Familial thrombocytosis

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ORPHA:71493OMIM:187950D68.8
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1Active trials16Specialists8Treatment centers

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

Familial thrombocytosis, also known as hereditary thrombocytosis or familial essential thrombocythemia, is a rare inherited blood disorder in which the body produces too many platelets. Platelets are tiny blood cells that help your blood clot when you have a cut or injury. In this condition, the platelet count stays persistently high — often well above the normal range — not because of another illness, but because of a change (mutation) in a gene that controls how blood cells are made in the bone marrow. Having too many platelets can cause the blood to clot too easily, which raises the risk of blood clots forming inside blood vessels. These clots can block blood flow to the brain, heart, or other organs. At the same time, some people with very high platelet counts can paradoxically experience unusual bleeding. Symptoms can include headaches, dizziness, tingling in the hands and feet, and in more serious cases, strokes or heart attacks. Treatment focuses on reducing the risk of dangerous clots. Doctors may recommend low-dose aspirin to thin the blood, and in higher-risk patients, medications that lower the platelet count such as hydroxyurea or anagrelide may be used. The condition is lifelong, but with careful monitoring and treatment, many people live relatively normal lives.

Also known as:

Key symptoms:

Abnormally high platelet count found on blood testsHeadachesDizziness or lightheadednessTingling or burning sensation in the hands and feetRedness and warmth in the hands or feetUnusual fatigueBlood clots in veins or arteriesEasy bruising or unusual bleedingEnlarged spleen causing discomfort in the upper left abdomenVision changes or temporary vision loss

Clinical phenotype terms (24)— hover any for plain English
ThrombocytosisHP:0001894Arterial thrombosisHP:0004420Transient ischemic attackHP:0002326Cerebral ischemiaHP:0002637Peripheral arterial stenosisHP:0004950
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 ↗OMIM ↗NORD ↗

FDA & Trial Timeline

1 event
Jul 2006Molecular Biology of Polycythemia and Thrombocytosis

University of Utah

TrialRECRUITING

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

Treatments

No FDA-approved treatments are currently listed for Familial thrombocytosis.

1 clinical trialare actively recruiting — trials can provide access to cutting-edge therapies.

View clinical trials →

Clinical Trials

1 recruitingView all trials with filters →
Other1 trial
Molecular Biology of Polycythemia and Thrombocytosis
Actively Recruiting
PI: Josef T. Prchal, MD (University of Utah) · Sites: Salt Lake City, Utah

Specialists

16 foundView all specialists →
TA
Talal Al-Harbi
Specialist
1 Familial thrombocytosis publication
MA
Mohsen Al-Zahrani
Specialist
1 Familial thrombocytosis publication
MA
Mohammed Al-Balwi
Specialist
1 Familial thrombocytosis publication
AA
Aiman Al-Hazmi
Specialist
1 Familial thrombocytosis publication
AA
Ahmed Alsuhaibani
Specialist
1 Familial thrombocytosis publication
NA
Nahed Aljafn
Specialist
1 Familial thrombocytosis publication
FA
Fatimah Alsumari
Specialist
1 Familial thrombocytosis publication
LA
Latefah Aleshaiwi
Specialist
1 Familial thrombocytosis publication
AA
Alanoud Alsuhibani
Specialist
1 Familial thrombocytosis publication
OA
Ohoud Alqasim
Specialist
1 Familial thrombocytosis publication
NA
Naveed Ahmad
Specialist
1 Familial thrombocytosis publication
GC
Gonzalo Carreño-Tarragona
Specialist
1 Familial thrombocytosis publication
LV
Leila N Varghese
Specialist
1 Familial thrombocytosis publication
ES
Elena Sebastián
Specialist
1 Familial thrombocytosis publication
EG
Eva Gálvez
Specialist
1 Familial thrombocytosis publication
JM
Josef T. Prchal, MD
SALT LAKE CITY, UT
Specialist
PI on 1 active trial

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

Travel Grants

No travel grants are currently matched to Familial thrombocytosis.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Familial thrombocytosis

Disease timeline:

New recruiting trial: Molecular Biology of Polycythemia and Thrombocytosis

A new clinical trial is recruiting patients for Familial thrombocytosis

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.

Questions for your doctor

Bring these to your next appointment

  • Q1.Which gene mutation is causing my condition, and what does that mean for my family members?,What is my personal risk level for blood clots, and how will you decide on my treatment plan?,Should my children or siblings be tested for this condition?,What symptoms should prompt me to go to the emergency room immediately?,Are there any activities, medications, or foods I should avoid?,How often do I need blood tests and check-ups?,If I want to become pregnant, what special precautions do I need to take?

Common questions about Familial thrombocytosis

What is Familial thrombocytosis?

Familial thrombocytosis, also known as hereditary thrombocytosis or familial essential thrombocythemia, is a rare inherited blood disorder in which the body produces too many platelets. Platelets are tiny blood cells that help your blood clot when you have a cut or injury. In this condition, the platelet count stays persistently high — often well above the normal range — not because of another illness, but because of a change (mutation) in a gene that controls how blood cells are made in the bone marrow. Having too many platelets can cause the blood to clot too easily, which raises the risk o

How is Familial thrombocytosis inherited?

Familial thrombocytosis follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

Are there clinical trials for Familial thrombocytosis?

Yes — 1 recruiting clinical trial is currently listed for Familial thrombocytosis on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Familial thrombocytosis?

16 specialists and care centers treating Familial thrombocytosis are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.