Kisho

Kisho is an information platform, not a medical provider. Nothing on this site constitutes medical advice, diagnosis, or treatment recommendations. All content is aggregated from publicly available sources (including ClinicalTrials.gov, PubMed, FDA.gov, and Orphanet) and is provided for informational purposes only. Clinical trial eligibility, treatment decisions, and any health-related actions should always be discussed with a qualified healthcare professional. Kisho does not endorse any specific therapy, organization, or clinical trial. Terms of use · Privacy policy

essential thrombocythemia (ET) - Clinical Profile, Treatments & Trials | Kisho | Kisho

Rare Disease

essential thrombocythemia

MONDO:0005029

Also known as:ETessential thrombocytemiaessential thrombocythaemia

Find Clinical TrialsTrials

Overview

A chronic myeloproliferative neoplasm that involves primarily the megakaryocytic lineage. It is characterized by sustained thrombocytosis in the blood, increased numbers of large, mature megakaryocytes in the bone marrow, and episodes of thrombosis and/or hemorrhage. (WHO, 2008)

Treatments3 FDA approved

Clinical Trials61 registered · 35 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 very common findings: Myocardial infarction, Abnormality of thrombocytes, Elevated platelet count (thrombocytosis), and Prolonged bleeding time and others; and common findings: Abnormal bleeding tendency (abnormal bleeding), Migraine, Enlarged spleen (splenomegaly), and Headache and others. 30 total HPO annotations.

Organ System	Phenotype Count	Example Features
Brain and nerves	7	Migraine, Paresthesia, Fatigue

Diagnosis

Biomarker and diagnostic research for essential thrombocythemia 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.

3 FDA-approved treatments are available for essential thrombocythemia, including MOMELOTINIB (OJJAARA, approved 2023), FEDRATINIB HYDROCHLORIDE (INREBIC, approved 2019), and ANAGRELIDE HYDROCHLORIDE (AGRYLIN, approved 1997). An additional 7 compounds hold orphan drug designation.

Approved Therapies

Brand Name	Generic Name	Mechanism	Approved

Prognosis

Phenotype severity distribution: 15 very common features, 6 common features.

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.

61 clinical trials registered, 35 recruiting. Interventions under study include drug therapy, other interventions, procedural interventions, and biologic therapy. Pipeline includes 10 PHASE3, 21 PHASE2, 14 PHASE1. Research is sponsored by a mix of industry and academic institutions.

NCT ID	Title	Phase	Sponsor	Status
[NCT06770842](https://clinicaltrials.gov/study/NCT06770842)

Data assembled from 6 of 12 sources · Last updated Sep 18, 2026, 3:00 PM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Medical References

External Resources

Orphanet

European rare disease database

Orphanet:3318

GARD

Genetic and Rare Diseases Info Center

GARD:6594

Questions for Your Care Team

→What are the benefits and potential side effects of OJJAARA for managing essential thrombocythemia?
→Are any of the 61 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
recombinant fully human IgG1 monoclonal antibody selectively targeting PD-L2	recombinant fully human IgG1 monoclonal antibody selectively targeting PD-L2	mAbTree Biologics AG	2026	—	Designated
peginterferon alfa-2a	peginterferon alfa-2a	Pharma& U.S. Inc.	2024	—	Designated
Jaktinib Dihydrochloride Monohydrate	Jaktinib Dihydrochloride Monohydrate	Suzhou Zelgen Biopharmaceuticals Co., Ltd.	2020	—	Designated
bomedemstat	bomedemstat	Merck & Co., Inc.	2018	—	Designated
pegylated proline interferon alpha-2b	pegylated proline interferon alpha-2b	PharmaEssentia	2014	—	Designated
ruxolitinib phosphate	ruxolitinib phosphate	Incyte Corporation	2010	—	Designated
Vonjo	pacritinib	Sobi, Inc.	2008	2029	Designated (drug approved for other indication)

Research Type	Count	% of Total
Disease patterns and progression	58	19%
Patient case studies	55	18%
Research summaries	54	18%
Laboratory research	52	17%
Clinical study results	33	11%
Testing and diagnosis research	30	10%
New treatment approaches	11	4%
Other research	8	3%

Kisho

essential thrombocythemia

Overview

Clinical Presentation

Diagnosis

Treatment

Approved Therapies

Prognosis

Research Landscape

Medical References

External Resources

Orphanet

GARD

Questions for Your Care Team

Questions for Your Care Team

Explore clinical features

Orphan Drug Designations

Clinical Trials

Recent Publications

Latest News & Developments

Cutaneous Clue to Clonal Evolution: Sweet's Syndrome Heralding Hematologic Progression in Essential Thrombocythemia.

Calreticulin mutations in essential thrombocythemia and primary myelofibrosis.

PharmaEssentia to Present New Clinical Data Supporting the Use of Ropeginterferon Alfa-2b in Essential Thrombocythemia at ASCO and EHA 2026 - BioSpace

MPN PROGRESSion Registry: Observational Study Tracking Symptoms, Treatments, and Disease Progression in People With Myeloproliferative Neoplasms (MPNs)

essential thrombocythemia

Overview

Clinical Presentation

Diagnosis

Treatment

Approved Therapies

Prognosis

Research Landscape

Medical References

External Resources

Orphanet

GARD

Related Diseases

Questions for Your Care Team

Questions for Your Care Team

Explore clinical features

Orphan Drug Designations

Clinical Trials

Recent Publications

Latest News & Developments

Cutaneous Clue to Clonal Evolution: Sweet's Syndrome Heralding Hematologic Progression in Essential Thrombocythemia.

Calreticulin mutations in essential thrombocythemia and primary myelofibrosis.

PharmaEssentia to Present New Clinical Data Supporting the Use of Ropeginterferon Alfa-2b in Essential Thrombocythemia at ASCO and EHA 2026 - BioSpace

MPN PROGRESSion Registry: Observational Study Tracking Symptoms, Treatments, and Disease Progression in People With Myeloproliferative Neoplasms (MPNs)