Autoimmune thrombocytopenic purpura

Summary

Autoimmune thrombocytopenic purpura (MONDO:0008558) is a disease with 1 cohort gene (27 GWAS associations across 5 studies) and 327 clinical trials. Top therapeutic interventions include eltrombopag, romiplostim, and avatrombopag.

At a glance

• Prevalence: 1-5 / 10 000 (Europe) [Orphanet-validated]
• Cohort genes: 1
• GWAS associations: 27
• ClinVar variants: 1
• Phenotypes (HPO): 20
• Clinical trials: 327

Clinical features

Epidemiology

Prevalence records

7 prevalence record(s), Orphanet:

Type	Class	Value	Geography	Validation
Point prevalence	1-5 / 10 000	25	Europe	Validated
Annual incidence	1-9 / 100 000	3.9	United Kingdom	Validated
Annual incidence	1-9 / 100 000	2.68	Denmark	Validated
Annual incidence	1-9 / 100 000	2.9	France	Validated
Point prevalence	1-9 / 100 000	9.5	United States	Validated
Point prevalence	1-5 / 10 000	45	Denmark	Validated
Annual incidence	1-9 / 100 000	6.75	Europe	Not yet validated

Signs & symptoms

Clinical features (HPO)

20 HPO clinical features (Orphanet curated; top 20 by frequency):

HPO ID	Term	Frequency
HP:0001873	Thrombocytopenia	Very frequent (80-99%)
HP:0000967	Petechiae	Frequent (30-79%)
HP:0000979	Purpura	Frequent (30-79%)
HP:0004420	Arterial thrombosis	Frequent (30-79%)
HP:0025329	Anti-glutamic acid decarboxylase antibody positivity	Frequent (30-79%)
HP:0025379	Anti-thyroid peroxidase antibody positivity	Frequent (30-79%)
HP:0030908	Liver kidney microsome type 1 antibody positivity	Frequent (30-79%)
HP:0032069	Anti-thyroglobulin antibody positivity	Frequent (30-79%)
HP:0034062	Anti-insulin antibody positivity	Frequent (30-79%)
HP:0034063	Anti-islet antigen-2 antibody positivity	Frequent (30-79%)
HP:4000170	Anti-platelet antigen antibody positivity	Frequent (30-79%)
HP:0034189	Anti-thyroid-stimulating hormone receptor antibody positivity	Frequent (30-79%)
HP:0000225	Gingival bleeding	Occasional (5-29%)
HP:0000421	Epistaxis	Occasional (5-29%)
HP:0000790	Hematuria	Occasional (5-29%)
HP:0000978	Bruising susceptibility	Occasional (5-29%)
HP:0002239	Gastrointestinal hemorrhage	Occasional (5-29%)
HP:0034263	Abnormal vaginal bleeding	Occasional (5-29%)
HP:0001342	Cerebral hemorrhage	Very rare (<1-4%)
HP:0011885	Hemorrhage of the eye	Very rare (<1-4%)

Identifiers

Disease identifiers

Field	Value
Canonical name	autoimmune thrombocytopenic purpura
Mondo ID	MONDO:0008558
EFO	EFO:0007160
OMIM	188030
Orphanet	3002
DOID	DOID:8924
ICD-10-CM	D69.3
ICD-11	364346400
NCIT	C3446
UMLS	C0398650
MedGen	584986
GARD	0005194
MedDRA	10021245
Is cancer (heuristic)	no

Also known as: AITP · autoimmune thrombocytopenic purpura · idiopathic thrombocytopenia · idiopathic thrombocytopenia purpura · idiopathic thrombocytopenic purpura · immune thrombocytopenia · ITP · thrombocytopenic purpura autoimmune · thrombocytopenic purpura, autoimmune

Data availability: 1 ClinVar variant · 27 GWAS associations (5 studies) · 12 cell lines.

