Essential thrombocythemia

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ORPHA:3318OMIM:187950D47.3
Who is this for?
Show terms as
2FDA treatments38Active trials77Specialists8Treatment centers5Financial resources

Where are you in your journey?

UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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Overview

Essential Thrombocythemia is treated with 3 medications in our database, including Anagrelide, anagrelide, FERRIPROX. 1 of these has manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Takeda, Chiesi reinforcement _Self-correction:_ The user asked for the primary manufacturer/company that owns the rights. Chiesi is the entity. Final string: Chiesi. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Essential Thrombocythemia treatment below.

Also known as:

Clinical phenotype terms— hover any for plain English:

Abnormality of thrombocytesHP:0001872ThrombocytosisHP:0001894Prolonged bleeding timeHP:0003010Arterial thrombosisHP:0004420Increased megakaryocyte countHP:0005513Abnormal bone marrow cell morphologyHP:0005561Abnormal platelet morphologyHP:0011875Megakaryocyte nucleus hyperlobulationHP:0031388Amaurosis fugaxHP:0100576Abnormal cerebral vascular morphologyHP:0100659
Inheritance

Sporadic

Usually appears on its own, not inherited from a parent

Age of Onset

Adult

Begins in adulthood (age 18 or older)

Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

10 events
Jun 2026WELL-MPN: A Digital Lifestyle App for Patients With MPNs

Brigham and Women's Hospital — NA

TrialNOT YET RECRUITING
May 2026Study of Pelabresib add-on to Ruxolitinib in Japanese Adult Patients With Myelofibrosis

Novartis Pharmaceuticals — PHASE1

TrialNOT YET RECRUITING
May 2026Selinexor and Pacritinib in JAK Inhibitor-naïve MF Patients With Cytopenias

John Mascarenhas — PHASE2

TrialNOT YET RECRUITING
Apr 2026A Phase 3 Study of Pelabresib (DAK539) and Ruxolitinib in Myelofibrosis (MF)

Novartis Pharmaceuticals — PHASE3

TrialNOT YET RECRUITING
Apr 2026A Phase 1 Study of PRT12396 in Participants With Select Myeloproliferative Neoplasms

Prelude Therapeutics — PHASE1

TrialRECRUITING
Feb 2026Registry Platform Myelofibrosis and Anemia

iOMEDICO AG

TrialRECRUITING
Dec 2025A ELN-Multicenter Study on Phenotypic Evolution and Clinical Outcomes

FROM- Fondazione per la Ricerca Ospedale di Bergamo- ETS

TrialRECRUITING
Oct 2025A Study to Evaluate INCA035784 in Participants With Myeloproliferative Neoplasms

Incyte Corporation — PHASE1

TrialRECRUITING
Jun 2025A Phase 1 Study to Assess STP938 as a Monotherapy in Adults With High Risk Essential Thrombocythaemia

Step Pharma, SAS — PHASE1

TrialRECRUITING
Feb 2025Study of Momelotinib in Combination With Luspatercept in Participants With Transfusion Dependent Myelofibrosis

GlaxoSmithKline — PHASE2

TrialRECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

2 available

Agrylin

Anagrelide· Takeda Development Center Americas, Inc.Orphan Drug

Treatment of patients with essential thrombocythemia to reduce the elevated platelet count and the risk of thrombosis and to ameliorate associated symptoms.

FERRIPROX

deferiprone· Chiesi reinforcement _Self-correction:_ The user asked for the primary manufacturer/company that owns the rights. Chiesi is the entity. Final string: Chiesi

Detailed copay and financial assistance information is not publicly available for this medication at this time. Please consult your pharmacist or the manufacturer's official patient support program fo

