Disease · MONDO_0008558

autoimmune thrombocytopenic purpura

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. autoimmune thrombocytopenic purpura. Diseases, record MONDO_0008558. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0008558

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Disease

Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.

Name: autoimmune thrombocytopenic purpura; Open Targets
Open Targets ID: MONDO_0008558; Open Targets
Other names: ITP, Immune thrombocytopenic purpura (disorder), Purpura, Thrombocytopenic, Idiopathic, idiopathic thrombocytopenia, idiopathic thrombocytopenia purpura, idiopathic thrombocytopenic purpura, primary thrombocytopenic purpura, thrombocytopenic purpura, autoimmune; Open Targets
Disease Ontology: DOID:8924; Disease Ontology
NCI Thesaurus: C3446; NCI Thesaurus
Orphanet: ORPHA:3002; Orphanet
Highest stage of its drugs: Approval, for 15 of its 105 drugs, from DailyMed, EMA Human Drugs, FDA, PMDA and TTD; Open Targets

Drugs105

The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.

Drug	ChEMBL ID	Type	Open Targets' stage	Stage reported by	Reports	Of them trials	Trials shown here	Source
DEXAMETHASONE	CHEMBL384467	Small molecule	Approval	DailyMed	50	43	42	Drugs and targets
ELTROMBOPAG	CHEMBL461101	Small molecule	Approval	EMA Human Drugs	44	40	40	Drugs and targets
ROMIPLOSTIM	CHEMBL1201832	Protein	Approval	DailyMed and EMA Human Drugs	28	26	25	Drugs and targets
HUMAN IMMUNOGLOBULIN G	CHEMBL1201599		Approval	EMA Human Drugs	26	22	3	Drugs and targets
PREDNISONE	CHEMBL635	Small molecule	Approval	DailyMed	119	13	12	Drugs and targets
RILZABRUTINIB	CHEMBL3702854	Small molecule	Approval	EMA Human Drugs	5	4	4	Drugs and targets
FOSTAMATINIB DISODIUM	CHEMBL3989516	Small molecule	Approval	FDA	4	3	3	Drugs and targets
GMA-161	CHEMBL4297961		Approval	TTD	2	1	1	Drugs and targets
CORTISONE ACETATE	CHEMBL1650	Small molecule	Approval	DailyMed	2	0	0	Drugs and targets
DEXAMETHASONE SODIUM PHOSPHATE	CHEMBL2021430	Small molecule	Approval	DailyMed	2	0	0	Drugs and targets

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Targets124

The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.

Target	Protein	Its drugs that act on it	Mechanisms	Highest stage of those drugs	Gene in Genes	Source
NR3C1	Glucocorticoid receptor	10	Glucocorticoid receptor agonist	Approval	https://conductscience.org/library/genes/2908	Drugs and targets
MPL	Thrombopoietin receptor	8	Thrombopoietin receptor agonist	Approval	https://conductscience.org/library/genes/4352	Drugs and targets
BTK	Tyrosine-protein kinase BTK	4	Tyrosine-protein kinase BTK inhibitor	Approval	https://conductscience.org/library/genes/695	Drugs and targets
ATP4A	Potassium-transporting ATPase	3	Potassium-transporting ATPase inhibitor	Approval	https://conductscience.org/library/genes/495	Drugs and targets
ATP4B	Potassium-transporting ATPase	3	Potassium-transporting ATPase inhibitor	Approval	https://conductscience.org/library/genes/496	Drugs and targets
CSF3R	Granulocyte colony-stimulating factor receptor	3	Granulocyte colony stimulating factor receptor agonist	Unknown	https://conductscience.org/library/genes/1441	Drugs and targets
EPOR	Erythropoietin receptor	3	Erythropoietin receptor agonist	Unknown	https://conductscience.org/library/genes/2057	Drugs and targets
FCGRT	IgG receptor FcRn large subunit p51	3	IgG receptor FcRn large subunit p51 antagonist; IgG receptor FcRn large subunit p51 inhibitor	Phase 3	https://conductscience.org/library/genes/2217	Drugs and targets
MS4A1	B-lymphocyte antigen CD20	3	B-lymphocyte antigen CD20 binding agent	Phase 3	https://conductscience.org/library/genes/931	Drugs and targets
SYK	Tyrosine-protein kinase SYK	3	Tyrosine-protein kinase SYK inhibitor	Approval	https://conductscience.org/library/genes/6850	Drugs and targets

1 to 10 of 124

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Trials253

Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs. 21 more are not shown, since the checks do not confirm them.