Disease family

Classification path: disease › human disease › disease by body system or component › hematologic disorder › autoimmune disorder of blood › primary thrombocytopenia › autoimmune thrombocytopenic purpura

Related subtypes (1): Evans syndrome

Genetics & variants

GWAS landscape

27 GWAS associations across 5 studies. Top hits map to 14 distinct genes (as reported by GWAS).

Top associations by p-value

rsID	p-value	Gene	Risk allele	Odds ratio
rs4785426	2e-16	LINC02178 - NKD1	?	0.48
rs10967515	3e-15	VLDLR - KCNV2	?	0.4
rs4785216	9e-15	LINC02178 - NKD1	?	0.43
rs2165798	3e-11	NAV2	?	0.52
rs8054390	1e-08	LINC00917 - FENDRR	?	3.98
rs75650997	4e-08	SLC24A4	?	2.38
rs75276578	9e-08	AMPD2	?	4.16
rs117609649	4e-07	ABCC4	?	0.26
rs145657859	5e-07	ETS1 - FLI1	?	2.88
rs2160725	9e-07	ARSG	?	1.37
rs117333642	1e-06	HRC	?	2.31
rs78144867	1e-06	MAP3K7 - MIR4643	?	2.55
rs1480022	1e-06	GRIP1	?	2.75
rs6977214	2e-06	LINC00972 - SOCS5P1	?	0.73
rs142085785	3e-06	CAP2	?	1.75
rs11021056	3e-06	LNCRNA-IUR	?	1.54
rs78978806	4e-06	LEF1-AS1 - RPSAP34	?	1.89
rs6660739	4e-06	DNAJC6	?	1.52
rs79197641	6e-06	SNPH, RAD21L1	?	3.21
rs73708248	6e-06	SEMA3E	?	3.23
rs139905149	6e-06	C11orf96 - ACCSL	?	2.14
rs62000424	8e-06	ARSG	?	1.47
rs115302564	8e-06	CCDC85A	?	3.98
rs79328664	8e-06	DKK1 - RPL31P44	?	3.45
rs2960794	9e-06	RPS3AP6 - NMNAT1P5	?	1.35
rs143125379	1e-05	EIF2S2P7 - ACTG1P22	?	2.92
rs111446179	1e-05	FABP1 - THNSL2	?	2.13

Top studies (by case count)

Study	Lead author	Year	Cases	Controls	Title
GCST90018865	Sakaue S	2021	675	488,749	A cross-population atlas of genetic associations for 220 human phenotypes.
GCST011588	Xu Y	2020	200	0	A Pooling Genome-Wide Association Study Identifies Susceptibility Loci and Signaling Pathways of Immune Thrombocytopenia in Chinese Han Population.
GCST90018645	Sakaue S	2021	168	178,558	A cross-population atlas of genetic associations for 220 human phenotypes.
GCST90654659	Kim TO	2024	0	0	Genetic Variants in Canonical Wnt Signaling Pathway Associated with Pediatric Immune Thrombocytopenia.
GCST90654660	Kim TO	2024	0	0	Genetic Variants in Canonical Wnt Signaling Pathway Associated with Pediatric Immune Thrombocytopenia.

Variant details and genetic-evidence tiers

Tier distribution (top 50 variants)

Tier	Variants
Tier 1: coding	1
Tier 2: splice/UTR	1
Tier 3: regulatory	0
Tier 4: intronic/intergenic	25

MAF distribution

Bucket	Variants
common (>=0.05)	11
low_freq (0.01-0.05)	16
rare (<0.01)	0
unknown	0

Functional consequences

Consequence	Count
intron_variant	18
intergenic_variant	7
5_prime_UTR_variant	1
missense_variant	1

