Evidence map›Paper›PMID 40547964›Full record

ArticleResearch and practice in thrombosis and haemostasis2025

Inhibitor development in nonsevere hemophilia: data from the European Haemophilia Safety Surveillance (EUHASS) registry.

Kathelijn Fischer, Riitta Lassila, Flora Peyvandi, Alexander Gatt, Samantha C Gouw, Robert Hollingsworth, Thierry Lambert, Radek Kaczmarek, Diana Carbonero Alvarez, Michael Makris and 1 more

Abstract read
In one paragraph

Article in Research and practice in thrombosis and haemostasis, 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. Nonneutralizing Antibodies in Haemophilia A Patients: To Be Ignored or Not to Be!Haemophilia : the official journal of the World Federation of Hemophilia
    Review
  3. Challenges and Opportunities in Post-Marketing Reporting of Factor VIII Inhibitors With Efanesoctocog Alfa.Haemophilia : the official journal of the World Federation of Hemophilia
    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

11 authors.

Kathelijn FischerCenter for Benign Haematology, Thrombosis and Haemostasis, Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, the Netherlands.
Riitta LassilaDepartment of Hematology, Unit of Coagulation Disorders, Helsinki University Central Hospital, Research Program Unit in Systems Oncology, University of Helsinki, Helsinki, Finland.
Flora PeyvandiAngelo Bianchi Bonomi, Hemophilia and Thrombosis Centre, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico.
Alexander GattMater Dei Hospital, Tal-QRoqq, Msida, Malta.
Samantha C GouwAmsterdam UMC location University of Amsterdam, Amsterdam, Netherlands.
Robert HollingsworthMedical Data Solutions and Services (MDSAS), Manchester, UK.
Thierry LambertReference Center for hemophilia and rare bleeding disorders, Hôpital Bicêtre, APHP, Université Paris Saclay. Le Kremlin Bicêtre, France.
Radek KaczmarekCoagulation Products Safety Supply and Access Committee, World Federation of Hemophilia, Montreal, Quebec, Canada.
Diana Carbonero AlvarezEuropean Association of Haemophilia and Associated Disorders, Brussels, Belgium.
Michael MakrisSchool of Medicine and Population Health, University of Sheffield, Sheffield, UK.
EUHASS participants

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Background: Information on inhibitor development in nonsevere hemophilia and its association with clotting factor concentrate type is limited. Objectives: To assess inhibitor development in patients with nonsevere hemophilia A (HA) and hemophilia B (HB) in the European Haemophilia Safety Surveillance system. Methods: Inhibitors and total treated patients are reported annually. Any exposure to concentrate per year was considered a treatment year. Incidence rates per 1000 treatment years and 95% CIs were calculated according to type of concentrate and compared using incidence rate ratios (IRRs). Results: During 2008 to 2023, 90 centers reported on 36,074 (HA) and 9238 (HB) treatment years. The inhibitor rate for nonsevere HA receiving factor (F)VIII was 4.2 per 1000 treatment years (95% CI, 3.5-4.9). Inhibitors developed at median 47.5 years (P25-P75 [IQR], 17.0-69.0), after median 40 exposure days (EDs; IQR, 17-80), with 58% occurring <50 EDs and 88% <100 EDs. Overall, 4 of 149 (2.7%) patients in the inhibitor group were female. Only one inhibitor was reported in nonsevere HB, in a female patient (FIX 7%, after 6 EDs), resulting in an inhibitor rate of 0.1 per 1000 treatment years (95% CI, 0.0-0.6). Compared with standard half-life recombinant FVIII, inhibitor rates on both plasma-derived FVIII (IRR, 0.27; 95% CI, 0.11-0.58; Conclusion: Inhibitors in nonsevere hemophilia occurred at a rate of 4.2 per 1000 treatment years in HA and 0.1 per 1000 treatment years in HB. Compared with standard half-life FVIII, inhibitor development on plasma-derived and extended half-life FVIII were reduced. These data show that inhibitor monitoring is relevant with nonsevere HA in both sexes and should be continued lifelong.

Indexed as

antibodiesfactor IXfactor VIIIhemophiliainhibitorneutralizing

Identifiers

PMID40547964
PMCPMC12178916

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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.