ORPHA:3002
Immune thrombocytopenia
Also known as: ITP · Immune thrombocytopenic purpura
Publications
30,973
99.1th percentile
Trials
386
Interventional, condition-specific
Researchers
1,138
Distinct authors in sample
Gene link
—
Readiness
2/6
Stages with a signal
Clinical definition (Orphanet)
A rare autoimmune coagulation disorder characterized by isolated thrombocytopenia (a platelet count <100,000/microL), in the absence of any underlying disorder that may be associated with thrombocytopenia.
How rare: 1-5 / 10 000 — about one to five people per ten thousand (still uncommon, but less ultra-rare).
Cross-references
Joined from Mondo / Orphanet. MeSH labels may enter searches; UMLS / OMIM / NCIT are stored for reference.
- MONDO:0008558
- OMIM:188030
- UMLS:C0398650
- NCIT:C3446
Additional Mondo synonyms (5)
autoimmune thrombocytopenic purpura · idiopathic thrombocytopenia · idiopathic thrombocytopenia purpura · idiopathic thrombocytopenic purpura · thrombocytopenic purpura, autoimmune
Research stages
Trial readiness signals
Where this condition sits on an open-data research pipeline — not how close a treatment is, and not medical advice. Empty stages often mean “not in these databases under this Mondo ID,” not “impossible.”
2/6 stages with a signal
An interventional trial matched this condition name on ClinicalTrials.gov — see trials below.
- Gene identifiedNot found
No GenCC disease–gene assertion in this build
- LiteraturePresent
30,973 matched papers (17,257 in last 10 years) Source
- Phenotype characterisedNot checked
Not yet enriched from Monarch / HPO
- Animal modelNot checked
Not yet enriched from Monarch / Alliance
- Orphan designationNot checked
FDA/EMA orphan-drug designation not enriched yet
- Interventional trialPresent
386 matched on ClinicalTrials.gov (110 recruiting in sample)
Biology
Genes and phenotypes
Gene–disease validity from GenCC, plus phenotypes and animal models joined from Monarch Initiative via Mondo ID — not a clinical diagnosis aid.
Do we know what causes it?
Not yet — the cause hasn't been pinned down in GenCC.
No strong gene–disease assertion joined for this Orphanet entity.
Phenotypes (Monarch / HPO)
Not enriched in this build — Monarch phenotype joins were not run for this record.
Animal models (Monarch / Alliance)
Not enriched in this build.
Literature
Is anyone studying this?
30,973
30,973 papers — among the better-studied rare conditions, though still a fraction of common-disease literature (breast cancer: over 700,000). Median papers in the last 10 years for a rare disease in this dataset (publications denominator n=5449) is 41.
30,973 papers since the earliest indexed year in this search — median last-10-year count for a rare disease in this dataset is 41 (publications denominator n=5449).
17,257 in the last 10 years · medium confidence · 99.1th percentile (publications denominator)
Phrase hits: 30,973 · MeSH hits: 0
Who's working on it?
1,138
Distinct author names in 200 sampled papers — named people below.
Who's working on it?
People publishing on this condition (sampled Europe PMC records). Affiliation is the most recent found in that sample.
- 01Li Y4 papers · 2026
Hebei Key Laboratory of Hematology, Department of Hematology, The Second Hospital of Hebei Medical University, Shijiazhuang, China.
Papers in Europe PMC - 02Saki N3 papers · 2026
Thalassemia & Hemoglobinopathy Research Center, Health Research Institute, Ahvaz Jundishapur University of Medical Sciences, Ahvaz, Iran.
Papers in Europe PMC - 03Wang Z3 papers · 2026
Songjiang District Sijing Hospital, Shanghai, 201600, China.
Papers in Europe PMC - 04Zhang X3 papers · 2026
School of Basic Medical Sciences, Southwest Medical University, Luzhou 646000, China; (J.D.); (Y.Z.)
Papers in Europe PMC - 05AbuBaha M2 papers · 2025
Department of Medicine An-Najah National University Nablus Palestine.
