Evidence map›Paper›PMID 38193044›Full record

ArticleResearch and practice in thrombosis and haemostasis2023

Inhibitor development according to concentrate in severe hemophilia: reporting on 1392 Previously Untreated Patients from Europe and Canada.

Kathelijn Fischer, Riitta Lassila, Flora Peyvandi, Alexander Gatt, Rob Hollingsworth, Thierry Lambert, Radek Kaczmarek, Amanda Bettle, Nasrin Samji, Georges-Étienne Rivard and 4 more

Open access · goldAbstract read
In one paragraph

Article in Research and practice in thrombosis and haemostasis, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.

0numbers the graph read from it
0cells of the map it votes in
11citing papers in PubMed
4.1field-weighted citation impact, top 6% 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

11 citing papers in PubMed, 14 citations in OpenAlex.

  1. Extension Study With rVIII-SingleChain in Previously Untreated Patients (PUPs) With Severe Haemophilia A.Haemophilia : the official journal of the World Federation of Hemophilia · 2025
    Trial
  2. Extended Half-Life Factor Concentrates in Haemophilia Treatment.Transfusion medicine and hemotherapy : offizielles Organ der Deutschen Gesellschaft fur Transfusionsmedizin und Immunhamatologie · 2026
    Review
  3. When and How to Start Prophylaxis in Children with Hemophilia.Transfusion medicine and hemotherapy : offizielles Organ der Deutschen Gesellschaft fur Transfusionsmedizin und Immunhamatologie · 2026
    Review
  4. Shaping hemophilia care: lessons and legacy of the SIPPET trial after 10 years.Research and practice in thrombosis and haemostasis · 2026
    Review
  5. Review
  6. Article
  7. Article
  8. Article
  9. Trends in Treatment of Severe Haemophilia and Impact on Inhibitor Assessment by the EUHASS Registry.Haemophilia : the official journal of the World Federation of Hemophilia · 2025
    Article
  10. Article
  11. 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

14 authors at 5 institutions in 8 countries.

Kathelijn FischerCenter for Benign Haematology, Thrombosis and Haemostasis, Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht, The Netherlands.
Riitta LassilaDepartment of Hematology, Unit of Coagulation Disorders, Helsinki University Central Hospital, Helsinki, Finland.
Flora PeyvandiAngelo Bianchi Bonomi Hemophilia and Thrombosis Centre, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
Alexander GattMater Dei Hospital, Tal-QRoqq, Msida, Malta.
Rob HollingsworthMDSAS, Manchester, United Kingdom.
Thierry LambertHopital Bicetre, Paris, France.
Radek KaczmarekCoagulation Products Safety Supply and Access Committee, World Federation of Hemophilia, Montreal, Quebec, Canada.
Amanda BettleIWK Health Centre, Halifax, Nova Scotia, Canada.
Nasrin SamjiDepartment of Pediatrics, Division of Hematology-Oncology, McMaster University, Hamilton, Ontario, Canada.
Georges-Étienne RivardDepartment of Pediatrics, Division of Hematology-Oncology, CHU Sainte-Justine, Université de Montréal, Montreal, Quebec, Canada.
Manuel CarcaoDivision of Haematology/Oncology, Hospital for Sick Children, Toronto, Ontario, Canada.
Alfonso IorioDepartment of Clinical Epidemiology and Biostatistics, McMaster University, Toronto, Ontario, Canada.
Mike MakrisDepartment of Infection, Immunity and Cardiovascular Disease, University of Sheffield, Sheffield, United Kingdom.
EUHASS and CHESS participants
University Medical Center Utrecht · NLBicêtre Hospital · FRIndiana University – Purdue University Indianapolis · USUniversity of Sheffield · GBUtrecht University · NL

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Background: Clotting factor concentrates have been the mainstay of severe hemophilia treatment over the last 50 years. Differences in risk of neutralizing antibody (inhibitor) formation according to concentrate used remain clinically relevant. Objectives: To assess inhibitor development according to type of clotting factor concentrate in previously untreated patients (PUPs) with severe hemophilia A and B. Methods: The European Haemophilia Safety Surveillance (EUHASS) and Canadian Bleeding Disorders Registry (CBDR) have been monitoring adverse events overall and according to concentrate for 11 and 8 years, respectively. Inhibitors were reported quarterly, and PUPs completed 50 exposure days without inhibitor development annually. Cumulative inhibitor incidences and 95% confidence intervals (CIs) were compared without adjustment for other risk factors. Results: Fifty-six European and 23 Canadian centers reported inhibitor development in 312 of 1219 (26%; CI, 23%-28%) PUPs with severe hemophilia A and 14 of 173 (8%; CI, 5%-13%) PUPs with severe hemophilia B. Inhibitor development was lower on plasma-derived factor (F)VIII (pdFVIII, 20%; CI, 14%-26%) than on standard half-life recombinant FVIII (SHL-rFVIII, 27%; CI, 24%-30% and odds ratio, 0.67; CI, 0.45%-0.98%; Conclusion: While confirming expected rates of inhibitors in PUPs, inhibitor development was lower in pdFVIII than in SHL-rFVIII. Preliminary data suggest variation in inhibitor development among different SHL-rFVIII and EHL-rFVIII concentrates.

Indexed as

antibodiesfactor VIIIhemophilia Ahemophilia Bneutralizingregistries

Identifiers

PMID38193044
PMCPMC10772871
OpenAlexW4388822200

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.