Top variants

rsID	Chr	Pos	Alleles	MAF	Consequence	Gene	p-value	Tier
rs4785426	16	50459519	A>C,G,T	0.28	intron_variant	LINC02178 - NKD1	2e-16	Tier 4: intronic/intergenic
rs10967515	9	2684117	T>C,G	0.18	intron_variant	VLDLR - KCNV2	3e-15	Tier 4: intronic/intergenic
rs4785216	16	50545483	T>C	0.19	intergenic_variant	LINC02178 - NKD1	9e-15	Tier 4: intronic/intergenic
rs2165798	11	19891235	G>A	0.18	intron_variant	NAV2	3e-11	Tier 4: intronic/intergenic
rs8054390	16	86429331	A>G	0.02	intergenic_variant	LINC00917 - FENDRR	1e-08	Tier 4: intronic/intergenic
rs75650997	14	92400915	C>G,T	0.02	intron_variant	SLC24A4	4e-08	Tier 4: intronic/intergenic
rs75276578	1	109630214	C>G,T	0.01	intron_variant	AMPD2	9e-08	Tier 4: intronic/intergenic
rs117609649	13	95088043	C>A	0.05	intron_variant	ABCC4	4e-07	Tier 4: intronic/intergenic
rs145657859	11	128647882	G>A,T	0.01	intron_variant	ETS1 - FLI1	5e-07	Tier 4: intronic/intergenic
rs2160725	17	68399585	A>C,T	0.45	intron_variant	ARSG	9e-07	Tier 4: intronic/intergenic
rs117333642	19	49151595	G>A,C,T	0.02	intron_variant	HRC	1e-06	Tier 4: intronic/intergenic
rs78144867	6	90901563	T>G	0.01	intron_variant	MAP3K7 - MIR4643	1e-06	Tier 4: intronic/intergenic
rs1480022	12	66743388	A>C	0.01	intergenic_variant	GRIP1	1e-06	Tier 4: intronic/intergenic
rs6977214	7	85717175	T>C	0.45	intergenic_variant	LINC00972 - SOCS5P1	2e-06	Tier 4: intronic/intergenic
rs142085785	6	17547630	C>A,G,T	0.04	intron_variant	CAP2	3e-06	Tier 4: intronic/intergenic
rs11021056	11	95156546	A>G,T	0.11	intron_variant	LNCRNA-IUR	3e-06	Tier 4: intronic/intergenic
rs78978806	4	108354670	G>A	0.03	intron_variant	LEF1-AS1 - RPSAP34	4e-06	Tier 4: intronic/intergenic
rs6660739	1	65336146	G>A	0.1	intron_variant	DNAJC6	4e-06	Tier 4: intronic/intergenic
rs79197641	20	1294668	C>T	0.02	5_prime_UTR_variant	SNPH, RAD21L1	6e-06	Tier 2: splice/UTR
rs73708248	7	83538896	T>C	0.01	intron_variant	SEMA3E	6e-06	Tier 4: intronic/intergenic
rs139905149	11	44020559	C>T	0.02	intergenic_variant	C11orf96 - ACCSL	6e-06	Tier 4: intronic/intergenic
rs62000424	17	68420216	C>G,T	0.11	missense_variant	ARSG	8e-06	Tier 1: coding
rs115302564	2	56373996	A>G,T	0.01	intron_variant	CCDC85A	8e-06	Tier 4: intronic/intergenic
rs79328664	10	52346213	C>A,T	0.01	intergenic_variant	DKK1 - RPL31P44	8e-06	Tier 4: intronic/intergenic
rs2960794	15	59963686	T>A,C	0.27	intergenic_variant	RPS3AP6 - NMNAT1P5	9e-06	Tier 4: intronic/intergenic
rs143125379	2	57531219	A>G	0.01	intron_variant	EIF2S2P7 - ACTG1P22	1e-05	Tier 4: intronic/intergenic
rs111446179	2	88142466	C>G,T	0.02	intron_variant	FABP1 - THNSL2	1e-05	Tier 4: intronic/intergenic

ClinVar germline variants

1 retrieved; paginated sample, class counts are floors:

1 pathogenic

ClinVar	Variant (HGVS)	Gene	Classification	Review
977212	NM_003745.2(SOCS1):c.368C>G (p.Pro123Arg)	SOCS1	Pathogenic	criteria provided, single submitter

Genes & proteins

Mendelian disease overlap and somatic drivers

GenCC: 0 · Orphanet: 1 · OMIM-shared: 0 · Dual-evidence (GWAS+Mendelian): 0

Orphanet rare-disease linkage (cohort genes)

Gene	Orphanet ID	Rare disease
SOCS1	Orphanet:619948	Early-onset autoimmunity-autoinflammation-immunodeficiency syndrome due to SOCS1 haploinsufficiency

Cohort genes → proteins

1 cohort genes, 1 distinct canonical proteins.

Evidence partition

Subset	Genes
multi_evidence	1

Cohort genes (full)

Symbol	HGNC	Ensembl	UniProt	Name	Evidence
SOCS1	HGNC:19383	ENSG00000185338	O15524	Suppressor of cytokine signaling 1	clinvar

Cohort function summary

Lead sentence per gene, UniProt-curated.

Symbol	Protein name	Function (lead sentence)
SOCS1	Suppressor of cytokine signaling 1	Essential negative regulator of type I and type II interferon (IFN) signaling, as well as that of other cytokines, including IL2, IL4, IL6 and leukemia inhibitory factor (LIF).

