ReviewResearch and practice in thrombosis and haemostasis2019
Thrombotic thrombocytopenic purpura: Toward targeted therapy and precision medicine.
Review in Research and practice in thrombosis and haemostasis, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 49 papers, 1 of them a synthesis that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
49 citing papers in PubMed, 1 synthesis or guideline pooled it, 119 citations in OpenAlex.
- Effectiveness and safety of caplacizumab in acquired thrombotic thrombocytopenic purpura: health technology assessment and classification according to the methodology established in Colombia.International journal of technology assessment in health care · 2023Pooled it
- Unbiased plasma proteomics reveals hemolysis during ADAMTS13 relapse in patients with immune TTP.Blood advances · 2026Article
- Article
- A red blood cell-based antigen delivery system to facilitate T cell epitope presentation to promote peripheral tolerance to ADAMTS13 in immune-mediated thrombotic thrombocytopenic purpura.Research and practice in thrombosis and haemostasis · 2026Article
- Article
- Current standards and novel concepts in thrombotic thrombocytopenic purpura.Frontiers in medicine · 2026Review
- Monitoring ADAMTS-13 conformation in immune-mediated thrombotic thrombocytopenic purpura: toward personalized management.Research and practice in thrombosis and haemostasis · 2026Article
- Refractory Acquired Thrombotic Thrombocytopenic Purpura in the Third Trimester of Twin Pregnancy: A Case Report.International medical case reports journal · 2026Article
- Article
- Article
- Clinical Manifestations, Treatment Characteristics, and Clinical Outcomes in Patients with Immune Thrombotic Thrombocytopenic Purpura (iTTP) in a Real-World Setting: An Interim Analysis of the Turkish iTTP RegistryTurkish journal of haematology : official journal of Turkish Society of Haematology · 2025Observational
- Linking preclinical models to clinical realities: VEGF/VEGFR inhibitors and thrombotic microangiopathy in cancer therapy.iMetaOmics · 2025Article
- Cost-effectiveness of caplacizumab in immune thrombotic thrombocytopenic purpura in the United States.Journal of managed care & specialty pharmacy · 2025Article
- The Highs and Lows of ADAMTS13 Activity.Journal of clinical medicine · 2024Review
- Article
- Thrombotic Microangiopathy in Pregnancy: Current Understanding and Management Strategies.Kidney international reports · 2024Review
- Caplacizumab improves clinical outcomes and is well tolerated across clinically relevant subgroups of patients with immune-mediated thrombotic thrombocytopenic purpura.Research and practice in thrombosis and haemostasis · 2024Article
- Cost-effectiveness of rapid vs in-house vs send-out ADAMTS13 testing for immune thrombotic thrombocytopenic purpura.Blood advances · 2024Article
- Immune thrombotic thrombocytopenic purpura: pathogenesis and novel therapies: a narrative review.Annals of blood · 2023Article
- ADAMTS13 and Non-ADAMTS13 Biomarkers in Immune-Mediated Thrombotic Thrombocytopenic Purpura.Journal of clinical medicine · 2023Review
Corrections and comments
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Authors and funding
3 authors at 3 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Thrombotic thrombocytopenic purpura (TTP) is a thrombotic microangiopathy characterized by severe congenital or immune-mediated deficiency in ADAMTS13, the enzyme that cleaves von Willebrand factor multimers. This rare condition leads invariably and rapidly to a fatal outcome in the absence of treatment, and therefore raises multiple diagnostic and therapeutic challenges. The novel concepts and mechanisms identified in the laboratory for this disease have been rapidly and successfully translated into the clinic for the benefit of patients, making TTP an archetypal disease that has benefited from targeted therapies. After decades of empirical treatment with plasma exchange, identification of ADAMTS13 as the key enzyme involved in TTP pathophysiology provided an explanation for the remarkable efficacy of plasma administration, in which the missing enzyme is replenished, and paved the way for development of a recombinant form of the enzyme. Similarly, the demonstration of a major role of anti-ADAMTS13 antibodies through models of passive transfer of autoimmunity spurred development of immunomodulatory strategies based on B-cell depletion. More recently, an inhibitor of the platelet-von Willebrand factor interaction demonstrated efficacy in large clinical trials through prevention of formation of further microthrombi and protection of organs from ischemia. These translational breakthroughs in TTP are described in our review.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.