Evidence map›Paper›PMID 40898745›Full record

Trial reportHaemophilia : the official journal of the World Federation of Hemophilia2025

Health-Related Quality of Life, Physical Activity and Joint Health in People With Severe Haemophilia A Receiving Emicizumab: Results From the Phase IV HemiNorth 2 Study.

Jan Astermark, Susanna Ranta, Linda Myrin-Westesson, Marianne Hoffmann, Sandrine Quere, Amparo Yovanna Castro Sanchez, Susan Robson, Tünde Czirok, Riitta Lassila, Pål André Holme

Abstract readClinical Trial, Phase IV
In one paragraph

Trial report 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 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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

1 citing paper in PubMed.

  1. Trial
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

10 authors.

Jan AstermarkDepartment of Translational Medicine, Lund University, Malmö, Sweden.ORCID https://orcid.org/0000-0001-8500-2483
Susanna RantaAstrid Lindgren's Children's Hospital, Karolinska University Hospital, Stockholm, Sweden.
Linda Myrin-WestessonRegion Västra Götaland, Department of Medicine, Sahlgrenska University Hospital, Gothenburg, Sweden.
Marianne HoffmannDepartment of Paediatrics and Adolescent Medicine, University Hospital Rigshospitalet, Copenhagen, Denmark.
Sandrine QuereF. Hoffmann-La Roche Ltd, Basel, Switzerland.
Amparo Yovanna Castro SanchezData Sciences, F. Hoffmann-La Roche Ltd, Basel, Switzerland.
Susan RobsonF. Hoffmann-La Roche Ltd, Basel, Switzerland.
Tünde CzirokF. Hoffmann-La Roche Ltd, Basel, Switzerland.
Riitta LassilaDepartment of Haematology, Coagulation Disorders Unit, and Research Program Unit in Systems Oncology Oncosys, Medical Faculty, University of Helsinki, Helsinki, Finland.ORCID https://orcid.org/0000-0002-1911-3094
Pål André HolmeDepartment of Haematology, Oslo University Hospital, Oslo, Norway.

Funding

F. Hoffmann-La Roche Ltd
6 · The paper itself

Abstract

introductionDespite factor (F)VIII prophylaxis, a perceived increased risk of bleeding for some people with severe haemophilia A (PwSHA) exists, limiting physical activity (PA) and restricting quality of life (QoL).

aimHemiNorth 2 (EudraCT# 2020-003256-32) is an interventional study evaluating the impact of switching from FVIII prophylaxis to emicizumab in PwSHA without FVIII inhibitors who have a need for improved prophylaxis in the Nordic countries.

methodsFollowing completion of the HemiNorth non-interventional study (NIS), eligible participants (aged ≥ 12-61 years) were enrolled in HemiNorth 2. The primary endpoint was health-related QoL via the Comprehensive Assessment Tool for Challenges in Hemophilia (CATCH). Secondary endpoints included PA (International Physical Activity Questionnaire-Short Form [IPAQ-SF]), treatment preference (Emicizumab Preference [EmiPref] survey), joint health, model-based annualised bleeding rates (ABRs) and adverse events.

resultsOverall, 28 physically active male PwSHA were enrolled. Most baseline CATCH domains were ≤ 25 and remained consistent; mean treatment burden considerably improved from baseline for adults (-17.8) and adolescents (+16.7). IPAQ-SF scores were consistent throughout the study. Overall, 23 of 25 (92.0%) EmiPref respondents preferred emicizumab over FVIII prophylaxis. Model-based ABRs for treated bleeds decreased from 5.9 (95% confidence interval [CI]: 3.8-9.1) to 1.6 (95% CI: 0.9-3.0) from the NIS to HemiNorth 2, and participants with zero treated bleeds increased from 8 (28.6%) to 16 (57.1%). No new safety signals were reported.

conclusionsEmicizumab improved treatment burden and was preferred by most participants over FVIII prophylaxis. PA levels were consistently high, and bleeding rates improved with emicizumab versus prior FVIII prophylaxis.

Indexed as

Antibodies, BispecificAntibodies, Monoclonal, HumanizedExerciseHemophilia AQuality of LifeAdolescentAdultChildHemorrhageHumansMaleMiddle AgedYoung AdultAntibodies, BispecificAntibodies, Monoclonal, Humanizedemicizumabemicizumabexercisefactor VIIIhaemophilia Aphysical activity

Identifiers

PMID40898745
PMCPMC12612379

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
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.