Overview
Autoimmune thrombocytopenia, also commonly known as immune thrombocytopenia (ITP) or immune thrombocytopenic purpura, is a condition where the body's immune system mistakenly attacks and destroys its own platelets. Platelets are tiny blood cells that help your blood clot when you have a cut or injury. When platelet counts drop too low, the body has trouble stopping bleeding, even from minor bumps or cuts. People with this condition may notice easy bruising, small red or purple dots on the skin called petechiae, or bleeding from the gums and nose. Women may experience heavier-than-normal menstrual periods. In most cases, the condition develops without a clear cause, though it can sometimes be triggered by a viral infection, medication, or another underlying autoimmune disease. Treatment depends on how low the platelet count is and whether the person is having significant bleeding. Many people with mild cases need only careful monitoring. Others may need medications to suppress the immune system or boost platelet production. Most children with ITP recover fully on their own, while adults are more likely to have a long-term form of the disease. With proper care, most people with autoimmune thrombocytopenia can live full, active lives.
Key symptoms:
Easy or unexplained bruisingTiny red or purple pinpoint spots on the skin (petechiae)Bleeding gumsFrequent or hard-to-stop nosebleedsHeavier than normal menstrual periodsBlood in urine or stoolProlonged bleeding from cutsFatigueFeeling unusually tired after minor activityRarely, bleeding inside the skull (a serious emergency)
Multifactorial
Caused by a mix of several genes and environmental factors
Variable
Can begin at different ages, from infancy through adulthood
FDA & Trial Timeline
10 eventsNovartis Pharmaceuticals — PHASE2
Qilu Pharmaceutical Co., Ltd. — PHASE3
Peking Union Medical College Hospital — PHASE2
Institute of Hematology & Blood Diseases Hospital, China — NA
Institute of Hematology & Blood Diseases Hospital, China — PHASE2
Institute of Hematology & Blood Diseases Hospital, China — PHASE2
Xiao Hui Zhang
Union Hospital, Tongji Medical College, Huazhong University of Science and Technology — EARLY_PHASE1
Union Hospital, Tongji Medical College, Huazhong University of Science and Technology — EARLY_PHASE1
Institute of Hematology & Blood Diseases Hospital, China — PHASE2
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
1 availableDoptelet
Treatment of thrombocytopenia in pediatric patients 1 year and older with persistent or chronic immune thrombocytopenia who have had an insufficient response to a previous treatment
Rare Disease Specialist
Rare Disease Specialist
Treatment Centers
8 centersBaylor College of Medicine Rare Disease Center ↗
Baylor College of Medicine
📍 Houston, TX
🏥 NORDStanford Medicine Rare Disease Center ↗
Stanford Medicine
📍 Stanford, CA
🔬 UDNNIH Clinical Center Undiagnosed Diseases Program ↗
National Institutes of Health
📍 Bethesda, MD
🔬 UDNUCLA UDN Clinical Site ↗
UCLA Health
📍 Los Angeles, CA
🔬 UDNBaylor College of Medicine UDN Clinical Site ↗
Baylor College of Medicine
📍 Houston, TX
🔬 UDNHarvard/MGH UDN Clinical Site ↗
Massachusetts General Hospital
📍 Boston, MA
🏥 NORDMayo Clinic Center for Individualized Medicine ↗
Mayo Clinic
📍 Rochester, MN
👤 Mayo Clinic Center for Individualized Medicine
🏥 NORDUCLA Rare Disease Day Program ↗
UCLA Health
📍 Los Angeles, CA
Travel Grants
No travel grants are currently matched to Autoimmune thrombocytopenia.
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Questions for your doctor
Bring these to your next appointment
- Q1.What is my current platelet count, and at what level would you recommend starting treatment?,What are the risks and benefits of the treatment options you are recommending for me?,How often will I need blood tests to monitor my platelet count?,Are there activities or medications I should avoid to reduce my bleeding risk?,What symptoms should prompt me to go to the emergency room immediately?,Is my ITP likely to be short-term or long-term, and what factors affect that?,Should I be tested for any underlying conditions that might be causing my low platelets?
Common questions about Autoimmune thrombocytopenia
What is Autoimmune thrombocytopenia?
Autoimmune thrombocytopenia, also commonly known as immune thrombocytopenia (ITP) or immune thrombocytopenic purpura, is a condition where the body's immune system mistakenly attacks and destroys its own platelets. Platelets are tiny blood cells that help your blood clot when you have a cut or injury. When platelet counts drop too low, the body has trouble stopping bleeding, even from minor bumps or cuts. People with this condition may notice easy bruising, small red or purple dots on the skin called petechiae, or bleeding from the gums and nose. Women may experience heavier-than-normal menst
How is Autoimmune thrombocytopenia inherited?
Autoimmune thrombocytopenia follows a multifactorial inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.
Are there clinical trials for Autoimmune thrombocytopenia?
Yes — 20 recruiting clinical trials are currently listed for Autoimmune thrombocytopenia on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Autoimmune thrombocytopenia?
25 specialists and care centers treating Autoimmune thrombocytopenia are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.