Autoimmune thrombocytopenia

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1FDA treatments82Active trials127Specialists8Treatment centers

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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)

Inheritance

Multifactorial

Caused by a mix of several genes and environmental factors

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗NORD ↗

FDA & Trial Timeline

10 events
Jun 2026A Study to Assess the Tolerability of Ianalumab (VAY736) With Investigator's Choice Thrombopoietin Receptor Agonist (IC TPO-RA) in Patients With Primary Immune Thrombocytopenia (ITP)

Novartis Pharmaceuticals — PHASE2

TrialNOT YET RECRUITING
Apr 2026Safety and Efficacy Study of QL0911 to Treat Immune Thrombocytopenia (ITP) in Pediatric Patients

Qilu Pharmaceutical Co., Ltd. — PHASE3

TrialNOT YET RECRUITING
Apr 2026Phase II Study of Orelabrutinib in Combination With Romiplostim N01 in Patients With Primary Immune Thrombocytopenia (ITP) Who Have Received At Least One Prior Line of Therapy

Peking Union Medical College Hospital — PHASE2

TrialNOT YET RECRUITING
Mar 2026rhTPO Dose Escalation vs Eltrombopag Switch in ITP

Institute of Hematology & Blood Diseases Hospital, China — NA

TrialNOT YET RECRUITING
Mar 2026Daratumumab Versus Rituximab in the Management of Pediatric Primary Immune Thrombocytopenia (ITP)

Institute of Hematology & Blood Diseases Hospital, China — PHASE2

TrialRECRUITING
Mar 2026Anti-CD38 Monoclonal Antibody Combined With Rituximab in the Management of Primary Immune Thrombocytopenia (ITP)

Institute of Hematology & Blood Diseases Hospital, China — PHASE2

TrialRECRUITING
Feb 2026Development and Prospective Validation of an AI Model for Prognosis in ITP Patients Undergoing Coronary Revascularization

Xiao Hui Zhang

TrialNOT YET RECRUITING
Feb 2026A Clinical Study Evaluating the Safety and Preliminary Efficacy of Universal Allogeneic CAR T-cell Therapy Targeting CD19 and BCMA(QT-019C)in Patients With Refractory Primary Immune Thrombocytopenia

Union Hospital, Tongji Medical College, Huazhong University of Science and Technology — EARLY_PHASE1

TrialNOT YET RECRUITING
Feb 2026UCAR-T Targeting CD19/BCMA in Subjects With Autoantibody-Mediated Autoimmune Benign Hematological Diseases

Union Hospital, Tongji Medical College, Huazhong University of Science and Technology — EARLY_PHASE1

TrialRECRUITING
Jan 2026Rituximab Combining Anti-CD38 Monoclonal Antibody Versus Rituximab in the Management of Primary Immune Thrombocytopenia (ITP)

Institute of Hematology & Blood Diseases Hospital, China — PHASE2

TrialRECRUITING

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

Treatments

1 available

Doptelet

avatrombopag· Sobi, Inc.

