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Disease

Purpura, Thrombocytopenic, Idiopathic

Late-stage therapeutic developmentEmerging research
MeSHPurpura, Thrombocytopenic, Idiopathic
2
Publications
9
Clinical trials
2025
Latest publication
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this disease.

Approval: Wayrilz (EMA)

Regulatory2025-12-22EMA

The function of T cells in immune thrombocytopenia.

Research2025-02-21Frontiers in immunology

2025 update on clinical trials in immune thrombocytopenia.

Research2024-08-06American journal of hematology

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Executive briefingUpdating summary…Momentum: Low
Key developments
  • 1 clinical trial with recent milestones.
Clinical Milestones1View
Other updates 1
Activity timeline1

Therapeutic landscape

Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.

Approved & established therapies
Azathioprineapproved

Approval — Jayempi is indicated in combination with other immunosuppressive agents for the prophylax… (2021)

Clinical trials

7 sponsors · 0 new · 0 completed in the last 12 months (net +0)

The current development programme across all trial phases.

Clinical programme
9
All trials
0
Active
6
Late-stage
6
Completed
Late-stage studies

The Combination of ATRA and Eltrombopag as the Treatment Strategy for Glucocorticoid-Resistant/Relapsed ITP Based on the Stratification of the New Biomarker MSC-C5b-9: A Prospective, Randomized, Open-Label, Multicenter Clinical Trial

Phase 3 · Completed · Peking University People's Hospital

A Multicenter, Randomized, Double-Blind and Open-label Phase III Study To Compare The Efficacy And Safety Of Eltrombopag With Placebo In Chinese Chronic ITP Patients

Phase 3 · Completed · Novartis Pharmaceuticals

An Extension Study of Eltrombopag in Pediatric Patients With Chronic Immune (Idiopathic) Thrombocytopenia Purpura (ITP)

Phase 3 · Completed · Novartis Pharmaceuticals

The Clinical Randomized Controlled Study of Different Dose Rituximab in the Treatment of Primary Immune Thrombocytopenia

Phase 4 · Completed · Shandong University

High-dose Dexamethasone and Conventional Dose Prednisone, for the First-line Treatment of Adults With ITP: a Multicenter, Randomized Controlled, Clinical Trial

Phase 4 · Completed · Shandong University

A Randomized Trial of Daily Prednisone Versus Pulsed Dexamethasone in Treatment-naïve Adult Patients With Immune Thrombocytopenia

Phase 3 · Completed · University Hospital, Essen
Recently completed

The Combination of ATRA and Eltrombopag as the Treatment Strategy for Glucocorticoid-Resistant/Relapsed ITP Based on the Stratification of the New Biomarker MSC-C5b-9: A Prospective, Randomized, Open-Label, Multicenter Clinical Trial

Phase 3 · Completed · Peking University People's Hospital

An Extension Study of Eltrombopag in Pediatric Patients With Chronic Immune (Idiopathic) Thrombocytopenia Purpura (ITP)

Phase 3 · Completed · Novartis Pharmaceuticals

The Clinical Randomized Controlled Study of Different Dose Rituximab in the Treatment of Primary Immune Thrombocytopenia

Phase 4 · Completed · Shandong University

A Randomized Trial of Daily Prednisone Versus Pulsed Dexamethasone in Treatment-naïve Adult Patients With Immune Thrombocytopenia

Phase 3 · Completed · University Hospital, Essen

A Multicenter, Randomized, Double-Blind and Open-label Phase III Study To Compare The Efficacy And Safety Of Eltrombopag With Placebo In Chinese Chronic ITP Patients

Phase 3 · Completed · Novartis Pharmaceuticals

High-dose Dexamethasone and Conventional Dose Prednisone, for the First-line Treatment of Adults With ITP: a Multicenter, Randomized Controlled, Clinical Trial

Phase 4 · Completed · Shandong University

Regulatory timeline

Drug regulatory events matched to this condition by indication — EMA.

