Trial reportBlood2022
Recombinant von Willebrand factor prophylaxis in patients with severe von Willebrand disease: phase 3 study results.
Trial report in Blood, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02973087 (A PROSPECTIVE, PHASE 3, OPEN-LABEL, INTERNATIONAL MULTICENTER STUDY ON EFFICACY AND SAFETY OF PROPHYLAXIS WITH rVWF IN SEVERE VON WILLEBRAND DISEASE), which is not on this map. Cited by 28 papers.
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.
A PROSPECTIVE, PHASE 3, OPEN-LABEL, INTERNATIONAL MULTICENTER STUDY ON EFFICACY AND SAFETY OF PROPHYLAXIS WITH rVWF IN SEVERE VON WILLEBRAND DISEASE
Who cites it
28 citing papers in PubMed, 34 citations in OpenAlex.
- 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
- Efficacy of Wilate Prophylaxis in Reducing Nosebleeds in Patients with Severe VWD - A Post-hoc Analysis of the WIL-31 Study.Clinical and applied thrombosis/hemostasis : official journal of the International Academy of Clinical and Applied Thrombosis/HemostasisTrial
- Cost comparison of pdVWF/FVIII prophylaxis and on-demand therapy in type 3 von Willebrand disease in the United States.Blood advances · 2026Article
- Seventh Åland Island Meeting on von Willebrand Disease.Haemophilia : the official journal of the World Federation of Hemophilia · 2026Article
- von Willebrand disease: an illustrated review.Research and practice in thrombosis and haemostasis · 2026Review
- Recombinant von Willebrand Factor (vonicog alfa) reduces platelet inhibition caused by antiplatelet drugs and has potential as an acute haemostatic agent.Haematologica · 2026Article
- A Rare Combination of von Willebrand Disease Type 2A and 2M: Diagnostic and Therapeutic Challenges - A case report.Research and practice in thrombosis and haemostasis · 2026Article
- Menstrual outcomes are frequently overlooked in von Willebrand disease trials.Research and practice in thrombosis and haemostasis · 2026Article
- Treatment of von Willebrand disease.Blood advances · 2026Review
- Factor VIII and von Willebrand factor activity levels during long-term prophylaxis with wilate-Analyses from the WIL-31 study.Research and practice in thrombosis and haemostasis · 2026Article
- Prophylaxis for von Willebrand disease: Is it time for parity with established practice in hemophilia A?Therapeutic advances in hematology · 2026Review
- Historical, current and future treatments for von Willebrand disease.Haematologica · 2026Review
- Recombinant von Willebrand factor for von Willebrand disease: mechanism of action and clinical application.Therapeutic advances in hematology · 2026Review
- Individualizing dosing frequency may improve the efficacy of prophylaxis in patients with von Willebrand disease-a WIL-31 subanalysis.Research and practice in thrombosis and haemostasis · 2025Article
- 2025 ASH ISTH NBDF WFH monitoring report on the 2021 clinical guidelines on the diagnosis and management of von Willebrand disease.Blood advances · 2025Review
- Article
- Venous Thromboembolism Prophylaxis in Hemophilic Patients Undergoing Total Hip or Knee Arthroplasty: Insights from a Single-Center Experience.Medicina (Kaunas, Lithuania) · 2025Article
- The future of siRNA-mediated approaches to treat von Willebrand disease.Expert review of hematology · 2025Review
- 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
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
12 authors at 8 institutions in 4 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
International guidelines conditionally recommend long-term prophylaxis in patients with von Willebrand disease (VWD) and severe and frequent bleeding. As recombinant von Willebrand factor (rVWF; vonicog alfa) may reduce the frequency of treated spontaneous bleeding events (BEs), we investigated the efficacy and safety of rVWF prophylaxis in adults with severe VWD. Patients with BEs requiring VWF therapy in the past year (on-demand VWF therapy [prior on-demand group] or plasma-derived VWF prophylaxis [pdVWF; switch group]) were enrolled in a prospective, open-label, nonrandomized, phase 3 study. The planned duration of rVWF prophylaxis was 12 months; starting rVWF dose was 50 ± 10 VWF: ristocetin cofactor (VWF:RCo) IU/kg twice weekly (prior on-demand group) or based on prior pdVWF weekly dose/dosing frequency (switch group). The primary endpoint was annualized bleeding rate (ABR) of treated spontaneous BEs (sABR) during rVWF prophylaxis. Over the 12-month study period, treated sABR decreased by 91.5% on-study vs historical sABR in 13 patients in the prior on-demand group, and by 45.0% in 10 patients in the switch group (model-based analysis ratio, 0.085; 95% confidence interval [CI], 0.021-0.346 and 0.550; 95% CI, 0.086-3.523, respectively). No treated spontaneous BEs were recorded in 84.6% (11/13) and 70.0% (7/10) of patients, respectively. The safety profile of rVWF was consistent with the previously established profile, with no new adverse drug reactions identified. Findings suggest that rVWF prophylaxis can reduce treated spontaneous BEs in patients previously receiving on-demand VWF therapy and maintains at least the same level of hemostatic control in patients who switch from prophylaxis with pdVWF to rVWF, with a favorable safety profile. This trial was registered at www.clinicaltrials.gov (#NCT02973087) and www.clinicaltrialsregister.eu (#EudraCT 2016-001478-14).
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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.