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autoimmune thrombocytopenic purpura - Clinical Profile, Treatments & Trials | Kisho | Kisho

Rare Disease

autoimmune thrombocytopenic purpura

MONDO:0008558

Also known as:immune thrombocytopeniaITPidiopathic thrombocytopenia

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Overview

An autoimmune disorder in which the number of circulating platelets is reduced due to their antibody-mediated destruction. ITP is a diagnosis of exclusion and is heterogeneous in origin.

GeneticsAutosomal dominant

Treatments5 FDA approved

Clinical Trials144 registered · 85 recruiting

Clinical Presentation

Built from Human Phenotype Ontology (HPO) phenotype annotations and GeneReviews clinical descriptions. Features represent findings documented across known cases — not an exhaustive list of possible symptoms.

Features include: Abnormal bleeding tendency (abnormal bleeding), Platelet antibody positive, and Low platelet count (thrombocytopenia).

Organ System	Phenotype Count	Example Features
Blood and immune system	3	Abnormal bleeding tendency (abnormal bleeding), Platelet antibody positive, Low platelet count (thrombocytopenia)

Diagnosis

Biomarker and diagnostic research for autoimmune thrombocytopenic purpura has been reported in the published literature.

Treatment

Compiled from FDA-approved drug records, orphan drug designations, and FDA Adverse Event Reporting System (FAERS) safety data. GeneReviews management guidance is included where available.

4 FDA-approved treatments are available for autoimmune thrombocytopenic purpura, including ELTROMBOPAG OLAMINE (PROMACTA, approved 2008), ROMIPLOSTIM (NPLATE, approved 2008), and Rho(D) Immune Globulin Intravenous (Human) (Rhophylac, approved 2004). An additional 14 compounds hold orphan drug designation.

Approved Therapies

Brand Name	Generic Name	Mechanism	Approved

Prognosis

Estimated prevalence: 1-5 in 10,000 (Uncommon).

Research Landscape

Sourced from ClinicalTrials.gov and PubMed. Publication types are classified using NLM-curated article metadata; research type breakdowns reflect the nature of published work for this disease.

144 clinical trials registered, 85 recruiting. Interventions under study include drug therapy, other interventions, biologic therapy, and procedural interventions. Pipeline includes 6 PHASE4, 21 PHASE3, 41 PHASE2. Research is sponsored by a mix of industry and academic institutions.

NCT ID	Title	Phase	Sponsor	Status
[NCT05338190](https://clinicaltrials.gov/study/NCT05338190)

Data assembled from 7 of 12 sources · Last updated Sep 20, 2026, 6:02 AM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Patient Support

Financial & Access Support (External Programs)

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Idiopathic Thrombocytopenic Purpura Fund

Premium/CopayClosedOperated by HealthWell Foundation

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Program availability and eligibility requirements are set by each foundation. Contact them directly to learn more about your options.

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Medical References

External Resources

OMIM

Online Mendelian Inheritance in Man

OMIM:188030

Orphanet

European rare disease database

Orphanet:3002

GARD

Genetic and Rare Diseases Info Center

GARD:5194

Questions for Your Care Team

→What are the benefits and potential side effects of PROMACTA for managing autoimmune thrombocytopenic purpura?
→Are any of the 144 active clinical trials a good fit for my situation?
→Are there specialists or centers of excellence for this condition in my area?
→Is there a patient registry I should consider joining?

← Back to All Diseases

Brand Name	Generic Name	Sponsor	Designated	Exclusivity End	Designation Status
Wayrilz	rilzabrutinib	Genzyme Corporation (A Sanofi Company)	2018	—	Designated (drug approved for other indication)
Rozanolixizumab	Rozanolixizumab	UCB, Inc.	2018	—	Designated
fostamatinib disodium hexahydrate	fostamatinib disodium hexahydrate	Rigel Pharmaceuticals, Inc.	2015	2025	Designated (drug approved for other indication)
veltuzumab	veltuzumab	Immunomedics, Inc.	2015	—	Designated
staphylococcal aureus protein A	staphylococcal aureus protein A	Protalex, Inc.	2015	—	Designated
PG2 (Astragalus polysaccharides extracted and purified from the dry root of Astragalus membranaceus)	PG2 (Astragalus polysaccharides extracted and purified from the dry root of Astragalus membranaceus)	PhytoHealth Corporation	2012	—	Designated
avatrombopag maleate	avatrombopag maleate	Sobi, Inc.	2011	2026	Designated (drug approved for other indication)
Recombinant Human soluble Fc-gamma Receptor IIb	Recombinant Human soluble Fc-gamma Receptor IIb	Baxalta US Inc., now part of Shire Plc	2010	—	Withdrawn
toralizumab	toralizumab	Biogen IDEC Inc.	2002	—	Withdrawn
rituximab	rituximab	Genentech, Inc.	2002	—	Withdrawn
Medroxyprogesterone acetate	Medroxyprogesterone acetate	ZaBeCor Pharmaceutical Company, LLC	2001	—	Designated
Recombinant humanized monclonal antibody 5c8	Recombinant humanized monclonal antibody 5c8	Biogen, Inc.	1998	—	Designated
Purified extract of Pseudomonas aeruginosa	Purified extract of Pseudomonas aeruginosa	Able Laboratories, Inc.	1997	—	Designated
WinRho SD	Rho (D) immune globulin intravenous (human)	Rh Pharmaceuticals, Inc.	1993	2002	Designated (drug approved for other indication)

Research Type	Count	% of Total
Research summaries	71	35%
Disease patterns and progression	33	16%
Laboratory research	32	16%
Patient case studies	24	12%
Clinical study results	19	9%
Other research	11	5%
Testing and diagnosis research	9	4%
New treatment approaches	6	3%

Kisho

autoimmune thrombocytopenic purpura

Overview

Clinical Presentation

Diagnosis

Treatment

Approved Therapies

Prognosis

Research Landscape

Patient Support

Financial & Access Support (External Programs)

Are you a patient organization for this condition?

Medical References

External Resources

OMIM

Orphanet

GARD

Questions for Your Care Team

Questions for Your Care Team

Explore clinical features

Orphan Drug Designations

Clinical Trials

Recent Publications

autoimmune thrombocytopenic purpura

Overview

Clinical Presentation

Diagnosis

Treatment

Approved Therapies

Prognosis

Research Landscape

Patient Support

Financial & Access Support (External Programs)

Are you a patient organization for this condition?

Medical References

External Resources

OMIM

Orphanet

GARD

Related Diseases

Questions for Your Care Team

Questions for Your Care Team

Explore clinical features

Orphan Drug Designations

Clinical Trials

Recent Publications