ArticleBlood2017
Recombinant ADAMTS-13: first-in-human pharmacokinetics and safety in congenital thrombotic thrombocytopenic purpura.
Article in Blood, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 108 papers.
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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.
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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.
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Who cites it
108 citing papers in PubMed, 257 citations in OpenAlex.
- Quantitative Systems Pharmacology Modeling of Platelet Responses to Recombinant ADAMTS13 in Patients With Congenital Thrombotic Thrombocytopenic Purpura.CPT: pharmacometrics & systems pharmacology · 2025Trial
- The International Hereditary Thrombotic Thrombocytopenic Purpura Registry: key findings at enrollment until 2017.Haematologica · 2019Trial
- Long Term Follow Up of Hereditary Thrombotic Thrombocytopenic Purpura on Plasma Therapy for 9 Years: A Case Report.Clinical case reports · 2026Article
- Revisiting clinical scoring tools for the diagnosis of thrombotic thrombocytopenic purpura (TTP).Journal of thrombosis and thrombolysis · 2026Review
- Von Willebrand Factor as a Therapeutic Target in Thrombotic Disorders.Thrombosis and haemostasis · 2026Review
- ADAMTS-13 R1206K variant exhibits an open conformation and an enhanced proteolytic activity toward von Willebrand factor.Journal of thrombosis and haemostasis : JTH · 2026Article
- Optimizing ADAMTS13 prophylaxis to reduce relapse and organ failure in congenital thrombotic thrombocytopenic purpura.Blood advances · 2026Article
- Recombinant ADAMTS13: An Enzyme Replacement Therapy for the Management of Congenital Thrombotic Thrombocytopenic Purpura.Journal of the advanced practitioner in oncology · 2026Article
- Precision medicine in thrombotic thrombocytopenic purpura: a narrative review.Therapeutic advances in hematology · 2026Review
- Systematic Literature Review of the Efficacy and Safety of Treatments in Congenital Thrombotic Thrombocytopenic Purpura.Journal of blood medicine · 2026Review
- An Overview of ADAMTS Proteases.Methods in molecular biology (Clifton, N.J.) · 2026Review
- Systematic Literature Review of the Economic and Quality of Life Burden of Congenital and Immune-Mediated Thrombotic Thrombocytopenic Purpura.Journal of blood medicine · 2026Review
- Modern management of congenital thrombotic thrombocytopenic purpura (cTTP).Hematology. American Society of Hematology. Education Program · 2025Article
- Recombinant ADAMTS-13 for congenital and immune thrombotic thrombocytopenic purpura in pregnancy.Blood vessels, thrombosis & hemostasis · 2025Article
- Use of PopPK and E-R Analyses toward Explaining Causal Link Between ADAMTS13 in Recombinant vs. Plasma-Based Therapies and Clinical Effects in cTTP.Clinical pharmacology and therapeutics · 2025Article
- Thrombotic thrombocytopenic purpura: early diagnosis and effective treatment in 2025.Intensive care medicine · 2025Review
- Thrombotic Thrombocytopenic Purpura in Pediatric Patients.Biomedicines · 2025Review
- ADAMTS13 Improves Hepatic Platelet Accumulation in Pyrrolizidine Alkaloids-induced Liver Injury.Journal of clinical and translational hepatology · 2025Article
- Preferences of Patients With cTTP and Other Chronic Conditions for At-Home Prophylactic Infusions: A Discrete Choice Experiment.Journal of patient experience · 2025Article
- Clinical and economic burden of suspected thrombotic thrombocytopenic purpura in US hospitals.American journal of health-system pharmacy : AJHP : official journal of the American Society of Health-System Pharmacists · 2024Article
48 more citing papers are in PubMed but not listed here.
Corrections and comments
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Authors and funding
14 authors at 10 institutions in 7 countries.
Funding
Abstract
Safety, tolerability, and pharmacokinetics of recombinant ADAMTS-13 (a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13; BAX 930; SHP655) were investigated in 15 patients diagnosed with severe congenital ADAMTS-13 deficiency (plasma ADAMTS-13 activity <6%) in a prospective phase 1, first-in-human, multicenter dose escalation study. BAX 930 was well tolerated, no serious adverse events occurred, and no anti-ADAMTS-13 antibodies were observed. After single-dose administration of BAX 930 at 5, 20, or 40 U/kg body weight to adolescents and adults, there was approximate dose proportionality with respect to maximum plasma concentration (C
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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.