Observational studyEuropean journal of haematology2022
Effectiveness of long-term prophylaxis using pdFVIII/VWF concentrate in patients with inherited von Willebrand disease.
Observational study in European journal of haematology, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 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.
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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
10 citing papers in PubMed, 1 synthesis or guideline pooled it, 11 citations in OpenAlex.
- A Systematic Review of Efficacy and Safety of Plasma-Derived von Willebrand Factor/Factor VIII Concentrate (Voncento) in von Willebrand Disease.Thrombosis and haemostasis · 2024Pooled it
- 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
- von Willebrand disease: an illustrated review.Research and practice in thrombosis and haemostasis · 2026Review
- Real-word evidence on healthcare resource use and associated costs in on-demand users of replacement therapies in von Willebrand disease in France: the FORvWARD study.Journal of comparative effectiveness research · 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
- Efficacy of regular prophylaxis with a plasma-derived von Willebrand factor/factor VIII concentrate with a 1:1 activity ratio in reducing heavy menstrual bleeding in girls/women with von Willebrand disease.AJOG global reports · 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
- Effectiveness of long-term prophylaxis using pdFVIII/VWF concentrate in patients with inherited von Willebrand disease.European journal of haematology · 2022Observational
Corrections and comments
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Authors and funding
14 authors at 7 institutions in 1 country.
Funding
Abstract
backgroundPatients with symptomatic von Willebrand disease (VWD) should be offered long-term prophylaxis (LTP) to prevent recurrent bleedings. Our objective was to evaluate the effectiveness and safety of Voncento®, a plasma-derived FVIII/VWF concentrate (ratio 1:2.4), administrated in LTP.
methodsWe included patients from the OPALE study (May 2016 to April 2021), a French multicenter observational study following patients with inherited VWD, who received a Voncento® LTP during the study period.
resultsAmong the 130 OPALE-study patients, 23 patients (12 women) received a LTP and were therefore included. The median (range) age was 16 (1-85) years; 16 patients were type 3, 1 was type 2A, 6 were type 2B. Before inclusion, 19 (83%) were under LTP and 4 (17%) received on-demand (OD) treatment. The indications for initiating prophylaxis in the overall population were joint bleeding (43%), ear, nose, and throat (ENT) bleeding including epistaxis or oral bleeding (39%), and recurrent muscle hematoma (22%). The medians (ranges) dose of Voncento® per infusion, frequency, and weekly dose were 45 (33-109) IU/kg, 2 infusions per week, and 96 (44-222) IU/kg/week, respectively. The median (range) annualized bleeding rate (ABR) was 0.8, 0.7 (0-3.5), and 0 (0-2.3) for type 2A, 2B, 3 patients, respectively. There was no difference regarding to the dose, frequency of infusion, or in terms of ABR in 9/19 patients who replaced previous concentrates with Voncento®. During the study period, no adverse event was reported.
conclusionThese results suggest that Voncento® is effective to prevent recurrent bleedings in patients symptomatic VWD.
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