Evidence map›Paper›PMID 32445594›Full record

Observational studyJournal of thrombosis and haemostasis : JTH2020

Management of von Willebrand disease with a factor VIII-poor von Willebrand factor concentrate: Results from a prospective observational post-marketing study.

Jenny Goudemand, Françoise Bridey, Ségolène Claeyssens, Nathalie Itzhar-Baïkian, Annie Harroche, Dominique Desprez, Claude Négrier, Pierre Chamouni, Hervé Chambost, Céline Henriet and 2 more

Abstract readObservational Study
In one paragraph

Observational study in Journal of thrombosis and haemostasis : JTH, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers.

0numbers the graph read from it
0cells of the map it votes in
18citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from 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.

2 · The registry

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.

3 · Its place in the literature

Who cites it

18 citing papers in PubMed.

  1. Trial
  2. Review
  3. Von Willebrand disease as a predictor of postoperative hemorrhagic complications in pediatric adenotonsillar surgery: a retrospective cohort study.European archives of oto-rhino-laryngology : official journal of the European Federation of Oto-Rhino-Laryngological Societies (EUFOS) : affiliated with the German Society for Oto-Rhino-Laryngology - Head and Neck Surgery · 2026
    Article
  4. Review
  5. Observational
  6. Article
  7. Article
  8. Article
  9. Article
  10. Review
  11. Article
  12. Observational
  13. Review
  14. Observational
  15. Article
  16. Safety and Efficacy of the Use of pdVWF/FVIII-C in Patients with von Willebrand Disease: A Prospective, Observational, Post-Authorization Study.Clinical and applied thrombosis/hemostasis : official journal of the International Academy of Clinical and Applied Thrombosis/Hemostasis
    Observational
  17. Real-World Efficacy and Safety of Plasma-Derived Von Willebrand Factor-Containing Factor VIII Concentrates in Patients With Von Willebrand Disease in Italy.Clinical and applied thrombosis/hemostasis : official journal of the International Academy of Clinical and Applied Thrombosis/Hemostasis
    Observational
  18. The Cost of Von Willebrand Disease in Europe: The CVESS Study.Clinical and applied thrombosis/hemostasis : official journal of the International Academy of Clinical and Applied Thrombosis/Hemostasis
    Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

12 authors.

Jenny GoudemandDepartment of Hemostasis and Transfusion, Lille University Hospital, Lille, France.
Françoise BrideyClinical Development, Laboratoire français du Fractionnement et des Biotechnologies (LFB), Les Ulis, France.
Ségolène ClaeyssensCentre de Ressources et Compétences, Maladies Hémorragiques Constitutionnelles, CRC MHC URM, Purpan Hospital, Toulouse, France.
Nathalie Itzhar-BaïkianService d'Hématologie Biologique, Hôpital Lariboisière, APHP, Paris, France.
Annie HarrocheHemophilia Care Center, Necker Hospital, Paris, France.
Dominique DesprezCentre de compétences trouble de l'hémostase, Hopital de Hautepierre, Strasbourg, France.
Claude NégrierHematology Division, Hemophilia Comprehensive Care Center, Louis Pradel Hospital, University Lyon1, Bron, France.
Pierre ChamouniHemophilia Care Center, Rouen University Hospital, Rouen, France.
Hervé ChambostAPHM Centre for Bleeding Disorders, La Timone Children Hospital and Aix-Marseille University, INRA, Inserm, Marseille, France.
Céline HenrietClinical Development, Laboratoire français du Fractionnement et des Biotechnologies (LFB), Les Ulis, France.
Sophie SusenDepartment of Hemostasis and Transfusion, Lille University Hospital, Lille, France.
Annie Borel-DerlonHemophilia Treatment Centre, University Hospital of Caen, Caen, France.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundA triple-secured plasma-derived von Willebrand factor (pdVWF) almost devoid of factor VIII (FVIII):WILFACTIN

objectiveTo investigate long-term safety and efficacy of the product in real-life over the first 5 post-approval years. PATIENTS/

methodsThis prospective, observational, national post-marketing study (PMS) enrolled patients of all ages and VWD types. Patients were observed for up to 3 years and treated for one or more occasions. Efficacy was assessed for each major event. Breakthrough bleeding rate 3 days post-infusion and annualized bleeding rate (ABR) were also evaluated for long-term prophylaxis.

resultsOverall, 155 of 174 patients enrolled from 31 centers were eligible for efficacy assessment. Most patients (76.8%) were severely affected (VWF:RCo ≤ 15 IU/dL). They were treated for 743 bleeds and 140 surgeries including childbirth. Efficacy outcomes were excellent/good for 98.2% of 56 major surgeries and 94.0% of 67 major bleeds. Approximately 75% of 49 major mucosal bleeds were effectively managed without FVIII co-administration. In 32 patients receiving prophylaxis, breakthrough bleeding occurred in 1.5% of infusions and median ABR was 1.0 for 20 patients treated ≥ 12 months. Excellent tolerability was confirmed with no safety concerns. No thrombotic events were observed.

conclusionsResults from this PMS increase the clinical experience of a FVIII-poor pdVWF in patients of all ages and VWD types including those with thrombotic risk factors and emphasize that giving FVIII is not always mandatory to effectively treat patients with severe VWD.

Indexed as

von Willebrand Diseasesvon Willebrand FactorFactor VIIIFemaleFranceHumansMarketingProspective StudiesFactor VIIIvon Willebrand Factorclinical trialfactor VIIIpost-marketingvon Willebrand diseasevon Willebrand factor

Identifiers

PMID32445594
PMCPMC7496521

What OpenQuestion holds

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LicenceCC BY-NC-ND
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Registered trials

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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.