Evidence map›Paper›PMID 40173026›Full record

ArticleHaemophilia : the official journal of the World Federation of Hemophilia2025

Trends in Treatment of Severe Haemophilia and Impact on Inhibitor Assessment by the EUHASS Registry.

Kathelijn Fischer, Riitta Lassila, Flora Peyvandi, Alex Gatt, Samantha C Gouw, Rob Hollingsworth, Thierry Lambert, Radoslaw Kaczmarek, Diana Carbonero, Michael Makris and 1 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Haemophilia : the official journal of the World Federation of Hemophilia, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.

0numbers the graph read from it
0cells of the map it votes in
5citing 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

5 citing papers in PubMed.

  1. Article
  2. Shaping hemophilia care: lessons and legacy of the SIPPET trial after 10 years.Research and practice in thrombosis and haemostasis · 2026
    Review
  3. Review
  4. Article
  5. The Swiss Haemophilia Registry-Report From the First 8 Years.Haemophilia : the official journal of the World Federation of Hemophilia
    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

11 authors.

Kathelijn FischerCenter for Benign Haematology, Thrombosis and Haemostasis, Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht, the Netherlands.ORCID https://orcid.org/0000-0001-7126-6613
Riitta LassilaDepartment of Hematology, Unit of Coagulation Disorders, Helsinki University Central Hospital, Research Program Unit in Systems Oncology, University of Helsinki, Helsinki, Finland.ORCID https://orcid.org/0000-0002-1911-3094
Flora PeyvandiAngelo Bianchi Bonomi, Hemophilia and Thrombosis centre, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, University of Milan, Milan, Italy.ORCID https://orcid.org/0000-0001-7423-9864
Alex GattMater Dei Hospital, Tal-QRoqq, Msida, Malta.
Samantha C GouwDepartment of Pediatric Hematology, Emma Children's Hospital, Amsterdam UMC location University of Amsterdam, Amsterdam, the Netherlands.
Rob HollingsworthMDSAS, Manchester, UK.
Thierry LambertCenter for Benign Haematology, Thrombosis and Haemostasis, Van Creveldkliniek, University Medical Center Utrecht, University Utrecht, Utrecht, the Netherlands.
Radoslaw KaczmarekCoagulation Products Safety Supply and Access Committee, World Federation of Hemophilia, Montreal, Quebec, Canada.ORCID https://orcid.org/0000-0001-8084-1958
Diana CarboneroEuropean Association of Haemophilia and Associated Disorders, Brussels, Belgium.ORCID https://orcid.org/0009-0005-9429-7558
Michael MakrisSchool of Medicine and Population Health, University of Sheffield, Sheffield, UK.ORCID https://orcid.org/0000-0001-7622-7939
EUHASS participants

Funding

BayerBiomarinBiotestBPLCSL BehringEuropean Association for Haemophilia and Allied DisordersEuropean Commission Health Programme through the Executive Agency for Health and Consumers (EAHC)GrifolsKedrionLFBNovoNordiskOctapharmaPfizerRocheSanofiSOBI/Biogen IdecTakeda (Baxter, Baxalta)
6 · The paper itself

Abstract

backgroundThe last 15 years have seen new extended half-life (EHL) recombinant FVIII/IX concentrates and nonreplacement therapy for haemophilia A (emicizumab) introduced in Europe. These changes affect FVIII/IX exposure in previously untreated patients (PUPs) and previously treated patients (PTPs) with severe haemophilia A and B (SHA and SHB) and may modify inhibitor development and/or detection.

aimTo report trends in treatment for severe haemophilia and concomitant changes in inhibitor incidence.

methodsBetween 2008 and 2022, 97 centres reported inhibitor development against FVIII/IX concentrates to the European Haemophilia Safety Surveillance System (EUHASS). Inhibitors were reported quarterly, and PUPs without inhibitor development annually. Cumulative inhibitor incidences (95% confidence intervals [CI]) were calculated for PUPs and incidence rates/1000 years (CI) for PTPs.

resultsBy 2022, SHA-PUPs (n = 1574) received emicizumab (44%), SHL-rFVIII (21.5%), pdFVIII (17.5%) and EHL-rFVIII (17%). SHB-PUPs (n = 236) received EHL-rFIX (79%) and SHL-rFIX (21%). SHA-PTPs (68,772 years) received EHL-rFVIII (31%), SHL-rFVIII (28%), emicizumab (25%), and pdFVIII (15%). SHB PTPs (11,185 years) received EHL-rFIX (69%), pdFIX (15%) and SHL-rFIX (15%). Observed Inhibitor incidence in SHA-PUPs decreased from 24% before 2016 to 6% in 2022 (p < 0.001), and potentially in SHB-PUPs too (from 9% to 3%; p = 0.066), but remained stable in SHA/SHB PTPs.

conclusionIn 2022, 44% of SHA-PUPs and 25% of SHA-PTPs received emicizumab prophylaxis. Concomitantly, observed inhibitor incidence reduced to 6% in SHA-PUPs. In SHB, EHL-rFIX treatment increased to 79% in SHB-PUPs and 69% in SHB-PTPs. Assessing inhibitor incidence for new concentrates is likely to be hampered by novel treatments causing delayed exposure to FVIII/FIX.

Indexed as

Blood Coagulation Factor InhibitorsHemophilia AAntibodies, BispecificAntibodies, Monoclonal, HumanizedChildEuropeFactor IXFactor VIIIHumansMaleRegistriesAntibodies, BispecificAntibodies, Monoclonal, HumanizedBlood Coagulation Factor InhibitorsemicizumabFactor IXFactor VIIIantibodiesemicizumabfactor VIIIhaemophiliainhibitorneutralisingPTPPUP

Identifiers

PMID40173026
PMCPMC12175111

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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.