OBSOLETE: Inherited predisposition to essential thrombocythemia

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Overview

Inherited predisposition to essential thrombocythemia (ET) refers to a genetic tendency that runs in families, making certain individuals more likely to develop essential thrombocythemia. Essential thrombocythemia is a blood disorder in which the bone marrow produces too many platelets — the blood cells that help with clotting. When platelet counts are too high, it can lead to abnormal blood clotting or, paradoxically, bleeding problems. This condition is sometimes called familial essential thrombocythemia or hereditary thrombocythemia. It is important to note that this Orphanet entry is now classified as 'OBSOLETE,' meaning the concept has been retired or merged into other disease classifications. However, the underlying condition — familial forms of essential thrombocythemia — is still recognized in clinical practice. People with this inherited predisposition may develop symptoms such as headaches, dizziness, visual disturbances, burning or tingling in the hands and feet, and an increased risk of blood clots (thrombosis) or unusual bleeding. Some individuals remain symptom-free for years and are only discovered through routine blood tests showing elevated platelet counts. Treatment focuses on reducing the risk of clotting events and managing symptoms. Low-dose aspirin is commonly used to prevent clots. In higher-risk patients, medications that lower platelet counts — such as hydroxyurea or anagrelide — may be prescribed. Interferon-alpha is another option, particularly for younger patients or those planning pregnancy. Regular monitoring of blood counts and careful management by a hematologist are essential for long-term care.

Also known as:

Key symptoms:

High platelet count found on blood testsHeadachesDizziness or lightheadednessVisual disturbances such as blurred visionBurning or tingling in hands and feet (erythromelalgia)Redness and warmth in hands or feetBlood clots in veins or arteries (thrombosis)Unusual or excessive bleedingFatigue or tirednessEnlarged spleenNight sweatsBruising easilyNumbness in fingers or toes

Inheritance

Autosomal dominant

Passed on from just one parent; each child has about a 50% chance of inheriting it

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗NORD ↗

Treatments

1 available

Vonjo

PACRITINIB· Sobi, Inc.Accelerated Approval

indicated for the treatment of adults with intermediate or high-risk secondary (post-essential thrombocythemia) myelofibrosis (MF) with a platelet count below 50 × 10 9 /L

No actively recruiting trials found for OBSOLETE: Inherited predisposition to essential thrombocythemia at this time.

New trials open frequently. Follow this disease to get notified.

Search ClinicalTrials.gov ↗Join the OBSOLETE: Inherited predisposition to essential thrombocythemia community →

No specialists are currently listed for OBSOLETE: Inherited predisposition to essential thrombocythemia.

View NORD Rare Disease Centers ↗Undiagnosed Disease Network ↗

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
Vonjo(PACRITINIB)Sobi, Inc.

Travel Grants

No travel grants are currently matched to OBSOLETE: Inherited predisposition to essential thrombocythemia.

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Latest news about OBSOLETE: Inherited predisposition to essential thrombocythemia

2 articles
NewsFDA
FDA Approves VONJO
VONJO (PACRITINIB CITRATE) received FDA approval.
NewsFDA
FDA Approves OJJAARA
OJJAARA (MOMELOTINIB DIHYDROCHLORIDE) received FDA approval.
See all news about OBSOLETE: Inherited predisposition to essential thrombocythemia

Caregiver Resources

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

Questions for your doctor

Bring these to your next appointment

  • Q1.What is my current risk level for blood clots, and how will you monitor it?,Should my family members be tested for this genetic predisposition?,What medications do you recommend, and what are their side effects?,How often will I need blood tests and follow-up appointments?,Are there any activities, medications, or foods I should avoid?,What are the warning signs that I should go to the emergency room?,Is there a risk that this condition could progress to a more serious blood disorder?

Common questions about OBSOLETE: Inherited predisposition to essential thrombocythemia

What is OBSOLETE: Inherited predisposition to essential thrombocythemia?

Inherited predisposition to essential thrombocythemia (ET) refers to a genetic tendency that runs in families, making certain individuals more likely to develop essential thrombocythemia. Essential thrombocythemia is a blood disorder in which the bone marrow produces too many platelets — the blood cells that help with clotting. When platelet counts are too high, it can lead to abnormal blood clotting or, paradoxically, bleeding problems. This condition is sometimes called familial essential thrombocythemia or hereditary thrombocythemia. It is important to note that this Orphanet entry is now

How is OBSOLETE: Inherited predisposition to essential thrombocythemia inherited?

OBSOLETE: Inherited predisposition to essential thrombocythemia follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.