Trial reportBlood transfusion = Trasfusione del sangue2019
A phase III study comparing secondary long-term prophylaxis versus on-demand treatment with vWF/FVIII concentrates in severe inherited von Willebrand disease.
Trial report in Blood transfusion = Trasfusione del sangue, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 32 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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
32 citing papers in PubMed, 2 syntheses or guidelines pooled it, 45 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
- Trial
- 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
- Type 3 Von Willebrand disease: two clinical cases of a rare disorder.Oxford medical case reports · 2026Article
- Historical, current and future treatments for von Willebrand disease.Haematologica · 2026Review
- Prophylaxis for von Willebrand disease: Is it time for parity with established practice in hemophilia A?Therapeutic advances in hematology · 2026Review
- Bridging the Evidence Gap in von Willebrand Disease: A Call to Action for Equitable, Evidence-Based Care.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Article
- Article
- Cost-effectiveness of Voncento prophylaxis vs on-demand treatment in von Willebrand disease in the United Kingdom.Blood advances · 2025Article
- Efficacy and safety of plasma-derived von Willebrand factor/factor VIII concentrate (wilate) prophylaxis in children and adolescents with von Willebrand disease - WIL-31 study post hoc analysis.Research and practice in thrombosis and haemostasis · 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
- Special considerations in GI bleeding in VWD patients.Hematology. American Society of Hematology. Education Program · 2022Review
- Impact of von Willebrand Disease on Women's Health Outcomes: A Matched Cohort Database Study.Journal of women's health (2002) · 2022Article
- Effectiveness of long-term prophylaxis using pdFVIII/VWF concentrate in patients with inherited von Willebrand disease.European journal of haematology · 2022Observational
- Gynecologic and obstetric management of women with von Willebrand disease: summary of 3 systematic reviews of the literature.Blood advances · 2022Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
13 authors at 2 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundThere is a lack of prospective clinical trials specifically designed to evaluate the benefits of prophylaxis with vWF/FVIII concentrates in patients with inherited von Willebrand disease (vWD). The aim of the study was to compare efficacy of secondary long-term prophylaxis (PRO) with vWF/FVIII in the prevention of bleeding episodes in severe vWD patients to standard of care (on-demand treatment; ODT). MATERIALS AND
methodsIn this 12-month, phase III, open-label study (PRO.WILL), vWD patients (aged ≥6 years) were randomised to PRO (n=9; 5 completed) or ODT (n=10; 7 completed) treatment with Fanhdi
resultsAll patients on ODT had vWD type 2 or 3 vs 70% of patients on PRO. All ODT patients experienced bleeds vs 60% on PRO. PRO patients showed fewer bleeds (n=32 vs n=172 [112 in the same patient, mostly mucosal]; p<0.0001) and lower risk of bleeding (relative attributable risk estimate: -0.667; 95% CI: -2.374, -0.107; p<0.001). Most frequent bleeds in ODT and PRO groups were, respectively, epistaxis (n=52 vs n=15) and gastrointestinal (n=13 [9 in the same patient] vs n=1). While most bleeds lasted one day under ODT (31/32), only epistaxis did so in PRO group (14/15). No AEs due to study medication were observed. DISCUSSION: Despite the small sample size and the heterogeneity of the study population, patients on vWF/FVIII prophylaxis showed a reduction in bleeding risk and rate compared to on-demand treatment.
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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.