Protein-family classification

Druggable: 0 · Difficult: 1 · Unknown: 0 · Druggable fraction: 0.0

Family distribution

Cohort families vs a genome-wide background (hypergeometric, BH-FDR; fold = observed/expected). Counts kept; sorted by enrichment, so the catch-all Other/Unknown bucket no longer leads.

Family	Genes	Fold	FDR
Scaffold/PPI	1	17.3×	0.058

Per-gene assignment

Symbol	Family	Druggable?	EC	InterPro (top 3)
SOCS1	Scaffold/PPI	no		SH2, SOCS_box, SOCS1_SH2

Expression context

Cohort genes with no expression data: 0.

1 cohort gene are a single-cell marker in ≥1 SCXA experiment.

Breadth distribution (Bgee present_calls)

Bucket	Genes
narrow (1-5 tissues)	0
moderate (6-20)	0
broad (>20)	1
unknown	0

Top tissues across cohort

Tissue	Cohort genes
endocervix	1
sperm	1
type B pancreatic cell	1

Per-gene tissue summary (top 30)

Symbol	Bgee breadth	FANTOM5 breadth	SCXA	Top tissues
SOCS1	211	ubiquitous	marker	type B pancreatic cell, sperm, endocervix

Protein interactions among cohort

Intra-cohort edges: 0.

Hub genes (top 10 by interactor count)

Symbol	Interactor count
SOCS1	3,435

Structural data

PDB: 0 · AlphaFold-only: 1 · No structure: 0

AlphaFold-only cohort genes (top 30 by pLDDT)

Symbol	UniProt	pLDDT
SOCS1	O15524	84.20

Function

Pathway analysis

Distinct Reactome pathways touched by cohort: 27. Enrichment computed across 1 evidence-associated genes (1 with Reactome annotation).

Pathways by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 1 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

Pathway	Cohort genes	Fold	FDR	Sample cohort genes
Regulation of IFNG signaling	1	815.7×	0.010	SOCS1
Regulation of KIT signaling	1	601.0×	0.010	SOCS1
Signaling by CSF3 (G-CSF)	1	571.0×	0.010	SOCS1
Growth hormone receptor signaling	1	475.8×	0.010	SOCS1
Regulation of IFNA/IFNB signaling	1	439.2×	0.010	SOCS1
Inactivation of CSF3 (G-CSF) signaling	1	439.2×	0.010	SOCS1
Interleukin-7 signaling	1	317.2×	0.012	SOCS1
Signaling by SCF-KIT	1	248.3×	0.013	SOCS1
Toll Like Receptor TLR6:TLR2 Cascade	1	175.7×	0.013	SOCS1
Toll Like Receptor 2 (TLR2) Cascade	1	173.0×	0.013	SOCS1
Toll Like Receptor TLR1:TLR2 Cascade	1	167.9×	0.013	SOCS1
MyD88:MAL(TIRAP) cascade initiated on plasma membrane	1	152.3×	0.013	SOCS1
Interferon alpha/beta signaling	1	152.3×	0.013	SOCS1
Toll Like Receptor 4 (TLR4) Cascade	1	131.3×	0.013	SOCS1
Interferon gamma signaling	1	125.5×	0.013	SOCS1
Toll-like Receptor Cascades	1	124.1×	0.013	SOCS1
Interferon Signaling	1	120.2×	0.013	SOCS1
Interleukin-4 and Interleukin-13 signaling	1	102.9×	0.015	SOCS1
Class I MHC mediated antigen processing & presentation	1	70.1×	0.020	SOCS1
Signaling by Interleukins	1	64.2×	0.021	SOCS1
Signaling by Receptor Tyrosine Kinases	1	51.7×	0.025	SOCS1
Cytokine Signaling in Immune system	1	40.8×	0.030	SOCS1
Antigen processing: Ubiquitination & Proteasome degradation	1	37.2×	0.032	SOCS1
Adaptive Immune System	1	29.8×	0.038	SOCS1
Innate Immune System	1	25.5×	0.042	SOCS1
Immune System	1	13.0×	0.080	SOCS1
Signal Transduction	1	10.2×	0.098	SOCS1

GO biological processes by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 1 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

