Evidence map›Paper›PMID 38272065›Full record

SynthesisThrombosis and haemostasis2024

A Systematic Review of Efficacy and Safety of Plasma-Derived von Willebrand Factor/Factor VIII Concentrate (Voncento) in von Willebrand Disease.

Lucia Rugeri, Will Thomas, Kathrin Schirner, Lisa Heyder, Günter Auerswald

Open access · hybridAbstract readSystematic Review
In one paragraph

Synthesis in Thrombosis and haemostasis, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed
0.5field-weighted citation impact, top 39% 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.

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

2 citing papers in PubMed, 1 citations in OpenAlex.

  1. Review
  2. 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 at 4 institutions in 3 countries.

Lucia RugeriUnite d'Hemostase Clinique, Hôpital Cardiologique, Hospices Civils de Lyon, Lyon, France.ORCID 0000-0002-3103-1737
Will ThomasCambridge University Hospitals NHS Foundation Trust, Cambridge Biomedical Campus, Cambridge, United Kingdom.ORCID 0000-0001-8740-0194
Kathrin SchirnerCSL Behring Innovation GmbH, Marburg, Germany.ORCID 0000-0002-4705-8475
Lisa HeyderCSL Behring Innovation GmbH, Marburg, Germany.ORCID 0000-0003-0977-6422
Günter AuerswaldCoagulation Centre, Bremen Central Clinic, GeNo Ltd., Parent-Child-Centre Prof. Hess, Bremen, Germany.ORCID 0000-0003-3517-7052
Behringwerke (Germany) · DECambridge University Hospitals NHS Foundation Trust · GBLille’s Cardiology Hospital · FRProf. Hess Kinderklinik · DE

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundFor the treatment of von Willebrand disease (VWD), von Willebrand factor (VWF) concentrates can be used in on-demand, long-term prophylaxis, and surgical prophylaxis regimens.

methodsThis systematic literature review was conducted to evaluate the efficacy, consumption, and safety of plasma-derived human coagulation FVIII/human VWF (pdVWF/FVIII; Voncento/Biostate) for the treatment of patients with any inherited VWD type. An electronic search was conducted in MEDLINE and Cochrane Library databases on VWD therapies. All retrieved publications were assessed against predefined inclusion/exclusion criteria following the Cochrane group recommendations. Associated pharmacovigilance data were collected across the same time period.

resultsEleven publications from eight study cohorts were identified for data retrieval. All were from multicenter studies and included both pediatric and adult patients. Eight publications included evaluations of the efficacy of pdVWF/FVIII for on-demand treatment, eight included long-term prophylactic treatment, and eight included surgical prophylaxis. Treatment protocols and VWF administration methods differed between studies, as did safety evaluations. The clinical response was rated as excellent/good for on-demand treatment in 66 to 100% of nonsurgical bleeds, 89 to 100% in the treatment of breakthrough bleeds during long-term prophylaxis treatment, and hemostatic efficacy in surgical procedures was 75 to 100%. Pharmacovigilance data confirmed a low incidence of adverse events in treated patients.

conclusionThis review provides a comprehensive summary of studies that evaluated the use of pdVWF/FVIII in VWD demonstrating the long-term effectiveness and safety of this pdVWF/FVIII across all ages, types of VWD, and treatment settings.

Indexed as

Factor VIIIHemorrhagevon Willebrand Diseasesvon Willebrand FactorAdultChildDrug CombinationsHumansPharmacovigilanceTreatment OutcomeDrug CombinationsFactor VIIIfactor VIII, von Willebrand factor drug combinationvon Willebrand Factor

Identifiers

PMID38272065
PMCPMC11349426
OpenAlexW4391215616

What OpenQuestion holds

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LicenceCC BY-NC-ND
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Registered trials

None linked

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.