Papers in Europe PMC - 06Aljunaidi R2 papers · 2025
Faculty of Medicine and Health Sciences Palestine Polytechnic University Hebron Palestine.
Papers in Europe PMC - 07Almasalma M2 papers · 2026
Faculty of Medicine, Mansoura University, Mansoura, Egypt.
Papers in Europe PMC - 08Alqadi M2 papers · 2025
Department of Medicine University of Toledo Toledo Ohio USA.
Papers in Europe PMC - 09Amin MK2 papers · 2025
Division of Hematologic Malignancies & Cellular Therapeutics, University of Kansas Medical Center, Kansas City, KS, USA; Mikael Rayaan Foundation Global Transplantation and Cellular Therapy Consortium, Kansas City, KS, USA.
Papers in Europe PMC - 10Baniowda M2 papers · 2025
Department of Medicine University of Missouri-Kansas City Kansas City Missouri USA.
Papers in Europe PMC
Clinical research
Is a treatment being tested?
386
interventional trials for this specific condition
386 interventional trials matched this specific condition name; 110 currently recruiting in our sample. 319 trials are registered for thrombocytopenia, the broader category — shown separately because they may or may not enrol this specific subtype.
Data as of 27 July 2026
386 interventional trials — more than 73% of diseases in the trials denominator have none at all (5114 of 7003; this disease is at the 99.6th percentile).
medium confidence · 99.6th percentile (trials denominator)
Recruiting interventional trials
From the matched ClinicalTrials.gov set
386 interventional trials matched after quoted-phrase search and title/condition post-filter.
- NCT07687134·NOT YET RECRUITING·Eltrombopag Plus Telitacicept vs. Eltrombopag Alone for Steroid-refractory/Relapsed ITP
Conditions: Immune Thrombocytopenia·Matched via name phrase
- NCT07599280·NOT YET RECRUITING·Universal STAR-T Cell Injection in R/R Autoimmune Diseases.
Conditions: Immune Thrombocytopenia·Matched via name phrase
- NCT07362238·RECRUITING·Daratumumab Versus Rituximab in the Management of Pediatric Primary Immune Thrombocytopenia (ITP)
Conditions: Immune Thrombocytopenia · Treatment·Matched via name phrase
- NCT07700238·NOT YET RECRUITING·A Study of Romiplostim Plus Predniso(lo)ne vs. Predniso(lo)ne Alone for the Treatment of Previously Untreated Primary Immune Thrombocytopenia (ITP)
Conditions: Primary Immune Thrombocytopenia·Matched via name phrase
- NCT07603557·NOT YET RECRUITING·Study of Zola-cel (BMS-986353), in Participants With Autoimmune Cytopenia (Breakfree-AiCE)
Conditions: Chronic Immune Thrombocytopenia · Autoimmune Hemolytic Anemia·Matched via name phrase
- NCT07549698·RECRUITING·Safety and Preliminary Efficacy of CTX112 in Adult Participants With Relapsed/Refractory Hematologic Autoimmune Disease
Conditions: Warm Autoimmune Hemolytic Anemia (WAIHA) · ITP - Immune Thrombocytopenia · Warm Autoimmune Hemolytic Anemia · Immune Thrombocytopenic Purpura·Matched via name phrase
- NCT07278908·NOT YET RECRUITING·Avatrombopag Combined With All-trans Retinoic Acid in the Treatment of Primary Immune Thrombocytopenia
Conditions: ITP - Immune Thrombocytopenia·Matched via name phrase
- NCT06658834·RECRUITING·A Study of Romiplostim N01 as the First-line Treatment for Newly Diagnosed Adult Patients With ITP
Conditions: Primary Immune Thrombocytopenia·Matched via name phrase
- NCT07068126·RECRUITING·Management of Children With Persistent ITP, A Novel Approach
Conditions: Persistent Immune Thrombocytopenia·Matched via name phrase
- NCT06478537·RECRUITING·TPO-RA Treatment on Immune Tolerance Induction of ITP Patients With Sustained Platelet Recovery After Treatment Termination
Conditions: Immune Thrombocytopenia·Matched via name phrase
- NCT07007962·RECRUITING·Study to Evaluate the Efficacy and Safety of Oral Rilzabrutinib in Adults With Immune Thrombocytopenia (ITP) Who Failed First-line Treatment
Conditions: Immune Thrombocytopenia·Matched via name phrase
- NCT07095127·RECRUITING·Safety, Tolerability, and Efficacy of NVG-2089 in Participants With Immune Thrombocytopenia
Conditions: Immune Thrombocytopenia (ITP)·Matched via name phrase
- NCT06531018·RECRUITING·Role of Eltrombopag as First Line Therapy in Primary Immune Thrombocytopenia.
