Thrombotic Microangiopathies
Recent clinical, regulatory, research and industry developments relating to this disease.
COVID-19: Coagulopathy, Risk of Thrombosis, and the Rationale for Anticoagulation.
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
- 4 clinical trials with recent milestones.
Clinical MilestonesViewHide
- 2027-05-31Defibrotide Prophylaxis of Transplant Associated-Thrombotic Microangiopathy in Patients With High-Risk Neuroblastoma Receiving Tandem TransplantsWithdrawn
- 2026-11-01A Multicenter, Phase 1b Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of HMPL-523, a Syk Inhibitor, in Adult Subjects With Immune ThrombocytopeniaWithdrawn
- 2025-09-01Multicenter, Uncontrolled Pilot Study Evaluating the Efficacy of Eculizumab in the Treatment of Gemcitabine-induced Thrombotic MicroangiopathiesWithdrawn
- 2027-05-31ClinicalDefibrotide Prophylaxis of Transplant Associated-Thrombotic Microangiopathy in Patients With High-Risk Neuroblastoma Receiving Tandem TransplantsWithdrawn
- 2026-11-01ClinicalA Multicenter, Phase 1b Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of HMPL-523, a Syk Inhibitor, in Adult Subjects With Immune ThrombocytopeniaWithdrawn
- 2025-12-01ClinicalA Phase 2 Study Evaluating the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Narsoplimab in Pediatric Patients (28 Days to ≤ 18 Years of Age.) With High-Risk Hematopoietic Stem Cell Transplant Thrombotic MicroangiopathyPrimary completion
- 2025-09-01ClinicalMulticenter, Uncontrolled Pilot Study Evaluating the Efficacy of Eculizumab in the Treatment of Gemcitabine-induced Thrombotic MicroangiopathiesWithdrawn
Clinical trials
The current development programme across all trial phases.
Research activity
Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.
Major themes2
- Betacoronavirus1
- Pandemics1
Leading journals2
- Clinical and applied thrombosis/hemostasis : official journal of the International Academy of Clinical and Applied Thrombosis/Hemostasis1
- The Journal of pediatrics1
Leading researchers8
- Arya K1
- Chand DH1
- Dandamudi R1
- Dharnidharka VR1
- Farrar MA1
- Goedeker NL1
- Mackie FE1
- Makris M1
Affiliations (unnormalised)6
- Department of Haemostaseology and Hemophilia Center1
- IL; Washington University School of Medicine and St Louis Children's Hospital1
- Sheffield Haemophilia and Thrombosis Centre1
- Sydney Children's Hospital Randwick and School of Women's and Children's Health1
- University of Arkansas for Medical Sciences1
- University of Sheffield1
Reference
Authoritative identity, definition & identifiers.
Diseases that result in THROMBOSIS in MICROVASCULATURE. The two most prominent diseases are PURPURA, THROMBOTIC THROMBOCYTOPENIC; and HEMOLYTIC-UREMIC SYNDROME. Multiple etiological factors include VASCULAR ENDOTHELIAL CELL damage due to SHIGA TOXIN; FACTOR H deficiency; and aberrant VON WILLEBRAND FACTOR formation.
- 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.