Cytosolic phospholipase-A2 alpha deficiency associated bleeding disorder

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ORPHA:477787OMIM:618372D69.1
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Overview

Cytosolic phospholipase-A2 alpha deficiency associated bleeding disorder, sometimes called cPLA2α deficiency, is a very rare inherited condition that affects how the body controls bleeding. It is caused by changes (mutations) in the PLA2G4A gene, which provides instructions for making an enzyme called cytosolic phospholipase-A2 alpha. This enzyme plays an important role in platelets — the tiny blood cells that help form clots to stop bleeding. When this enzyme does not work properly, platelets cannot function as they should, leading to problems with normal blood clotting. People with this condition tend to bleed more easily and for longer than usual. Common problems include easy bruising, prolonged bleeding from cuts or injuries, heavy menstrual periods in women, and bleeding after surgery or dental procedures. The condition is classified under platelet function disorders, meaning the number of platelets in the blood is usually normal, but the platelets do not work correctly. Treatment is mainly focused on managing bleeding episodes and preventing excessive blood loss during procedures. There is currently no cure. Doctors may use medications that help promote clotting, such as desmopressin (DDAVP) or antifibrinolytic drugs like tranexamic acid, and platelet transfusions may be needed in serious situations. Because this condition is extremely rare, most care is managed by hematologists with experience in platelet disorders.

Also known as:

Key symptoms:

Easy or unexplained bruisingProlonged bleeding from cuts or woundsHeavy or unusually long menstrual periodsBleeding that is hard to stop after surgery or dental workNosebleeds that last a long timeBleeding into the skin (small red or purple spots called petechiae)Gastrointestinal bleeding in some cases

Inheritance

Autosomal recessive

Passed on when both parents carry the same gene change; often skips generations

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 Cytosolic phospholipase-A2 alpha deficiency associated bleeding disorder.

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No actively recruiting trials found for Cytosolic phospholipase-A2 alpha deficiency associated bleeding disorder at this time.

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No specialists are currently listed for Cytosolic phospholipase-A2 alpha deficiency associated bleeding disorder.

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

Mental Health Support

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Social Security Disability

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Questions for your doctor

Bring these to your next appointment

  • Q1.What specific platelet function tests do I need, and how often should they be repeated?,Do I need a written bleeding management plan for emergencies and surgical procedures?,Which medications are safe for me to take, and which ones (like aspirin or ibuprofen) should I avoid?,Should my family members be tested for this condition?,What should I do before a dental procedure or surgery to reduce my bleeding risk?,Are there any clinical trials or research studies I could participate in?,How should I manage heavy menstrual bleeding, and what options are available?

Common questions about Cytosolic phospholipase-A2 alpha deficiency associated bleeding disorder

What is Cytosolic phospholipase-A2 alpha deficiency associated bleeding disorder?

Cytosolic phospholipase-A2 alpha deficiency associated bleeding disorder, sometimes called cPLA2α deficiency, is a very rare inherited condition that affects how the body controls bleeding. It is caused by changes (mutations) in the PLA2G4A gene, which provides instructions for making an enzyme called cytosolic phospholipase-A2 alpha. This enzyme plays an important role in platelets — the tiny blood cells that help form clots to stop bleeding. When this enzyme does not work properly, platelets cannot function as they should, leading to problems with normal blood clotting. People with this con

How is Cytosolic phospholipase-A2 alpha deficiency associated bleeding disorder inherited?

Cytosolic phospholipase-A2 alpha deficiency associated bleeding disorder follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.