GO term	Cohort genes	Fold	FDR	Sample cohort genes
negative regulation of CD8-positive, alpha-beta T cell differentiation	1	5617.3×	0.002	SOCS1
positive regulation of CD4-positive, alpha-beta T cell differentiation	1	2808.7×	0.002	SOCS1
positive regulation of regulatory T cell differentiation	1	936.2×	0.004	SOCS1
negative regulation of receptor signaling pathway via JAK-STAT	1	887.0×	0.004	SOCS1
macrophage differentiation	1	468.1×	0.006	SOCS1
negative regulation of insulin receptor signaling pathway	1	374.5×	0.006	SOCS1
cellular response to amino acid stimulus	1	306.4×	0.006	SOCS1
cell surface receptor signaling pathway via JAK-STAT	1	290.6×	0.006	SOCS1
regulation of cytokine production	1	247.8×	0.006	SOCS1
fat cell differentiation	1	181.2×	0.007	SOCS1
cytokine-mediated signaling pathway	1	130.6×	0.009	SOCS1
protein ubiquitination	1	41.4×	0.026	SOCS1
intracellular signal transduction	1	38.1×	0.026	SOCS1

Therapeutics

Drugs indicated for this disease

5 approved, 23 in late-stage (phase 3) trials. Disease-direct ChEMBL indications, not inferred from the associated-gene cohort below.

Drug	Development status
Cortisone Acetate	Approved (phase 4)
Dexamethasone	Approved (phase 4)
Human Immunoglobulin G	Approved (phase 4)
Prednisone	Approved (phase 4)
Romiplostim	Approved (phase 4)
Avatrombopag	Phase 3 (in late-stage trials)
Belimumab	Phase 3 (in late-stage trials)
Bortezomib	Phase 3 (in late-stage trials)
Caffeic Acid	Phase 3 (in late-stage trials)
Cyclosporine	Phase 3 (in late-stage trials)
Decitabine	Phase 3 (in late-stage trials)
Efgartigimod Alfa	Phase 3 (in late-stage trials)
Eltrombopag	Phase 3 (in late-stage trials)
Fostamatinib	Phase 3 (in late-stage trials)
Hetrombopag Olamine	Phase 3 (in late-stage trials)
Ianalumab	Phase 3 (in late-stage trials)
Methylprednisolone	Phase 3 (in late-stage trials)
Mycophenolate Mofetil	Phase 3 (in late-stage trials)
Oseltamivir	Phase 3 (in late-stage trials)
Platelet Concentrate	Phase 3 (in late-stage trials)
Prednisolone	Phase 3 (in late-stage trials)
Rafutrombopag	Phase 3 (in late-stage trials)
Recombinant Human Thrombopoietin	Phase 3 (in late-stage trials)
Rilzabrutinib	Phase 3 (in late-stage trials)
Rituximab	Phase 3 (in late-stage trials)
Rozanolixizumab	Phase 3 (in late-stage trials)
Sovleplenib	Phase 3 (in late-stage trials)
Tretinoin	Phase 3 (in late-stage trials)

Earlier-phase candidates (phase 2, investigational — efficacy not yet established): Acetylcysteine, Alemtuzumab, Aspirin, Atorvastatin, Baricitinib, Batoclimab, Blisibimod, Danazol, Daratumumab, Diammonium Glycyrrhizinate, Iguratimod, Imlifidase, Ixazomib, Lusutrombopag, Obinutuzumab, Orelabrutinib, Riliprubart, Tacrolimus Anhydrous, Terbutaline, Teriflunomide, Thalidomide, Vincristine, Zanubrutinib.

Drug target analysis

Approved (phase 4): 0 · Phase ≥3: 0 · Phased (≥1): 0 · Undrugged: 1

Druggability breadth: 0 of 1 evidence-associated genes (0%) have a ChEMBL target (buckets above are over the deeply-mined display cohort).

Top cohort targets by molecule count

Symbol	Molecules	Max phase
SOCS1	0	0

Bioactivity and enzyme data

Enzyme cohort genes (≥1 EC): 0.

Pharmacogenomics

Cohort genes with a PharmGKB record: 1; with CPIC/DPWG dosing guidelines: 0.

No cohort gene has a CPIC/DPWG genotype-guided dosing guideline (PharmGKB).

Chemical tractability of cohort targets

0 approved/phased compounds have measured bioactivity against a cohort gene (and aren’t yet in disease-level trials). This is a research / tractability signal, NOT a therapeutic recommendation — a bioactivity row often reflects off-target or screening binding (e.g. promiscuous kinase inhibitors against a cohort kinase), implying no disease mechanism.

