Evidence map›Paper›PMID 37351812›Full record

ArticleAdvances in therapy2023

Real-World Effectiveness of rFIXFc Prophylaxis in Patients with Haemophilia B Switched from Standard Half-Life Therapy in Three European Countries.

Eva Funding, Gillian Lowe, Lone H Poulsen, Susan Shapiro, Johannes Oldenburg, Daniel Eriksson, Aletta Falk, Carly Rich

Open access · hybridAbstract read
In one paragraph

Article in Advances in therapy, 2023. 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
2.9field-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

5 citing papers in PubMed, 10 citations in OpenAlex.

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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

8 authors at 7 institutions in 4 countries.

Eva FundingDepartment of Hematology, University Hospital Rigshospitalet, Copenhagen, Denmark.
Gillian LoweWest Midlands Adult Comprehensive Care Haemophilia Centre, University Hospitals Birmingham, Birmingham, UK.ORCID http://orcid.org/0000-0003-2527-9557
Lone H PoulsenThe Haemophilia Centre, Aarhus University Hospital, Aarhus, Denmark.
Susan ShapiroOxford Haemophilia and Thrombosis Centre, NIHR Oxford Biomedical Research Centre, Oxford University Hospitals NHS Foundation Trust, Oxford, UK.ORCID http://orcid.org/0000-0003-0402-0802
Johannes OldenburgInstitute for Experimental Haematology and Transfusion Medicine, University Hospital Bonn, Bonn, Germany.
Daniel ErikssonSobi, Stockholm, Sweden.ORCID http://orcid.org/0000-0001-7699-242X
Aletta FalkSobi, Stockholm, Sweden.
Carly RichSobi, Suite 2, Riverside 3, Granta Park, Great Abington, Cambridgeshire, CB21 6AD, UK. Carly.Rich@sobi.com.ORCID http://orcid.org/0000-0003-4717-5855
Swedish Orphan Biovitrum (Sweden) · SEAarhus University Hospital · DKGranta Design (United Kingdom) · GBNIHR Surgical Reconstruction and Microbiology Research Centre · GBUniversity Hospital Bonn · DEUniversity of Copenhagen · DKUniversity of Oxford · GB

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionThe current study describes real-world clinical outcomes and factor usage among patients with haemophilia B switching from standard half-life factor IX (SHL FIX) treatment to recombinant factor IX Fc fusion protein (rFIXFc) prophylaxis in European treatment centres.

methodsThis non-interventional, retrospective, multicentre chart review evaluated medical records from adult and paediatric patients with haemophilia B in Denmark, Germany and the UK. Patients had documented SHL FIX treatment, on-demand or prophylaxis, for ≥ 6 months before starting rFIXFc prophylaxis, and subsequent data for ≥ 6 months afterwards (up to 24 months). Primary endpoints included annualised bleeding rates (ABRs), prophylactic factor consumption and injection frequency.

resultsData from 30 patients (24/30 [80.0%] with severe disease) showed overall mean (standard deviation, SD) ABRs of 4.7 (6.3) on SHL FIX treatment and 1.7 (2.3) after switching to rFIXFc prophylaxis. The reduction in mean (SD) ABRs was greater when switching from SHL FIX on-demand treatment (n = 6), with a decrease from 10.5 (9.9) to 2.6 (4.5), than when switching from SHL FIX prophylaxis (n = 24), with a decrease from 3.3 (4.3) to 1.5 (1.4). Among prior SHL FIX prophylaxis patients, switching to rFIXFc prophylaxis increased the proportion of those with zero bleeds from 21.7% to 45.8% during the 6 months before and after switching, respectively. In the total population, five of six target joints (83.3%) present when patients started rFIXFc prophylaxis subsequently resolved. In patients switching from SHL FIX prophylaxis to rFIXFc prophylaxis, mean (SD) weekly injection frequency was reduced by 1.0 (0.7) and mean (SD) factor consumption was reduced by 27.7 (49.6) IU/kg/week.

conclusionThis study demonstrates the effectiveness of rFIXFc prophylaxis in real-world clinical practice. Improvements in both clinical effectiveness and factor usage associated with rFIXFc prophylaxis may potentially reduce patient burden and improve quality of life.

Indexed as

Hemophilia BAdultChildHalf-LifeHemorrhageHumansQuality of LifeRecombinant Fusion ProteinsRetrospective StudiesRecombinant Fusion ProteinsAnnualised bleeding rateFactor consumptionFactor IXHaemophilia BInjection frequencyProphylaxisReal-worldRecombinant factor IX Fc fusion proteinStandard half-life therapy

Identifiers

PMID37351812
PMCPMC10427542
OpenAlexW4381715690

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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.