Evidence map›Paper›PMID 33117020›Full record

ArticleJournal of blood medicine2020

An Open-Label Extension Study to Assess the Long-Term Efficacy and Safety of a Plasma-Derived von Willebrand Factor (VWF)/Factor VIII (FVIII) Concentrate in Patients with von Willebrand Disease (SWIFT-VWDext Study).

Toshko Lissitchkov, Anna Klukowska, Evgeny Buevich, Irina Maltceva, Guenter Auerswald, Oleksandra Stasyshyn, Wilfried Seifert, Tobias Rogosch

Open access · goldAbstract read
In one paragraph

Article in Journal of blood medicine, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
5citing papers in PubMed, 1 pooled it
0.1field-weighted citation impact, top 50% 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

5 citing papers in PubMed, 1 synthesis or guideline pooled it, 4 citations in OpenAlex.

  1. Pooled it
  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. Review
  4. Article
  5. Observational
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

8 authors at 6 institutions in 5 countries.

Toshko LissitchkovSpecialized Hospital for Active Treatment (SHAT), Sofia, Bulgaria.
Anna KlukowskaDepartment of Paediatrics, Haematology and Oncology of Warsaw Medical University, Warsaw, Poland.ORCID 0000-0003-3859-7757
Evgeny BuevichGOUVPO Altaysky State Medical University of Roszdrav, Bernaul, Russian Federation.
Irina MaltcevaGOUVPO Altaysky State Medical University of Roszdrav, Bernaul, Russian Federation.
Guenter AuerswaldProf. Hess Kinderklinik, Klinikum Bremen-Mitte, Bremen, Germany.
Oleksandra StasyshynInstitute of Pathology and Transfusion Medicine AMN, Lviv, Ukraine.ORCID 0000-0002-8366-5926
Wilfried SeifertClinical Development, CSL Behring, Marburg, Germany.
Tobias RogoschClinical Development, CSL Behring, Marburg, Germany.ORCID 0000-0002-1674-1423
Altai State Medical University · RUBehringwerke (Germany) · DEInstitute of Blood Pathology and Transfusion Medicine of the National Academy of Medical Sciences of Ukraine · UAMedical University of Warsaw · PLProf. Hess Kinderklinik · DEUniversity Specialized Hospital for Active Treatment of Endocrinology · BG

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

objectivePlasma-derived von Willebrand factor/factor VIII (pdVWF/FVIII; VONCENTO

methodsPediatric, adolescent, and adult patients with VWD who required treatment of non-surgical bleeds (NSBs), treatment during surgical events or who were receiving prophylaxis and who had completed one of two previous clinical trials of pdVWF/FVIII were included. Efficacy and safety analyses were performed for on-demand (n=10), prophylaxis (n=8), or on-demand and prophylaxis (n=2) treatment in patients pre-treated with pdVWF/FVIII for ≥12 months.

resultsSeven patients experienced a total of 402 NSBs in the on-demand arm, of which 77 required treatment and nine NSB events in three patients were considered major. Nine patients reported 118 NSBs in the prophylaxis arm, with 96 events requiring treatment and seven patients experiencing 12 major NSB events. Excellent or good hemostatic efficacy was reported by the investigator for 98.7% (on-demand) and 97.9% (prophylaxis) of NSB events treated with pdVWF/FVIII, without relevant differences between subgroups by age. pdVWF/FVIII was well tolerated, and the adverse events seen were mild-moderate and consistent with the safety profile for this product seen in other studies. There were no cases of anaphylactic reactions and angioedema, development of VWF/FVIII inhibitors, thromboembolic events, or viral infections.

conclusionThis contemporary comprehensive development program evaluating pdVWF/FVIII across all ages demonstrates long-term safety and efficacy for treatment and prevention of bleeds in patients with severe VWD, supporting the benefit-risk profile of pdVWF/FVIII.

Indexed as

hemostatic efficacyon-demandprophylaxissafetysurgeryvon Willebrand disease

Identifiers

PMID33117020
PMCPMC7553254
OpenAlexW3092283822

What OpenQuestion holds

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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.