Clinical Trials

20 recruitingView all trials with filters →
Phase 36 trials
Phase 3 Study of Pelabresib (CPI-0610) in Myelofibrosis (MF) (MANIFEST-2)
Phase 3
Active
PI: Novartis Pharmaceuticals (Novartis Pharmaceuticals) · Sites: Birmingham, Alabama; Chandler, Arizona +154 more · Age: 1899 yrs
Ropeginterferon Alfa-2b (P1101) vs. Anagrelide in Essential Thrombocythemia Patients With Hydroxyurea Resistance or Intolerance
Phase 3
Active
PI: Toshiaki Sato, MD/PhD (PharmaEssentia Japan K.K.) · Sites: St Louis, Missouri; Houston, Texas +63 more · Age: 1899 yrs
A Study to Evaluate Safety and Efficacy of Bomedemstat (MK-3543-017)
Phase 3
Actively Recruiting
PI: Medical Director (Merck Sharp and Dohme LLC) · Sites: Ann Arbor, Michigan; Durham, North Carolina +19 more · Age: 1899 yrs
Bomedemstat vs Hydroxyurea for Essential Thrombocythemia (MK-3543-007)
Phase 3
Actively Recruiting
PI: Medical Director (Merck Sharp & Dohme LLC) · Sites: Glendale, Arizona; Glendale, California +164 more · Age: 1899 yrs
AVAJAK: Apixaban/Rivaroxaban Versus Aspirin for Primary Prevention of Thrombo-embolic Complications in JAK2V617F-positive Myeloproliferative Neoplasms
Phase 3
Actively Recruiting
· Sites: Angers; Annecy +40 more · Age: 1899 yrs
A Study of Bomedemstat (IMG-7289/MK-3543) Compared to Best Available Therapy (BAT) in Participants With Essential Thrombocythemia and an Inadequate Response or Intolerance of Hydroxyurea (MK-3543-006)
Phase 3
Actively Recruiting
PI: Medical Director (Merck Sharp & Dohme LLC) · Sites: Glendale, Arizona; Glendale, California +159 more · Age: 1899 yrs
Phase 26 trials
Pegylated Interferon Alfa-2b Versus Interferon Alfa Therapy in Childhood and Adolescent Essential Thrombocythemia
Phase 2
Actively Recruiting
PI: Lei Zhang, MD (Institute of Hematology & Blood Diseases Hospital,) · Sites: Tianjin, Tianjin Municipality · Age: 019 yrs
Hematology, IMG-7289, LSD1 (Lysine-Specific Demethylase 1) Inhibitor, Essential Thrombocythemia (ET), Ph 2
Phase 2
Active
PI: Zohra Nooruddin, MD (Mays Cancer Center) · Sites: San Antonio, Texas · Age: 1899 yrs
IMG-7289 in Patients With Essential Thrombocythemia (ET) or Polycythemia Vera (PV)
Phase 2
Active
PI: Terrence J Bradley, MD (University of Miami) · Sites: Miami, Florida · Age: 1899 yrs
Reparixin in Patients With Myelofibrosis Myeloproliferative Neoplasms Research Consortium (MPN-RC 120)
Phase 2
Actively Recruiting
PI: Marina Kremyanskaya, PhD, MD (Icahn School of Medicine at Mount Sinai) · Sites: Tampa, Florida; Atlanta, Georgia +7 more · Age: 1899 yrs
Study of Momelotinib in Combination With Luspatercept in Participants With Transfusion Dependent Myelofibrosis
Phase 2
Actively Recruiting
· Sites: Nashville, Tennessee; Toronto, Ontario +12 more · Age: 1899 yrs
Ruxolitinib in Thrombocythemia and Polycythemia Vera
Phase 2
Actively Recruiting
PI: Gabriela Hobbs, MD (Massachusetts General Hospital) · Sites: Boston, Massachusetts; Boston, Massachusetts +2 more · Age: 1899 yrs
Other8 trials
A ELN-Multicenter Study on Phenotypic Evolution and Clinical Outcomes
Actively Recruiting
PI: TIZIANO BARBUI, MD (FROM- Fondazione per la Ricerca Ospedale di Bergam) · Sites: Rochester, Minnesota; Halle +23 more · Age: 18100 yrs
Unraveling the Role of Extracellular Vesicles-driven Senescence in Myeloproliferative Neoplasms
Active
PI: Lucia Catani, MD (IRCCS Azienda Ospedaliero-Universitaria di Bologna) · Sites: Bologna, Bologna; Brescia, BS +3 more · Age: 1899 yrs
Identification of Factors Associated With Treatment Response in Patients With Polycythemia Vera, Essential Thrombocythemia, and Pre-myelofibrosis.
Actively Recruiting
· Sites: Angers; Brest +4 more · Age: 1899 yrs
Registry Platform Myelofibrosis and Anemia
Actively Recruiting
PI: Konstanze Döhner, Prof. Dr. med. (University Hospital Ulm, Germany) · Sites: Multiple Locations · Age: 1899 yrs
A Post-Marketing Surveillance Study to Assess the Safety of Fedratinib in Korean Patients With Myelofibrosis
Actively Recruiting
PI: Bristol-Myers Squibb (Bristol-Myers Squibb) · Sites: Seoul; Seoul +1 more · Age: 1999 yrs
Ruxolitinib in Primary Myelofibrosis and Secondary to Essential Thrombocythemia or Polycythemia Vera
Actively Recruiting
· Sites: Cosenza, Calabria; Reggio Calabria, Calabria +24 more · Age: 1899 yrs
Myeloproliferative Neoplastic Diseases Observatory From Brest
Actively Recruiting
PI: Jean-Christophe Ianotto (CHRU de Brest) · Sites: Brest
Asian Myeloproliferative Neoplasm (MPN) Registry
Actively Recruiting
PI: Harinder Gill, MD (The University of Hong Kong) · Sites: Hong Kong · Age: 1899 yrs

