Evidence map›Paper›PMID 40224272›Full record

ArticleResearch and practice in thrombosis and haemostasis2025

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.

Robert F Sidonio, Leonid Dubey, Kateryna V Vilchevska, Adlette Inati, Claudia Djambas Khayat

Abstract read
In one paragraph

Article in Research and practice in thrombosis and haemostasis, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.

0numbers the graph read from it
0cells of the map it votes in
5citing 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

5 citing papers in PubMed.

  1. Trial
  2. Article
  3. von Willebrand disease: an illustrated review.Research and practice in thrombosis and haemostasis · 2026
    Review
  4. Article
  5. 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

5 authors.

Robert F SidonioDepartment of Pediatrics, Emory University School of Medicine, Atlanta, Georgia, USA.
Leonid DubeyCommunal Nonprofit Enterprise "Western Ukrainian Specialized Children's Medical Center" of Lviv Regional Council, Lviv, Ukraine.
Kateryna V VilchevskaNational Specialized Children's Hospital Okhmatdyt, Center of Hemostasis Pathologies, Kyiv, Ukraine.
Adlette InatiNINI Hospital and University of Balamand School of Medicine and Medical Sciences, Balamand, Lebanon.
Claudia Djambas KhayatHotel Dieu de France Hospital, Saint Joseph University, Beirut, Lebanon.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Background: Prophylaxis with von Willebrand factor is recommended in people with severe von Willebrand disease (VWD), regardless of age. WIL-31, the only prospective study with an on-demand run-in study as an intraindividual comparator, demonstrated the efficacy and safety of prophylaxis with the plasma-derived von Willebrand factor/factor VIII concentrate wilate (Octapharma) in adults and children with VWD of all types. Prophylaxis is often considered in young children and adolescents with severe VWD and recurrent bleeding, although limited data support this strategy. Objectives: To assess the efficacy of wilate prophylaxis in children (6-11 years) and adolescents (12-16 years) in WIL-31. Methods: Patients received 20 to 40 IU/kg (Octapharma) wilate prophylaxis 2 to 3 times weekly for 12 months. Results were compared with prospective on-demand treatment. Results: Mean total annualized bleeding rates (ABRs) during on-demand vs prophylaxis were 32.5 vs 3.7 in children ( Conclusion: wilate prophylaxis was efficacious and well-tolerated in children and adolescents with all types of VWD.

Indexed as

adolescentchildhemostaticsprevention and controlvon Willebrand diseasevon Willebrand factor

Identifiers

PMID40224272
PMCPMC11992423

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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.