Evidence map›Paper›PMID 38237075›Full record

ArticleBlood advances2024

von Willebrand factor/factor VIII concentrate (Wilate) prophylaxis in children and adults with von Willebrand disease.

Robert F Sidonio, Ana Boban, Leonid Dubey, Adlette Inati, Csongor Kiss, Zoltan Boda, Toshko Lissitchkov, Laszlo Nemes, Dzmitry Novik, Elina Peteva and 7 more

2 registry-linked trialsOpen access · goldAbstract read
In one paragraph

Article in Blood advances, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 20 papers.

0numbers the graph read from it
0cells of the map it votes in
20citing papers in PubMed
8.2field-weighted citation impact, top 2% of its field
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.

NCT04052698 phase3completednot on this map

Clinical Study to Investigate the Efficacy and Safety of Wilate During Prophylaxis in Previously Treated Patients With Von Willebrand Disease (VWD)

TypeinterventionalSponsorOctapharmaRan2020 to 2022Enrolled43ConditionsVon Willebrand DiseasesArmsWilate
NCT04053699 completednot on this map

A Prospective, Multicenter, Non-Interventional Study Evaluating the Bleeding Incidence in Patients With Von Willebrand Disease Undergoing On-Demand Treatment

TypeobservationalSponsorOctapharmaRan2019 to 2021Enrolled56ConditionsVon Willebrand DiseasesArmsVon Willebrand Factor-Containing Product
3 · Its place in the literature

Who cites it

20 citing papers in PubMed, 18 citations in OpenAlex.

  1. Trial
  2. 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/Hemostasis
    Trial
  3. Article
  4. Seventh Åland Island Meeting on von Willebrand Disease.Haemophilia : the official journal of the World Federation of Hemophilia · 2026
    Article
  5. Menstrual outcomes are frequently overlooked in von Willebrand disease trials.Research and practice in thrombosis and haemostasis · 2026
    Article
  6. Article
  7. Review
  8. Article
  9. Review
  10. Observational
  11. Von Willebrand disease diagnosis: from complexity to simplicity.Research and practice in thrombosis and haemostasis · 2025
    Article
  12. Article
  13. 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 · 2025
    Article
  14. Review
  15. Article
  16. Article
  17. Article
  18. Article
  19. Article
  20. Heavy Menstrual Bleeding and Hormonal Therapy in Women with Type 1 von Willebrand Disease Enrolled on the Zimmerman Program.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

17 authors at 16 institutions in 9 countries.

Robert F SidonioDepartment of Pediatrics, Emory University School of Medicine, Atlanta, GA.
Ana BobanDepartment of Internal Medicine, University Hospital Centre Zagreb, Zagreb University School of Medicine, Zagreb, Croatia.ORCID 0000-0003-3532-2336
Leonid DubeyCommunal Nonprofit Enterprise "Western Ukrainian Specialized Children's Medical Center" of Lviv Regional Council, Lviv, Ukraine.
Adlette InatiDepartment of Pediatrics, NINI Hospital, Tripoli, Lebanon.
Csongor KissDivision of Pediatric Hematology-Oncology, Department of Pediatrics, Faculty of Medicine, University of Debrecen, Debrecen, Hungary.ORCID 0000-0001-5170-5965
Zoltan BodaDivision of Hematology, Department of Internal Medicine, Faculty of Medicine, University of Debrecen, Debrecen, Hungary.
Toshko LissitchkovDepartment of Chemotherapy, Hemotherapy and Hereditary Blood Diseases at Clinical Hematology Clinic, Specialized Hospital for Active Treatment of Hematological Diseases, Sofia, Bulgaria.
Laszlo NemesMedical Centre of Hungarian Defence Forces, Budapest, Hungary.
Dzmitry NovikGovernment Agency "Republican Research Center for Radiation Medicine and Human Ecology," Gomel, Belarus.
Elina PetevaPediatric Clinic of Haematology and Oncology, University Hospital St. Marina-Varna, Varna, Bulgaria.
Ali T TaherDivision of Hematology-Oncology, Department of Internal Medicine, American University of Beirut Medical Center, Beirut, Lebanon.
Margarita Arkadevna TimofeevaFederal State Budgetary Scientific Institution Kirov Scientific-Research Institute of Hematology and Blood Transfusion of Federal Medical and Biological Agency, Kirov, Russia.
Kateryna V VilchevskaNational Specialized Children's Hospital Okhmatdyt, Kyiv, Ukraine.
Vladimir VdovinMorozovskaya Children's City Clinical Hospital, Moscow, Russia.
Sylvia WernerOctapharma USA, Paramus, NJ.
Sigurd KnaubOctapharma AG, Lachen, Switzerland.
Claudia Djambas KhayatHotel Dieu de France Hospital, Saint Joseph University, Beirut, Lebanon.
University of Debrecen · HUAmerican University of Beirut Medical Center · LBChildren's Specialized Hospital · USEmory University · USKirov Research Institute of Hematology and Blood Transfusion FMBA · RULviv City Children's Hospital · UANational Specialized Hospital for Active Treatment of Hematologic Diseases · BGNew Jersey Department of Education · USNini Hospital · LBOctapharma (Switzerland) · CHRepublican Research Center for Radiation Medicine and Human Ecology · BYRussian Children's Clinical Hospital · RUSaint Joseph University · LBUniversity Hospital Centre Zagreb · HRUniversity Hospital St. Marina · BGZrínyi Miklós National Defence University · HU

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

abstractLong-term prophylaxis with a von Willebrand factor (VWF) concentrate is recommended in patients with von Willebrand disease (VWD) who have a history of severe and frequent bleeds. However, data from prospective studies are scarce. WIL-31, a prospective, noncontrolled, international phase 3 trial, investigated the efficacy and safety of Wilate prophylaxis in severe patients with VWD. Male and female patients 6 years or older with VWD types 1, 2 (except 2N), or 3 who had completed a prospective, 6-month, on-demand, run-in study (WIL-29) were eligible to receive Wilate prophylaxis for 12 months. At baseline, patients (n = 33) had a median age of 18 years. Six (18%) patients had severe type 1, 5 (15%) had type 2, and 22 (67%) had type 3 VWD. The primary end point of a >50% reduction in mean total annualized bleeding rate (TABR) with Wilate prophylaxis vs prior on-demand treatment was met; mean TABR during prophylaxis was 5.2, representing an 84.4% reduction. The bleeding reduction was consistent across age, sex, and VWD types. The mean spontaneous ABR was 3.2, representing an 86.9% reduction vs on-demand treatment. During prophylaxis, 10 (30.3%) patients had 0 bleeding events and 15 (45.5%) patients had 0 spontaneous bleeding events. Of 173 BEs, 84.4% were minor and 69.9% treated. No serious adverse events related to study treatment and no thrombotic events were recorded. Overall, WIL-31 showed that Wilate prophylaxis was efficacious and well-tolerated in pediatric and adult patients with VWD of all types. The WIL-29 and WIL-31 trials were registered at www.ClinicalTrials.gov as #NCT04053699 and #NCT04052698, respectively.

Indexed as

von Willebrand Diseasesvon Willebrand FactorAdolescentAdultChildFactor VIIIFemaleHemorrhageHumansMaleProspective StudiesFactor VIIIvon Willebrand Factor

Identifiers

PMID38237075
PMCPMC10950830
OpenAlexW4390966025

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

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.