Trial reportBlood2015
Hemostatic efficacy, safety, and pharmacokinetics of a recombinant von Willebrand factor in severe von Willebrand disease.
Trial report in Blood, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. It is linked to trial NCT01410227 (A Phase 3 Clinical Study to Determine the Pharmacokinetics, Safety and Efficacy of Recombinant Von Willebrand Factor), which is not on this map. Cited by 64 papers, 2 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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.
A Phase 3 Clinical Study to Determine the Pharmacokinetics, Safety and Efficacy of Recombinant Von Willebrand Factor : Recombinant Factor VIII (rVWF:rFVIII) and rVWF in the Treatment of Bleeding Episodes in Subjects Diagnosed With Von Willebrand Disease
Who cites it
64 citing papers in PubMed, 2 syntheses or guidelines pooled it, 154 citations in OpenAlex.
- Past, Present, and Future of von Willebrand Disease.Advances in therapy · 2026Pooled it
- Correlation Analysis of DNA Methylation in the von Willebrand Factor Promoter Region and the Risk of Unexplained Recurrent Hemophilia: Systematic Review and Meta-Analysis.Contrast media & molecular imaging · 2022Pooled it
- Efficacy and safety of recombinant von Willebrand factor in on-demand treatment of children with von Willebrand disease: up to 4 years of phase 3/3b follow-up.Research and practice in thrombosis and haemostasis · 2026Trial
- Recombinant von Willebrand factor and tranexamic acid for heavy menstrual bleeding in patients with mild and moderate von Willebrand disease in the USA (VWDMin): a phase 3, open-label, randomised, crossover trial.The Lancet. Haematology · 2023Trial
- Trial
- Phase 3 study of recombinant von Willebrand factor in patients with severe von Willebrand disease who are undergoing elective surgery.Journal of thrombosis and haemostasis : JTH · 2019Trial
- Recombinant von Willebrand Factor (vonicog alfa) reduces platelet inhibition caused by antiplatelet drugs and has potential as an acute haemostatic agent.Haematologica · 2026Article
- Population PK of VWF: toward personalized surgery.Blood advances · 2026Article
- Prophylaxis for von Willebrand disease: Is it time for parity with established practice in hemophilia A?Therapeutic advances in hematology · 2026Review
- Recombinant von Willebrand factor for von Willebrand disease: mechanism of action and clinical application.Therapeutic advances in hematology · 2026Review
- Outcomes and Healthcare Resource Utilisation in Adults With von Willebrand Disease Treated With Recombinant von Willebrand Factor in Surgical Settings in the United Kingdom.European journal of haematology · 2026Article
- Outcomes and Healthcare Resource Utilisation in Adults With von Willebrand Disease Receiving On-Demand Recombinant von Willebrand Factor in the United Kingdom.European journal of haematology · 2026Article
- Historical, current and future treatments for von Willebrand disease.Haematologica · 2026Review
- Article
- Rewriting the script: gene therapy and genome editing for von Willebrand Disease.Frontiers in genome editing · 2025Review
- Safety of Recombinant von Willebrand Factor in the Treatment of von Willebrand Disease: Real-World Data from an EU Post-Authorization Safety Study.Journal of blood medicine · 2025Article
- Prophylaxis in von Willebrand disease with von Willebrand factor concentrate and nonfactor therapies.Research and practice in thrombosis and haemostasis · 2024Article
- von Willebrand disease.Nature reviews. Disease primers · 2024Review
- Article
- Exposure-Response Relationship between VWF/FVIII Activity and Spontaneous Bleeding Events Following Recombinant VWF Prophylaxis in Severe VWD.TH open : companion journal to thrombosis and haemostasis · 2024Article
4 more citing papers are in PubMed but not listed here.
Corrections and comments
- Commented on by
- Erratum issued
- Erratum issued
Authors and funding
12 authors at 7 institutions in 4 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
This phase 3 trial evaluated the safety and hemostatic efficacy of a recombinant von Willebrand factor (rVWF) for treatment of bleeds in severe von Willebrand disease (VWD). rVWF was initially administered together with recombinant factor VIII (rFVIII) and subsequently alone, as long as hemostatic factor VIII activity (FVIII : C) levels were maintained. Pharmacokinetics (PK) were evaluated in a randomized cross-over design (rVWF vs rVWF:rFVIII at 50 IU VWF:ristocetin cofactor activity [RCo]/kg). Bleed control for all treated bleeds (N = 192 bleeds in 22 subjects) was rated good or excellent (96.9% excellent; 119 of 122 minor, 59 of 61 moderate, and 6 of 7 major bleeds) on a 4-point scale (4 = none to 1 = excellent). A single infusion was effective in 81.8% of bleeds. Treatment success, defined as the number of subjects with a mean efficacy rating of <2.5, was 100%. The PK profile of rVWF was not influenced by rFVIII (mean VWF:RCo terminal half-life: 21.9 hours for rVWF and 19.6 hours for rVWF:rFVIII). FVIII : C levels increased rapidly after rVWF alone, with hemostatic levels achieved within 6 hours and sustained through 72 hours after infusion. Eight adverse events (AEs; 6 nonserious AEs in 4 subjects and 2 serious AEs [chest discomfort and increased heart rate, without cardiac symptomatology] concurrently in 1 subject) were associated with rVWF. There were no thrombotic events or severe allergic reactions. No VWF or FVIII inhibitors, anti-VWF binding antibodies, or antibodies against host cell proteins were detected. These results show that rVWF was safe and effective in treating bleeds in VWD patients and stabilizes endogenous FVIII : C, which may eliminate the need for rFVIII after the first infusion. This trial was registered at www.clinicaltrials.gov as #NCT01410227.
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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.