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT03219723	Drug Use Surveillance of Takecab for "Supplement to Helicobacter Pylori Eradication"		560	2019-03-22	Gastric/Duodenal Ulcer, Gastric MALT Lymphoma, Idiopathic Thrombocytopenic Purpura, or H. Pylori Gastritis, and Other	AMOXICILLIN, CLARITHROMYCIN, METRONIDAZOLE, VONOPRAZAN	Its record names the disease	ClinicalTrials.gov
NCT00508820	An Open Label Study of Romiplostim in Adult Thrombocytopenic Subjects With ITP	Phase 3	407	2012-02-13	Idiopathic Thrombocytopenic Purpura, Thrombocytopenia, Thrombocytopenic Purpura	ROMIPLOSTIM	Its record names the disease	ClinicalTrials.gov
NCT00351468	EXTEND (Eltrombopag Extended Dosing Study)	Phase 3	302	2017-04-17	Purpura, Thrombocytopaenic, Idiopathic	ELTROMBOPAG	Open Targets' reading of its record	ClinicalTrials.gov
NCT00415532	Romiplostim (AMG 531) Versus Medical Standard of Care for Immune (Idiopathic) Thrombocytopenic Purpura	Phase 3	234	2013-07-08	Idiopathic Thrombocytopenic Purpura, Thrombocytopenia, Thrombocytopenia in Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP), Thrombocytopenic Purpura	ROMIPLOSTIM	Its record names the disease	ClinicalTrials.gov
NCT04562766	Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP)	Phase 3	232	2026-02-23	Immune Thrombocytopenia	RILZABRUTINIB	Open Targets' reading of its record	ClinicalTrials.gov
NCT04687072	A Study to Evaluate the Efficacy and Safety of Efgartigimod PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia	Phase 3	207	2024-10-31	Primary Immune Thrombocytopenia	EFGARTIGIMOD ALFA	Open Targets' reading of its record	ClinicalTrials.gov
NCT02279173	Long-term Study of Romiplostim in Thrombocytopenic Pediatric Patients With Immune Thrombocytopenia (ITP)	Phase 3	203	2019-08-28	Immune Thrombocytopenia	ROMIPLOSTIM	Open Targets' reading of its record	ClinicalTrials.gov
NCT00370331	RAISE: Randomized Placebo-Controlled Idiopathic Thrombocytopenic Purpura (ITP) Study With Eltrombopag	Phase 3	197	2010-02-23	Purpura, Thrombocytopaenic, Idiopathic	ELTROMBOPAG	Its record names the disease	ClinicalTrials.gov
NCT00907478	Study on Bone Marrow Morphology in Adults Receiving Romiplostim for Treatment of Thrombocytopenia Associated With Immune Thrombocytopenia Purpura (ITP)	Phase 4	169	2015-01-01	Thrombocytopenia, Idiopathic Thrombocytopenic Purpura	ROMIPLOSTIM	Its record names the disease	ClinicalTrials.gov
NCT01762761	Eltrombopag Phase III Study In Chinese Chronic ITP Patients	Phase 3	155	2015-03-09	Purpura, Thrombocytopenic, Idiopathic and Hepatitis C	ELTROMBOPAG	Open Targets' reading of its record	ClinicalTrials.gov

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Comparisons in its trials70

Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.

Comparison	Group 1, with the outcome	Group 2, with the outcome	Participants	Trial	Source
Eltrombopag, Treatment + 1 Day vs Eltrombopag, >1 to 30 Days Post-Therapy: serious adverse events	96 of 302	10 of 302	604	NCT00351468	Trial comparisons
Romiplostim (AMG 531) Cohort 1 vs Romiplostim (AMG 531) Cohort 2: Adverse Events	162 of 168	215 of 239	407	NCT00508820	Trial comparisons
Romiplostim (AMG 531) Cohort 1 vs Romiplostim (AMG 531) Cohort 2: Platelet Response (Definition 2)	160 of 168	220 of 239	407	NCT00508820	Trial comparisons
Romiplostim (AMG 531) Cohort 1 vs Romiplostim (AMG 531) Cohort 2: Platelet Response (Definition 1)	156 of 168	214 of 239	407	NCT00508820	Trial comparisons
Romiplostim Cohort 1 vs Romiplostim Cohort 2: serious adverse events	61 of 168	61 of 239	407	NCT00508820	Trial comparisons
AMG 531 vs Standard of Care: serious adverse events	36 of 156	28 of 73	229	NCT00415532	Trial comparisons
Efgartigimod PH20 SC vs Placebo PH20 SC: deaths from any cause	1 of 137	0 of 70	207	NCT04687072	Trial comparisons
Efgartigimod PH20 SC vs Placebo PH20 SC: serious adverse events	14 of 137	10 of 70	207	NCT04687072	Trial comparisons
Eltrombopag 50 mg QD vs Placebo: serious adverse events	16 of 135	11 of 61	196	NCT00370331	Trial comparisons
Eltrombopag vs Placebo: deaths from any cause	0 of 104	3 of 51	155	NCT01762761	Trial comparisons

1 to 10 of 70

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Papers from its trials137

The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.

PMID	Listed as	Trials listing it	Source
41411046	RESULT	1	PubMed
40828490	RESULT	1	PubMed
40418942	RESULT	1	PubMed
40392465	RESULT	1	PubMed
39552477	RESULT	1	PubMed
36658588	RESULT	1	PubMed
35973190	RESULT	1	PubMed
35288899	RESULT	1	PubMed
35201417	RESULT	1	PubMed
34487679	RESULT	1	PubMed

1 to 10 of 137

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Data dates

The date of each source this release read.

Open Targets: 2026-09-18; Open Targets
Drug links and their checks, from Drugs and targets: 2026-09-18; Drugs and targets
ClinicalTrials.gov, from Trials: 2026-11-12; Trials
Comparisons, from Trial comparisons: 2026-03-23; Trial comparisons
PubMed, for the checks: 2026-11-06; PubMed
MeSH, for the checks: 2026-11-10; MeSH

Sources 6

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ChEMBL, through Open TargetsCC BY-SA 3.0
ClinicalTrials.govNo license stated
NLM's MeSHNLM's terms
Open TargetsCC0 1.0
PubMedNLM's terms

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
ClinicalTrials.govnone stated
NLM's MeSHNLM's
Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
PubMedNLM's terms

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this disease from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/diseases/v1/MONDO_0008558

GET https://conductscience.org/api/diseases/v1/MONDO_0008558/drugs?page=2

API reference For AI assistants