Conditions: Primary Immune Thrombocytopenia·Matched via name phrase
- NCT07064889·NOT YET RECRUITING·The Combination of Upadacitinib and High-dose Dexamethasone as First-line Treatment in Adult Immune Thrombocytopenia
Conditions: Immune Thrombocytopenia·Matched via name phrase
- NCT07189910·NOT YET RECRUITING·Sequential rhTPO and Eltrombopag Following Glucocorticoids for Severe Adult ITP
Conditions: Primary Immune Thrombocytopenia (ITP)·Matched via name phrase
Broader category: thrombocytopenia
319
Interventional trials for the parent category, exclusive of NCT IDs already counted above. Eligibility for this subtype is not guaranteed.
Worth raising with a clinician. How we count trials.
Recruiting under the broader category
- NCT06412497·RECRUITING·MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure.
Conditions: Severe Aplastic Anemia · Acquired Amegakaryocytic Thrombocytopenia · Acquired Pure Red Cell Aplasia · Paroxysmal Nocturnal Hemoglobinuria·Matched via name phrase
- NCT06673498·RECRUITING·Lusutrombopag Combined With Recombinant Human Thrombopoietin for the Treatment of Thrombocytopenia in Patients With Chronic Liver Disease Destined to Undergo Elective Invasive Surgery
Conditions: Thrombocytopenia·Matched via name phrase
- NCT06533098·RECRUITING·A Study of Nipocalimab or Intravenous Immunoglobulin (IVIG) in Pregnancies At Risk of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT)
Conditions: Thrombocytopenia, Neonatal Alloimmune·Matched via name phrase
- NCT07586813·RECRUITING·Romiplostim N01 Plus ATRA for Persistent Isolated Chemotherapy-Induced Thrombocytopenia After Complete Remission of Gastrointestinal Solid Tumors
Conditions: Persistent Isolated Chemotherapy-Induced Thrombocytopenia · Chemotherapy-Induced Thrombocytopenia · Gastrointestinal Neoplasms · Pancreatic Cancer·Matched via name phrase
- NCT06202625·RECRUITING·Efficacy and Safety of Avatrombopag in the Treatment of Thrombocytopenia After Haplo-HSCT
Conditions: Thrombocytopenia · Stem Cell Transplant Complications·Matched via name phrase
- NCT06886516·RECRUITING·Apixaban in Thrombocytopenia
Conditions: Deep Vein Thrombosis · Pulmonary Embolism and Thrombosis · Thrombocytopenia · Recurrent Venous Thromboembolism·Matched via name phrase
- NCT06788691·RECRUITING·Luspatercept for Clonal Cytopenias of Uncertain Significance
Conditions: CCUS Clonal Cytopenia of Undetermined Significance · Anemia · Leukopenia · Thrombocytopenia·Matched via name phrase
- NCT06599385·NOT YET RECRUITING·Platelet Transfusion in Critically Ill Patients With Thrombocytopenia
Conditions: Thrombocythemia·Matched via name phrase
- NCT06759636·RECRUITING·A Study on the Treatment of CTIT of the Efficacy and Safety With Romiplostim N01 Compared to Recombinant Human Interleukin-11
Conditions: CTIT: Cancer Therapy Induced Thrombocytopenia·Matched via name phrase
- NCT05983952·RECRUITING·Anti-CD38 Antibody Treating APS With Thrombocytopenia
Conditions: Antiphospholipid Syndrome · Thrombocytopenia·Matched via name phrase
- NCT07315087·RECRUITING·CAR T-cell Therapy Targeting CD19 and BCMA(QT-019C) in Patients With Relapse/Refractory Autoimmune Diseases
Conditions: SLE - Systemic Lupus Erythematosus · SSc-Systemic Sclerosis · IIM- Idiopathic Inflammatory Myopathies · ANCA Associated Vasculitis (AAV)·Matched via name phrase
