Evidence map›Paper›PMID 39924968›Full record

SynthesisHaemophilia : the official journal of the World Federation of Hemophilia2025

Plasma-Derived von Willebrand Factor/Factor VIII Concentrate (Haemate P) in von Willebrand Disease: A Systematic Review and Pharmacovigilance Update.

Carmen Escuriola Ettingshausen, Riitta Lassila, Gines Escolar, Christoph Male, Kathrin Schirner, Lisa Heyder, Erik Berntorp

Abstract readSystematic Review
In one paragraph

Synthesis in Haemophilia : the official journal of the World Federation of Hemophilia, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing 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

3 citing papers in PubMed.

  1. Article
  2. Review
  3. Review
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

7 authors.

Carmen Escuriola EttingshausenHaemophilia Centre Rhein Main (HZRM), Frankfurt/Main, Germany.ORCID https://orcid.org/0000-0003-0802-5280
Riitta LassilaDepartment of Haematology, Helsinki University Hospital, Coagulation Disorders Unit, Comprehensive Cancer Centre, and Research Program Unit in System Oncology, University of Helsinki, Helsinki, Finland.ORCID https://orcid.org/0000-0002-1911-3094
Gines EscolarDepartment of Pathology, Hematopathology, Centre de Diagnostic Biomedic (CDB), Hospital Clinic de Barcelona, Institut d'Investigacions Biomediques August Pi i Sunyer (IDIBAPS), Universitat de Barcelona, Barcelona, Spain.ORCID https://orcid.org/0000-0002-6658-0381
Christoph MaleDepartment of Paediatrics, Medical University of Vienna, Vienna, Austria.ORCID https://orcid.org/0000-0002-4989-6421
Kathrin SchirnerCSL Innovation GmbH, Marburg, Germany.ORCID https://orcid.org/0000-0002-4705-8475
Lisa HeyderCSL Innovation GmbH, Marburg, Germany.ORCID https://orcid.org/0000-0003-0977-6422
Erik BerntorpDepartment of Translational Medicine, Clinical Coagulation Research Unit, Lund University, Malmö, Sweden.ORCID https://orcid.org/0000-0002-1337-7195

Funding

CSL Behring
6 · The paper itself

Abstract

backgroundVon Willebrand disease (VWD) is an inherited bleeding disorder caused by deficient or dysfunctional von Willebrand factor (VWF). VWF replacement therapy is indicated in VWD management.

methodsThis systematic review was conducted to evaluate all available evidence of the efficacy, safety, dosing and consumption of pasteurized plasma-derived human coagulation FVIII/human VWF (pdVWF/FVIII; Haemate P/Humate-P) concentrate for on-demand (OD) treatment, surgical prophylaxis and long-term prophylaxis of patients with VWD. A systematic search was performed in MEDLINE and Cochrane Library databases to identify studies (7 June 1982-31 May 2023) reporting the use of pdVWF/FVIII in VWD according to predefined selection criteria. Pharmacovigilance data were also retrieved for the same period.

resultsFifteen studies were identified, 12 being observational and three interventional. Efficacy and safety assessments and treatment protocols varied across the studies which hindered direct comparisons. Haemostatic efficacy of pdVWF/FVIII was rated excellent/good for OD treatment in 95%-98% of bleeds and in 94%-100% of surgeries. In two separate studies, prophylactic efficacy was rated excellent/good in 100% of treatment cycles. Where reported, median annualized bleeding rates decreased from 3-24 prior prophylaxis to 0.5-6 during prophylaxis. Analysis of pharmacovigilance safety reports showed that pdVWF/FVIII was associated with a low rate of adverse events.

conclusionsThis systematic literature review and analysis of pharmacovigilance data summarize evidence of over 40 years of clinical use of pdVWF/FVIII, supporting its safety and efficacy in VWD.

Indexed as

Factor VIIIvon Willebrand Diseasesvon Willebrand FactorDrug CombinationsHemorrhageHumansPharmacovigilanceDrug CombinationsFactor VIIIfactor VIII, von Willebrand factor drug combinationvon Willebrand Factorfactor VIIIsafetysystematic reviewvon Willebrand diseasevon Willebrand factor

Identifiers

PMID39924968
PMCPMC11957426

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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.