Druggability pyramid

Cohort genes binned by druggability tier (high → low):

Tier	Definition	Genes	Symbols
A	Approved (phase 4 drug)	0
B	Phased (≥1) drug, not yet approved	0
C	Druggable family + PDB, no drug	0
D	Druggable family + AlphaFold only, no drug	0
E	Difficult family or no structure, no drug	1	SOCS1

Undrugged target profiles

1 cohort genes are undrugged. Ranked by ‘starting-point quality’ (assay depth + drugged-partner adjacency).

Symbol	ChEMBL assays	Drugged partners (top 3)
SOCS1	0	—

Clinical trials & evidence

Clinical trials

Clinical trials: 327.

Phase distribution (across all retrieved trials)

Phase	Trials
PHASE2	97
Not specified	86
PHASE3	72
PHASE1	21
PHASE4	18
PHASE1/PHASE2	18
PHASE2/PHASE3	8
EARLY_PHASE1	7

Top trials by phase / activity

NCT	Phase	Status	Title
NCT03201848	PHASE4	ACTIVE_NOT_RECRUITING	The Efficacy and Safety of Huaiqihuang Granule in Children With Chronic Primary Immune Thrombocytopenia
NCT05522465	PHASE4	RECRUITING	Short-course High-dose Prednisone and Dexamethasone in Children With ITP
NCT00888901	PHASE4	COMPLETED	Platelet Function in Idiopathic Thrombocytopenic Purpura (ITP) Patients With Eltrombopag
NCT00907478	PHASE4	COMPLETED	Study on Bone Marrow Morphology in Adults Receiving Romiplostim for Treatment of Thrombocytopenia Associated With Immune Thrombocytopenia Purpura (ITP)
NCT02556814	PHASE4	COMPLETED	Caffeic Acid Combining High-dose Dexamethasone in Management of ITP
NCT02642380	PHASE4	WITHDRAWN	Different Cycles of High-dose Dexamethasone for Initial Management of Primary Immune Thrombocytopenia (ITP)
NCT02760251	PHASE4	COMPLETED	Immunomodulation With Romiplostim in Young Adults With ITP
NCT03172676	PHASE4	UNKNOWN	Effects of Helicobacter Pylori Eradication in Children With Chronic Immune Thrombocytopenic Purpura
NCT03229746	PHASE4	COMPLETED	Reposition of Second Line Treatment in Chronic Immune Thrombocytopenia
NCT03258866	PHASE4	COMPLETED	The Study of Different Dose Rituximab in the Treatment of ITP
NCT03771378	PHASE4	UNKNOWN	Efficacy and Safety of rhTPO and Eltrombopag in Patients With Primary Immune Thrombocytopenia (ITP)
NCT03866798	PHASE4	TERMINATED	Study to Evaluate the Efficacy and Safety of PANZYGA in Pediatric Patients With Chronic Immune Thrombocytopenia (ITP)
NCT03998982	PHASE4	UNKNOWN	Glycyrrhetinic Acid Combined With Dexamethasone in Management of Newly Diagnosed ITP
NCT04089267	PHASE4	COMPLETED	Evaluating the Efficacy and Safety of Different Human Thrombopoietin (rhTPO) Regimens in the Treatment of Patients With Primary Immune Thrombocytopenia (ITP)
NCT04094805	PHASE4	UNKNOWN	A Multicenter Randomized Study of Vitamin D Combined With HD-DXM Versus HD-DXM for the Treatment of ITP
NCT04102033	PHASE4	UNKNOWN	Eltrombopag in Chronic ITP
NCT04638829	PHASE4	COMPLETED	Safety and Treatment Satisfaction in Adults With Chronic ITP After Switching to Avatrombopag From Eltrombopag or Romiplostim
NCT05311930	PHASE4	UNKNOWN	Efficacy and Safety of TPO Receptor Agonists in the Treatment of Elderly ITP Patients
NCT04562766	PHASE3	ACTIVE_NOT_RECRUITING	Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP)