- NCT07575854·NOT YET RECRUITING·A Study of Selective Internal Radiation Therapy for the Management of Chemotherapy-induced Thrombocytopenia With Splenomegaly in Adult Subjects With Gastrointestinal Cancer
Conditions: Gastrointestinal Cancer·Matched via name phrase
- NCT07048249·RECRUITING·Single Arm Romiplostim to Prevent CIT
Conditions: Ewings Sarcoma · Chemotherapy Induced Thrombocytopenia·Matched via name phrase
- NCT06534021·RECRUITING·IASO-782 in Autoimmune Hematological Diseases
Conditions: Autoimmune Thrombocytopenia · Warm Autoimmune Haemolytic Anaemia·Matched via name phrase
- NCT07441720·RECRUITING·Safety and Efficacy of Umbilical Cord Blood Therapy for Cancer Therapy-Induced Thrombocytopenia (CTIT)
Conditions: Thrombocytopenia · Cancer Treatment-induced Thrombocytopenia·Matched via name phrase
Observational and natural-history studies
105 observational studies match this condition. These do not test a treatment and are not counted in the interventional-trial headline, but they are genuine research: natural-history work often defines the endpoints needed for a future rare-disease trial, and families may be able to enroll.
Recruiting or not-yet-recruiting
- NCT07059910·NOT YET RECRUITING·Prevalence of Thyroid Disorders in Patients With Immune Thrombocytopenic Purpura and Its Impact on Treatment
Conditions: Immune Thrombocytopenic Purpura · Thyroid Disorders·Matched via name phrase
- NCT07046013·NOT YET RECRUITING·Level of Serum Lactate Dehydrogenase in Immune Thrombocytopenia
Conditions: Immune Thrombocytopenia (ITP)·Matched via name phrase
- NCT05937828·RECRUITING·OBS'CEREVANCE: French Cohort of Pediatric Autoimmune Cytopenia
Conditions: Immune Thrombocytopenia · Autoimmune Hemolytic Anemia · Evans Syndrome·Matched via name phrase
- NCT06137105·NOT YET RECRUITING·Thrombopoietin Agonists in Patients With Idiopathic Thrombocytopenic Purpura
Conditions: ITP - Immune Thrombocytopenia·Matched via name phrase
- NCT06107582·RECRUITING·Longitudinal Cohort of Pediatric Primary Immune Thrombocytopenia (ITP)
Conditions: Primary Immune Thrombocytopenia·Matched via name phrase
- NCT07014904·NOT YET RECRUITING·"Cytotoxic T Lymphocyte-Associated Antigen-4 (CTLA-4) Gene Single-nucleotide Polymorphism in Primary Immune Thrombocytopenic Purpura in Children"
Conditions: ITP - Immune Thrombocytopenia·Matched via name phrase
- NCT07587723·RECRUITING·A Real-world Study of Treatment Patterns and Outcomes in Primary Immune Thrombocytopenia Patients Treated With Eltrombopag
Conditions: Immune Thrombocytopenia·Matched via name phrase
- NCT07182760·NOT YET RECRUITING·Ultrasound to Detect Splenomegally in Patients With Isolated Thrombocytopenia
Conditions: Primary Immune Thrombocytopenic Purpura · Amegakaryocytic Aplasia · Unilineage Myelodysplastic Syndrome ( Megakaryocyte Dysplasia) · Lymphoproliferative Disorder With Secondary ITP·Matched via name phrase
- NCT06556888·RECRUITING·Observatory of Immunological Thrombopenic Purpura in the Brest Region
Conditions: Immune Thrombocytopenia·Matched via name phrase
- NCT07426107·NOT YET RECRUITING·Development and Prospective Validation of an AI Model for Prognosis in ITP Patients Undergoing Coronary Revascularization
Conditions: Immune Thrombocytopenic Purpura · Coronary Artery Disease·Matched via name phrase
- NCT05931718·RECRUITING·Prospective Evaluation of Diagnosis and Treatment of Patients With Autoimmune Cytopenias Including Autoimmune Hemolytic Anemia, Immune Thrombocytopenia, and Chronic Idiopathic/Autoimmune Neutropenia
