Evidence map›Paper›PMID 40289300›Full record

ArticleEuropean journal of haematology2025

Immune Tolerance Induction With a Recombinant Factor VIII Fc in Haemophilia A: Data From a Chart Review Study.

Robert Klamroth, Mahasen Al Saleh, Heidi Glosli, Michele Schiavulli, Benoît Guillet, Linda Bystrická, Anton Schönstein, Stefan Lethagen

Erratum issued Registry-linked trialAbstract readMulticenter Study
PubMed Full text at PMC Publisher
In one paragraph

Article in European journal of haematology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. It is linked to trial NCT03951103 (A Chart Review Study of Patients With Haemophilia A With Inhibitors Treated With rFVIIIFc), which is not on this map. Not yet cited in PubMed.

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

NCT03951103 completednot on this map

A Chart Review Study of Patients With Haemophilia A With Inhibitors Treated With rFVIIIFc (Elocta®) for Immune Tolerance Induction

TypeobservationalSponsorSwedish Orphan BiovitrumRan2018 to 2022Enrolled44ConditionsHemophilia A With InhibitorArmsrFVIIIFc
3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

8 authors.

Robert KlamrothVivantes Klinikum Friedrichshain, Berlin, Germany.ORCID https://orcid.org/0000-0003-4194-8183
Mahasen Al SalehKing Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia.ORCID https://orcid.org/0009-0007-3207-5698
Heidi GlosliCentre for Rare Disorders, Oslo University Hospital, Oslo, Norway.ORCID https://orcid.org/0000-0003-1153-4131
Michele SchiavulliHaemophilia and Congenital Bleeding Disorders, Treatment Center Santobono-Pausilipon Children Hospital, AORN, Naples, Italy.ORCID https://orcid.org/0000-0003-0384-7795
Benoît GuilletCentre for Bleeding Disorders, Univ Rennes, CHU Rennes, Inserm, EHESP, Irset (Institut de recherche en santé, environnement et travail), Rennes, France.ORCID https://orcid.org/0000-0003-2938-8013
Linda BystrickáSobi, Stockholm, Sweden.ORCID https://orcid.org/0000-0002-9787-7866
Anton SchönsteinVeramed, Frankfurt am Main, Germany.ORCID https://orcid.org/0000-0001-8683-5452
Stefan LethagenSobi, Stockholm, Sweden.ORCID https://orcid.org/0000-0002-8436-1780

Funding

Swedish Orphan Biovitrum
6 · The paper itself

Abstract

objectiveTo report data from an ITI chart review study (NCT03951103) for first-time and rescue ITI with recombinant factor VIII Fc fusion protein (rFVIIIFc) in persons with haemophilia A.

methodsRetrospective and prospective real-world data are reported from a non-interventional, multicentre study of patients who had been or were currently being treated with rFVIIIFc ITI. ITI treatment outcome (defined by investigators) and regimens are reported.

resultsForty-one patients from 16 sites were included. First-time ITI was used in 24 patients; 16 had an ITI outcome at study end. Thirteen patients (81.3%) had ITI success, and three had failure. Median (range) rFVIIIFc consumption was 300 (61-2800) IU/kg/week, and most (70.8%) used ≤ 300 IU/kg/week. The vast majority of patients (87.5%) received less than daily ITI. Rescue ITI was used in 17 patients; 16 had an ITI outcome at study end. Eight patients (50.0%) had ITI success/partial success, seven had failure, and one withdrew early. Median (range) rFVIIIFc consumption was 536 (98-1435) IU/kg/week; 35.3% used ≤ 300 IU/kg/week and 52.9% used > 500 IU/kg/week. Most patients (64.7%) received daily ITI.

conclusionITI with rFVIIIFc is likely to be successful in first-time ITI patients and is an effective option for those who have previously experienced ITI failure.

trial registrationClinicalTrials.gov identifier: NCT03951103.

Indexed as

Factor VIIIHemophilia AImmune ToleranceImmunoglobulin Fc FragmentsRecombinant Fusion ProteinsAdolescentAdultAgedChildChild, PreschoolFemaleHumansMaleMiddle AgedProspective StudiesRetrospective StudiesFactor VIIIfactor VIII-Fc fusion proteinImmunoglobulin Fc FragmentsRecombinant Fusion Proteinsfactor VIIIhaemophilia Aimmune tolerancequality of liferecombinant fusion proteintreatment outcome

Identifiers

PMID40289300
PMCPMC12224564

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