Evidence map›Paper›PMID 33124022›Full record

Observational studyThrombosis and haemostasis2021

The Use of Recombinant Activated Factor VII in Patients with Glanzmann's Thrombasthenia.

Man-Chiu Poon

Open access · hybridAbstract readMulticenter StudyObservational Study
In one paragraph

Observational study in Thrombosis and haemostasis, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
22citing papers in PubMed, 1 pooled it
2.4field-weighted citation impact, top 9% 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

22 citing papers in PubMed, 1 synthesis or guideline pooled it, 32 citations in OpenAlex.

  1. Efficacy and safety of recombinant activated factor VII in Glanzmann thrombasthenia: A systematic literature review.Haemophilia : the official journal of the World Federation of Hemophilia · 2025
    Pooled it
  2. Article
  3. Observational
  4. Article
  5. European Management of Glanzmann's Thrombasthenia: A Survey of Current Clinical Practice.Haemophilia : the official journal of the World Federation of Hemophilia · 2025
    Article
  6. Review
  7. Article
  8. Obstetric and Gynaecological Challenges and Outcomes in Women and Girls With Glanzmann's Thrombasthenia.Haemophilia : the official journal of the World Federation of Hemophilia · 2025
    Article
  9. Article
  10. Review
  11. Saudi expert consensus on acquired hemophilia A diagnosis and management.Journal of Taibah University Medical Sciences · 2024
    Article
  12. Stopping the bleed when platelets don't stick.Nature cardiovascular research · 2024
    Article
  13. Research and practice in thrombosis and haemostasis · 2024
    Article
  14. Article
  15. Review
  16. Article
  17. Article
  18. Article
  19. Novel homozygous silent mutation ofFrontiers in pediatrics · 2022
    Article
  20. 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

1 author at 1 institution in 1 country.

Man-Chiu PoonDepartment of Medicine, Pediatrics and Oncology, University of Calgary Cumming School of Medicine, Calgary, Alberta, Canada.ORCID 0000-0002-7012-2420
University of Calgary · CA

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Platelet transfusion is the standard treatment to control or prevent bleeding in patients with Glanzmann's thrombasthenia (GT), but platelets are often unavailable. Recombinant activated factor VII (rFVIIa) is an effective alternative to platelets in patients with GT with past/present refractoriness to platelet transfusions and antibodies to platelets. However, there is an unmet need for an alternative to platelets in patients without antibodies. This report summarizes evidence of efficacy and safety of rFVIIa in patients with GT without refractoriness or antibodies to platelets from three different sources: the Glanzmann's Thrombasthenia Registry (GTR), published literature (January 01, 1999 to December 01, 2017), and the Novo Nordisk safety surveillance database. In the GTR, 133 patients received rFVIIa for the treatment of 333 bleeding episodes and prevention of bleeding in 157 surgical procedures. Overall efficacy rates were 79 and 88%, respectively, in patients treated for bleeding episodes or for the prevention of bleeding during surgery; effectiveness was generally similar across refractoriness/antibody status categories. Median dose per infusion of rFVIIa was close to that recommended for patients with GT (90 µg/kg). Data from 14 published case reports also demonstrated that rFVIIa is effective with an acceptable safety profile in patients with GT without antibodies to platelets. Analysis of adverse events reported in GTR and in Novo Nordisk safety surveillance database did not raise any new safety concerns. These data supported the label extension of rFVIIa to include cases where platelets are not readily available, which was approved by the European Medicines Agency in December 2018.

Indexed as

Factor VIIaThrombastheniaAdolescentAdultChildFemaleHemorrhageHumansMaleProspective StudiesRecombinant ProteinsRegistriesTreatment OutcomeYoung AdultFactor VIIarecombinant FVIIaRecombinant Proteins

Identifiers

PMID33124022
PMCPMC7895543
OpenAlexW3094955757

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

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.