Conditions: Autoimmune Hemolytic Anemia · Immune Thrombocytopenia · Chronic Idiopathic Neutropenia · Autoimmune Neutropenia·Matched via name phrase
- NCT06757257·RECRUITING·Special Drug Use-results Survey for Long-term Use (Fostamatinib)
Conditions: Chronic Idiopathic Thrombocytopenic Purpura·Matched via name phrase
- NCT07268898·NOT YET RECRUITING·National Multicenter Retrospective Real-World Study on the Treatment Status of Newly Diagnosed and Persistent Primary Immune Thrombocytopenia (ITP) Patients
Conditions: Immune Thrombocytopenia (ITP)·Matched via name phrase
- NCT07298733·NOT YET RECRUITING·The Value of Interleukin-1β and Interleukin-33 Genetic Expression in the Pathogenesis and Differentiation of Primary ITP and SLE-Related Thrombocytopenia
Conditions: Immune Thrombocytopenia · System; Lupus Erythematosus·Matched via name phrase
- NCT06601543·NOT YET RECRUITING·Steroid Resistance in Pediatric Immune Thrombocytopenic Purpura
Conditions: Pediatric Mmune Thrombocytopenic Purpura·Matched via name phrase
Where to find support
We do not yet link condition-specific patient organisations. These umbrella groups support undiagnosed and ultra-rare families:
India — NPRD
Last verified 2026-07-26This ORPHAcode is not on our curated NPRD list (direct or Mondo-parent match). That does not decide clinical eligibility; families in India should ask a notified Centre of Excellence about current coverage.
Hand-curated for this project. ORPHAcode mappings are best-effort and may be incomplete or imprecise for umbrella categories. Parent (Mondo) matches mean the policy lists a broader category — confirm eligibility with a Centre of Excellence. Financial entitlements summarised from public policy statements and may change. This is not official government guidance.
How we counted this
Europe PMC query (preferred label + any corrected label + Orphanet and Mondo exact synonyms, stoplisted; unioned with resolved MeSH labels when available). UMLS / OMIM / NCIT cross-references are stored on the overview but are not added to the query string.
"Immune thrombocytopenia" OR "Immune thrombocytopenic purpura" OR "autoimmune thrombocytopenic purpura" OR "idiopathic thrombocytopenia" OR "idiopathic thrombocytopenia purpura" OR "idiopathic thrombocytopenic purpura" OR "thrombocytopenic purpura, autoimmune"
ClinicalTrials.gov query (quoted phrases + MeSH via query.cond, plus recall-expansion terms when used):
"Immune thrombocytopenia" OR "Immune thrombocytopenic purpura" OR "autoimmune thrombocytopenic purpura" OR "idiopathic thrombocytopenia" OR "idiopathic thrombocytopenia purpura" OR "idiopathic thrombocytopenic purpura" OR "thrombocytopenic purpura, autoimmune"
Interventional trials matched via: phrase (mesh = registered under a MeSH descriptor no name phrase would catch; recall-expansion = gene / selected parent terms used only for trials).
Study-type breakdown: 386 interventional · 105 observational · 2 expanded access. Only interventional studies enter the trial headline.
Parent-category trials query:
"thrombocytopenia"
Query health: ok — strategies attempted: phrase; with hits: phrase
Run this search on ClinicalTrials.gov
Synonyms dropped by stoplist: ITP
Confidence reasoning
- Preferred label is multi-word and distinctive
- 1 synonym(s) dropped by stoplist (may under-count)
- No label/synonym collisions with other diseases in this corpus
Ingested 2026-07-26T21:58:04.493Z