First approvals
2021emaApproval — Azathioprine· Jayempi is indicated in combination with other immunosuppressive agents for the prophylaxis of transplant rejection in patients receiving allogenic kidney, liver, heart, lung or pancreas transplants. Azathioprine is indicated in immunosuppressive regimens as an adjunct to immunosuppressive agents that form the mainstay of treatment (basis immunosuppression). Jayempi is used as an immunosuppressant antimetabolite either alone or, more commonly, in combination with other agents (usually corticosteroids) and/ or procedures which influence the immune response. Jayempi is indicated in patients who are intolerant to glucocorticosteroids or if the therapeutic response is inadequate despite treatment with high doses of glucocorticosteroids, in the following diseases: severe active rheumatoid arthritis (chronic polyarthritis) that cannot be kept under control by less toxic agents (disease-modifying anti-rheumatic -medicinal products – DMARDs) auto-immune hepatitis  systemic lupus erythematosus dermatomyositis polyarteritis nodosa pemphigus vulgaris and bullous pemphigoid Behçet’s disease refractory auto-immune haemolytic anaemia, caused by warm IgG antibodies chronic refractory idiopathic thrombocytopenic purpura Jayempi is used for the treatment of moderately severe to severe forms of chronic inflammatory bowel disease (IBD) (Crohn’s disease or ulcerative colitis) in patients in whom glucocorticosteroid therapy is necessary, but where glucocorticosteroids are not tolerated, or in whom the disease is untreatable with other common means of first choice. It is also indicated in adult patients in relapsing multiple sclerosis, if an immunomodulatory therapy is indicated but beta interferon therapy is not possible, or a stable course has been achieved with previous treatment with azathioprine. 3 Jayempi is indicated for the treatment of generalised myasthenia gravis. Depending on the severity of the disease, Jayempi should be given in combination with glucocorticosteroids because of slow onset of action at the beginning of treatment and the glucocorticosteroid dose should be gradually reduced after several months of treatment. source ↗

European Medicines Agency (CC BY 4.0). Events are matched to this condition by drug indication text — approvals/updates for drugs indicated for it, not disease-specific acts.

Research activity

2 papers

Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.

Publications over time
20242025
Most influential

2025 update on clinical trials in immune thrombocytopenia.

American journal of hematology · 2024 · 12 cites

The function of T cells in immune thrombocytopenia.

Frontiers in immunology · 2025 · 9 cites
Recent publications

The function of T cells in immune thrombocytopenia.

Frontiers in immunology · 2025 · 9 cites

2025 update on clinical trials in immune thrombocytopenia.

American journal of hematology · 2024 · 12 cites
Major themes3
  • Purpura, Thrombocytopenic, Idiopathic2
  • T-Lymphocyte Subsets1
  • T-Lymphocytes1
Leading journals2
  • American journal of hematology1
  • Frontiers in immunology1
Leading researchers8
  • Al-Samkari H1
  • Benlagha K1
  • Bu S1
  • Buttgereit F1
  • Byazrova M1
  • Filatov A1
  • Gaber T1
  • Gong Q1
Affiliations (unnormalised)6
  • Asan Medical Center1
  • Department of Rheumatology and Clinical Immunology1
  • German Rheumatism Research Centre (DRFZ) Berlin1
  • Institut de Recherche Saint-Louis1
  • Laboratory of Immunochemistry1
  • Massachusetts General Hospital1

Reference

Authoritative identity, definition & identifiers.

Defined in MeSH

Thrombocytopenia occurring in the absence of toxic exposure or a disease associated with decreased platelets. It is mediated by immune mechanisms, in most cases IMMUNOGLOBULIN G autoantibodies which attach to platelets and subsequently undergo destruction by macrophages. The disease is seen in acute (affecting children) and chronic (adult) forms.

Identifiers
MeSHPurpura, Thrombocytopenic, Idiopathic
References & data sources
  • Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
  • Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
  • Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
  • Related entities are derived from literature co-mention (studied together) — associative, not causal.