Autosomal thrombocytopenia with normal platelets

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ORPHA:168629OMIM:616216D69.4
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Overview

Autosomal thrombocytopenia with normal platelets is a rare inherited blood disorder in which a person has a lower-than-normal number of platelets (the tiny blood cells that help with clotting), yet the platelets that are present appear normal in size and function. This condition is passed down through families in an autosomal pattern, meaning it can be inherited from either parent regardless of sex. Because the platelet count is reduced, people with this condition may experience easy bruising, prolonged bleeding after injuries or surgery, and sometimes spontaneous bleeding such as nosebleeds or bleeding gums. However, because the platelets themselves work normally, bleeding symptoms can sometimes be milder than in other forms of thrombocytopenia where platelet function is also impaired. This condition is often discovered incidentally during routine blood tests or when unexplained bruising or bleeding is investigated. It is important to distinguish this disorder from other causes of low platelet counts, such as immune thrombocytopenia (ITP), bone marrow disorders, or medication side effects. Diagnosis typically involves a thorough family history, complete blood count, examination of a blood smear under a microscope, and genetic testing. Treatment depends on the severity of symptoms. Many individuals with mild cases may not need regular treatment but should take precautions before surgeries or dental procedures. For those with more significant bleeding, treatments may include platelet transfusions or medications that help reduce bleeding risk.

Key symptoms:

Easy bruisingProlonged bleeding after cuts or injuriesNosebleeds that are hard to stopBleeding gumsHeavy or prolonged menstrual periods in womenExcessive bleeding after surgery or dental workSmall red or purple spots on the skin (petechiae)Bleeding into the skin causing larger purple patches (purpura)Fatigue related to blood loss in severe cases

Inheritance

Variable

Can be inherited in different ways depending on the underlying gene

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Autosomal thrombocytopenia with normal platelets.

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No actively recruiting trials found for Autosomal thrombocytopenia with normal platelets at this time.

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Specialists

1 foundView all specialists →

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

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Community

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Caregiver Resources

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Mental Health Support

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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.How severe is my (or my child's) thrombocytopenia, and what platelet count level should I be concerned about?,What medications and over-the-counter drugs should I avoid?,What should I do if I experience significant bleeding or an injury?,Do I need any special preparation before surgery, dental work, or childbirth?,Should my family members be tested for this condition?,How often should I have my platelet count checked?,Are there any activities or sports I should avoid?

Common questions about Autosomal thrombocytopenia with normal platelets

What is Autosomal thrombocytopenia with normal platelets?

Autosomal thrombocytopenia with normal platelets is a rare inherited blood disorder in which a person has a lower-than-normal number of platelets (the tiny blood cells that help with clotting), yet the platelets that are present appear normal in size and function. This condition is passed down through families in an autosomal pattern, meaning it can be inherited from either parent regardless of sex. Because the platelet count is reduced, people with this condition may experience easy bruising, prolonged bleeding after injuries or surgery, and sometimes spontaneous bleeding such as nosebleeds o

Which specialists treat Autosomal thrombocytopenia with normal platelets?

1 specialists and care centers treating Autosomal thrombocytopenia with normal platelets are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.