Diseases (UNKNOWN) NA CT.gov search · Cochrane · PubMed	DGIdb	—
APALUTAMIDE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	—
APOMORPHINE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	—
ARSENIC TRIOXIDE Repurposing via DNMT1	Small molecule	Approved	Strong	95	—	DGIdb	—
ASTEMIZOLE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	—
ATALUREN Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	—
ATEZOLIZUMAB Repurposing via CTLA4	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
ATRACURIUM BESYLATE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	—
ATROPINE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	—
AZACITIDINE Repurposing via DNMT1	Small molecule	Approved	Strong	95	—	DGIdb	—
AZARIBINE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BECLOMETHASONE DIPROPIONATE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	—
BENAZEPRIL HYDROCHLORIDE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BENORILATE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BETAMETHASONE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	—
BETAMETHASONE DIPROPIONATE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BETAMETHASONE SODIUM PHOSPHATE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BETAMETHASONE VALERATE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BICALUTAMIDE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	—
BICALUTAMIDE Repurposing via ADRM1	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
BIFONAZOLE Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	—
BISACODYL Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	—
BITHIONOL Repurposing via AR	Small molecule	Approved	Strong	95	—	DGIdb	—

Natural Products

16 natural products with the same evidence schema as repurposed drugs — registry trials, PubMed literature, and reference links (pipeline-ranked). Only agents with direct clinical evidence are shown by default.

Reference lookups: Examine.com

Show agents without direct trial evidence

Drug	Type	Phase	Evidence	Score	Trials & literature	Links
Vitamin C Natural product Human Study Vitamin C may be effective in treating idiopathic thrombocytopenic purp… Human Study Vitamin K2 plus Vitamin D3 are therapeutic for the treatment of anemia…	Nutraceutical	Preclinical	Emerging	10	1 pubs View evidence CT.gov search · Cochrane · PubMed	PubChem
Vitamin D3 Natural product Human Study Vitamin K2 plus Vitamin D3 are therapeutic for the treatment of anemia… Human Study Vitamin C may be effective in treating idiopathic thrombocytopenic purp…	Nutraceutical	Approved	Emerging	10	4 trials View evidence Registry trials NCT04320225 — Connection Between Vitamin D Level and the Efficacy of Dexamethasone in Immune Thrombocytopenia (UNKNOWN) NCT04094805 — A Multicenter Randomized Study of Vitamin D Combined With HD-DXM Versus HD-DXM for the Treatment of ITP (UNKNOWN) PHASE4 NCT07146555 — Vitamin D Satus in Adults With Newly Diagnosed ITP (NOT_YET_RECRUITING) NCT06073925 — Use of Vitamin D as An Adjuvant Treatment in Children With Chronic Idiopathic Thrombocytopenic Purpura (UNKNOWN) EARLY_PHASE1 CT.gov search · Cochrane · PubMed	PubChem
Vitamin K Natural product Human Study Vitamin K2 plus Vitamin D3 are therapeutic for the treatment of anemia… Human Study Vitamin C may be effective in treating idiopathic thrombocytopenic purp…	Nutraceutical	Preclinical	Emerging	10	No indexed hits View evidence CT.gov search · Cochrane · PubMed	PubChem
Olive Oil Natural product Human Study Mediterranean diet maintained platelet count within a healthy range and… Human Study Carica papaya leaf extract increases the platelet count in dengue fever… Human: Case Report A case study using papaya leaf extract to reverse chemotherapy-induced…	Nutraceutical	Preclinical	Emerging	10	No indexed hits View evidence CT.gov search · Cochrane · PubMed	—
Melatonin Natural product Human Study Melatonin may be effective in treating refractory idiopathic thrombocyt… Human Study Melatonin may be effective in treating severe bleeding associated with…	Nutraceutical	Preclinical	Emerging	7	No indexed hits View evidence CT.gov search · Cochrane · PubMed	PubChem
Niacin Natural product Human In Vitro Nicotinamide enhances Treg differentiation by promoting Foxp3 acetylati… Human Study Vitamin C may be effective in treating idiopathic thrombocytopenic purp… Human Study Vitamin K2 plus Vitamin D3 are therapeutic for the treatment of anemia…	Nutraceutical	Phase II	Emerging	7	2 trials View evidence Registry trials NCT06776510 — A Clinical Study of NAD in the Treatment of Immune Thrombocytopenia (RECRUITING) PHASE1, PHASE2 NCT05095896 — The Serum Lipid Profiles in ITP: a Retrospective Study (COMPLETED) CT.gov search · Cochrane · PubMed	PubChem
Astragalus Natural product	TCM herb	Preclinical	Emerging	7	No indexed hits View evidence CT.gov search · Cochrane · PubMed	—
Berberine Natural product	Botanical	Phase II	Emerging	7	1 trials View evidence Registry trials NCT03909763 — Combination of Danazole With Berberine in the Treatment of ITP (UNKNOWN) PHASE2 CT.gov search · Cochrane · PubMed	PubChem
Papaya Natural product Human Study Carica papaya leaf extract increases the platelet count in dengue fever… Human: Case Report A case study using papaya leaf extract to reverse chemotherapy-induced…	Nutraceutical	Preclinical	Emerging	0	No indexed hits View evidence CT.gov search · Cochrane · PubMed	—
Chinense Herbal Formula: Sheng Ban Recipe Natural product Human Study The Chinese Herbal Formula Sheng Ban Recipe is effective in treating th…	Nutraceutical	Preclinical	Emerging	0	No indexed hits View evidence CT.gov search · Cochrane · PubMed	—
Caffeic Acid Natural product Human Study This study confirms caffeic acid tablets plus dexamethasone as a well-t…	Nutraceutical	Approved	Emerging	0	2 trials View evidence Registry trials NCT02556814 — Caffeic Acid Combining High-dose Dexamethasone in Management of ITP (COMPLETED) PHASE4 NCT02351622 — Caffeic Acid Tablets as a Second-line Therapy for ITP (COMPLETED) PHASE3 CT.gov search · Cochrane · PubMed	—
Angelica Natural product	Nutraceutical	Preclinical	Emerging	0	No indexed hits View evidence CT.gov search · Cochrane · PubMed	—
Goji Natural product	Nutraceutical	Preclinical	Emerging	0	No indexed hits View evidence CT.gov search · Cochrane · PubMed	—
Chloranthus Glaber Natural product	Nutraceutical	Preclinical	Emerging	0	No indexed hits View evidence CT.gov search · Cochrane · PubMed	—
Sweet Potato Natural product	Nutraceutical	Preclinical	Emerging	0	No indexed hits View evidence CT.gov search · Cochrane · PubMed	—
EGCG (Epigallocatechin Gallate) Natural product	Nutraceutical	Preclinical	Emerging	0	No indexed hits View evidence CT.gov search · Cochrane · PubMed	—

Alterations and therapeutics are aggregated from public databases (Open Targets, HPO, ChEMBL, DGIdb, and others). Natural agents are candidates from OSMF narrative reviews on opensourcemed.info/chronic-disease.html — not approved treatments. This page is for research orientation only — not medical advice.