NCT04812925	PHASE3	ACTIVE_NOT_RECRUITING	A Phase 3 Study to Evaluate the Safety and Efficacy of Efgartigimod PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia
NCT04968899	PHASE3	RECRUITING	IgIV Plus Prednisone vs High-dose Dexamethasone for ITP
NCT05325593	PHASE3	ACTIVE_NOT_RECRUITING	Romiplostim Plus Dexamethasone vs Dexamethasone in Patients With Newly Diagnosed Primary Immune Thrombocytopenia
NCT05653219	PHASE3	ACTIVE_NOT_RECRUITING	A Study of Efficacy and Safety of Ianalumab Versus Placebo in Addition to Eltrombopag in Primary Immune Thrombocytopenia Patients Who Failed Steroids
NCT06900920	PHASE3	RECRUITING	A Study of TQB3473 Tablets Compared to Placebo in the Treatment of Adult Primary Immune Thrombocytopenia
NCT06962631	PHASE3	RECRUITING	V-IMMUNE® for Immune Thrombocytopenia
NCT07007962	PHASE3	RECRUITING	Study to Evaluate the Efficacy and Safety of Oral Rilzabrutinib in Adults With Immune Thrombocytopenia (ITP) Who Failed First-line Treatment
NCT07194850	PHASE2/PHASE3	RECRUITING	A Study of Efgartigimod IV in Participants From 12 Years to Less Than 18 Years of Age With Chronic Immune Thrombocytopenia (ITP)
NCT07216079	PHASE3	ACTIVE_NOT_RECRUITING	Rilzabrutinib for the Adult Participants With Chronic ITP Who Have Completed Phase 3 Study in Japan
NCT07597395	PHASE3	NOT_YET_RECRUITING	ATRA for Management of Primary ITP
NCT00102323	PHASE3	COMPLETED	AMG 531 Treatment of Thrombocytopenic Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP) Refractory to Splenectomy
NCT00102336	PHASE3	COMPLETED	AMG 531 Treatment of Thrombocytopenic Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP) Prior to Splenectomy
NCT00116688	PHASE3	COMPLETED	Open Label Extension Study of Romiplostim (AMG 531) in Thrombocytopenic Patients With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
NCT00344149	PHASE3	COMPLETED	Rituximab as Second Line Treatment for ITP
NCT00362349	PHASE3	COMPLETED	Ig NextGen 10% in Idiopathic Thrombocytopenic Purpura (ITP) Patients
NCT00415532	PHASE3	COMPLETED	Romiplostim (AMG 531) Versus Medical Standard of Care for Immune (Idiopathic) Thrombocytopenic Purpura
NCT00451594	PHASE3	COMPLETED	High Dose Dexamethasone Vs. Conventional Dose Prednisolone in Adult ITP
NCT00508820	PHASE3	COMPLETED	An Open Label Study of Romiplostim in Adult Thrombocytopenic Subjects With ITP
NCT00511147	PHASE3	COMPLETED	IGIV Study for Chronic ITP Patients Ages 3-70
NCT00603642	PHASE3	COMPLETED	P3 Study to Evaluate Efficacy and Safety of AMG 531 in Thrombocytopenic Japanese Subjects With Immune (Idiopathic) Thrombocytopenic Purpura
NCT00749112	PHASE2/PHASE3	COMPLETED	Alemtuzumab and Rituximab in the Treatment of Refractory Autoimmune Cytopenias

Drugs tested across these trials (top 30)

Molecule	Max phase	Trials referencing
ELTROMBOPAG	4	21
ROMIPLOSTIM	4	17
AVATROMBOPAG	4	11
DANAZOL	4	9
DARATUMUMAB	4	6
FOSTAMATINIB	4	6
EFGARTIGIMOD ALFA	4	5
TERIFLUNOMIDE	4	4
ZANUBRUTINIB	4	4
LUSUTROMBOPAG	4	3
ROZANOLIXIZUMAB	4	3
TRETINOIN	4	3
BARICITINIB	4	2
TERBUTALINE	4	2
ALEMTUZUMAB	4	1
AMOXICILLIN	4	1
AZATHIOPRINE	4	1
BORTEZOMIB	4	1
CALCITRIOL	4	1
CLARITHROMYCIN	4	1
DECITABINE	4	1
HYDROXYCHLOROQUINE	4	1
IPTACOPAN	4	1
IXAZOMIB CITRATE	4	1
LACTIC ACID	4	1
LUSPATERCEPT	4	1
OSELTAMIVIR	4	1
PIRTOBRUTINIB	4	1
RITUXIMAB	4	1
UPADACITINIB	4	1