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

Clinical Trials

20 recruitingView all trials with filters →
Phase 39 trials
A Continuation Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Phase 3
Actively Recruiting
PI: Study Director (Takeda) · Sites: Los Angeles, California; Denver, Colorado +95 more · Age: 1899 yrs
A Study to Assess the Efficacy and Safety of Efgartigimod IV in Adult Participants With Primary Immune Thrombocytopenia
Phase 3
Actively Recruiting
· Sites: Phoenix, Arizona; Los Angeles, California +92 more · Age: 1899 yrs
Study to Evaluate the Efficacy and Safety of Oral Rilzabrutinib in Adults With Immune Thrombocytopenia (ITP) Who Failed First-line Treatment
Phase 3
Actively Recruiting
· Sites: Rochester, Minnesota; Albany, New York +8 more · Age: 1899 yrs
A Study of TQB3473 Tablets Compared to Placebo in the Treatment of Adult Primary Immune Thrombocytopenia
Phase 3
Actively Recruiting
· Sites: Hefei, Anhui; Wuhu, Anhui +51 more · Age: 1875 yrs
V-IMMUNE® for Immune Thrombocytopenia
Phase 3
Actively Recruiting
PI: Sandra Regina Loggetto, PhD (HCor Research Institute) · Sites: Recife, Pernambuco; São Paulo, São Paulo · Age: 199 yrs
A Study of Efficacy and Safety of Ianalumab Versus Placebo in Addition to Eltrombopag in Primary Immune Thrombocytopenia Patients Who Failed Steroids
Phase 3
Active
PI: Novartis Pharmaceuticals (Novartis Pharmaceuticals) · Sites: Yuma, Arizona; Aurora, Colorado +72 more · Age: 18100 yrs
IgIV Plus Prednisone vs High-dose Dexamethasone for ITP
Phase 3
Actively Recruiting
· Sites: Créteil · Age: 1880 yrs
Rilzabrutinib for the Adult Participants With Chronic ITP Who Have Completed Phase 3 Study in Japan
Phase 3
Active
· Sites: Tsuchiura, Ibaraki; Kanazawa, Ishikawa-ken +1 more · Age: 1899 yrs
Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP)
Phase 3
Active
· Sites: Los Angeles, California; San Francisco, California +153 more · Age: 1099 yrs
Phase 42 trials
The Efficacy and Safety of Huaiqihuang Granule in Children With Chronic Primary Immune Thrombocytopenia
Phase 4
Active
PI: Runming Jin, Professor (Union Hospital, Tongji Medical College, Huazhong U) · Sites: Guangzhou, Guangdong; Guangzhou, Guangdong +12 more · Age: 114 yrs
Two Regimens of IVIG in the Treatment of Newly Diagnosed ITP in Children
Phase 4
Actively Recruiting
· Sites: Fuzhou, Fujian · Age: 014 yrs
Other9 trials
RESTI Registry: Spanish Registry of Primary Immune Thrombocytopenia and Other Immune Thrombocytopenia
Actively Recruiting
· Sites: A Coruña, A Coruña; Palma, Balearic Islands +34 more
ITP Registry and Accompanying Biospecimen Collection
Actively Recruiting
PI: Thomas Stauch, Dr. med. (Jena University Hospital) · Sites: Aachen; Altenburg +62 more · Age: 1899 yrs
Observatory of Immunological Thrombopenic Purpura in the Brest Region
Actively Recruiting
PI: Brigitte Pan-Petesch, MD (UHB) · Sites: Brest, Brittany Region
Special Drug Use-results Survey for Long-term Use (Fostamatinib)
Actively Recruiting
· Sites: Tokyo and Japanese Other Cities
OBS'CEREVANCE: French Cohort of Pediatric Autoimmune Cytopenia
Actively Recruiting
PI: Nathalie ALADJIDI, MD (University Hospital, Bordeaux) · Sites: Amiens; Angers +7 more · Age: 017 yrs
Cognitive Dysfunction in Chronic and Persistent Immune Thrombocytopenia
Actively Recruiting
· Sites: Boston, Massachusetts · Age: 1899 yrs
Evaluation of Immune Status Before and After Splenectomy in Immune Thrombocytopenia Patients
Actively Recruiting
PI: Lei Zhang, MD (Chinese Academy of Medical Science and Blood Disea) · Sites: Tianjin, Tianjin Municipality · Age: 1860 yrs
Longitudinal Cohort of Pediatric Primary Immune Thrombocytopenia (ITP)
Actively Recruiting
PI: Lei Zhang, MD (Chinese Academy of Medical Science and Blood Disea) · Sites: Tianjin · Age: 617 yrs
Biological Bank for the Patients Followed in a Constitutive Reference Center for Autoimmune Cytopenia
Actively Recruiting
PI: Jean-François VIALLARD, Prof (University Hospital, Bordeaux) · Sites: Pessac · Age: 1899 yrs

Specialists

Showing 25 of 127View all specialists →
XZ
Xiaohui Zhang
TAMPA, FL
Specialist
PI on 4 active trials
CM
Carlo Visco, MD
Specialist
PI on 2 active trials
HM
Heng Mei
Specialist
PI on 4 active trials76 Autoimmune thrombocytopenia publications
LM
Lei Zhang, MD
Specialist
PI on 3 active trials1 Autoimmune thrombocytopenia publication
YS
Yoshitaka Shimizu
Specialist
PI on 4 active trials61 Autoimmune thrombocytopenia publications
LM
lei zhang, MD
Specialist
PI on 1 active trial1 Autoimmune thrombocytopenia publication
CB
Charlotte Bradbury
Specialist
PI on 2 active trials1 Autoimmune thrombocytopenia publication
LM
Lei Zhang, M.D.
Specialist
PI on 3 active trials
VM
Victor Blanchette, MD
Specialist
PI on 1 active trial
MM
Manuel Carcao, MD
Specialist
PI on 1 active trial
FZ
Francesco Zaja
Specialist
PI on 2 active trials2 Autoimmune thrombocytopenia publications
GM
Gregory Cheng, PhD, MD
Specialist
PI on 1 active trial1 Autoimmune thrombocytopenia publication
AM
Alexandra Schifferli, Dr. med.
Specialist
PI on 1 active trial
NM
Neal S Young, M.D.
Bethesda, Maryland
Specialist

Rare Disease Specialist

PI on 12 active trials

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 Autoimmune thrombocytopenia.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Autoimmune thrombocytopenia

1 articles
NewsRSSApr 22, 2026
Test Your Knowledge About Life Expectancy in FNAIT
This article invites people to take a quiz about life expectancy in FNAIT (Fetal and Neonatal Alloimmunization Thrombocytopenia), a rare blood disorder that aff
See all news about Autoimmune thrombocytopenia

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.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.