Specialists

Showing 25 of 77View all specialists →
SM
Srdan Verstovsek, MD
HOUSTON, TX
Specialist
PI on 9 active trials
NP
Naveen Pemmaraju
HOUSTON, TX
Specialist
PI on 3 active trials10 Essential thrombocythemia publications
GM
Gabriela Hobbs, MD
BOSTON, MA
Specialist
PI on 4 active trials1 Essential thrombocythemia publication
CD
Claire Harrison, Dr
Specialist
PI on 1 active trial
AP
Anthony R Green, Prof
Specialist
PI on 1 active trial
AP
Anand A Patel
Specialist
PI on 1 active trial256 Essential thrombocythemia publications
SS
Simran Singh
Birmingham, Alabama
Specialist

Rare Disease Specialist

PI on 3 active trials428 Essential thrombocythemia publications
LP
Lesley A Anderson, PhD
Specialist
PI on 1 active trial
ZP
ZhenYa Hong, Ph.D
Specialist
PI on 1 active trial
AM
Ales Blinc, MD
Specialist
PI on 1 active trial1 Essential thrombocythemia publication
PM
Paul Martin
Specialist
PI on 3 active trials73 Essential thrombocythemia publications
SP
Srdan Verstovsek, MD, PhD
HOUSTON, TX
Specialist
PI on 3 active trials
GP
Gang Lu, Ph.D.
CLEVELAND, OH
Specialist
PI on 1 active trial
JM
John Mascarenhas, M.D.
NEW YORK, NY
Specialist
PI on 1 active trial
SP
Srdan Verstovsek, M.D., Ph.D.
HOUSTON, TX
Specialist
PI on 2 active trials
RP
Raajit Rampal, MD, PhD
Chicago, Illinois
Specialist

Rare Disease Specialist

PI on 4 active trials

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

Financial Resources

1 resources
Agrylin(Anagrelide)Takeda Development Center Americas, Inc.

4 travel grants are also available for Essential thrombocythemia patients — see Travel Grants below ↓

Travel Grants

4 grants

CancerCare Co-pay Assistance for Myeloproliferative Neoplasms

CancerCare

Patients must be diagnosed with Myeloproliferative Neoplasms (ET, PV, or MF) and require financial assistance for treatment, co-pays, or clinical trial-related travel. Specific criteria vary by partner organization, with some requiring household income at or below 500% of the federal poverty level.

Apply ↗

Takeda Oncology Co-Pay Assistance Progam

Takeda Oncology

Apply ↗

Takeda Here2Assist Program

Takeda Oncology

Patients must be US residents at least 18 years old with commercial insurance and a valid prescription for ALUNBRIG. The co-pay assistance program explicitly excludes patients covered by government-sponsored programs like Medicare or Medicaid, although these patients may receive help finding third-party support.

Apply ↗

Ferriprox Patient Enrollment (Chiesi USA)

Patients must be U.S. residents with a valid prescription for a Chiesi product and a completed provider start form on file. Financial assistance is restricted to those with commercial insurance, excluding beneficiaries of government-funded programs like Medicare or Medicaid.

Apply ↗

Community

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Latest news about Essential thrombocythemia

1 articles
Clinical trialUNITERAREApr 3, 2026
New Recruiting Trial: A Phase 1 Study of PRT12396 in Participants With Select Myeloproliferative Neoplasms
Prelude Therapeutics is testing a new drug called PRT12396 in people with certain blood cancers called myeloproliferative neoplasms. This is an early-stage stud
See all news about Essential thrombocythemia

Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Common questions about Essential thrombocythemia

What is Essential thrombocythemia?

Essential Thrombocythemia is treated with 3 medications in our database, including Anagrelide, anagrelide, FERRIPROX. 1 of these has manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Takeda, Chiesi reinforcement _Self-correction:_ The user asked for the primary manufacturer/company that owns the rights. Chiesi is the entity. Final string: Chiesi. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Essential Thrombocythemia treatment below.

How is Essential thrombocythemia inherited?

Essential thrombocythemia follows a sporadic inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Essential thrombocythemia typically begin?

Typical onset of Essential thrombocythemia is adult. Age of onset can vary across affected individuals.

Are there clinical trials for Essential thrombocythemia?

Yes — 20 recruiting clinical trials are currently listed for Essential thrombocythemia on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Essential thrombocythemia?

25 specialists and care centers treating Essential thrombocythemia are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.

What treatment and support options exist for Essential thrombocythemia?

2 FDA-approved treatments and 3 patient support programs are currently tracked on UniteRare for Essential thrombocythemia. See the treatments and support programs sections for copay assistance, eligibility, and contact details.