ArticleHaemophilia : the official journal of the World Federation of Hemophilia2021
The effect of emicizumab prophylaxis on long-term, self-reported physical health in persons with haemophilia A without factor VIII inhibitors in the HAVEN 3 and HAVEN 4 studies.
Article in Haemophilia : the official journal of the World Federation of Hemophilia, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 28 papers, 1 of them a synthesis that pooled it.
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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.
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Who cites it
28 citing papers in PubMed, 1 synthesis or guideline pooled it, 54 citations in OpenAlex.
- Non-clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B.The Cochrane database of systematic reviews · 2024Pooled it
- 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.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Trial
- Pain-Related Quality of Life Outcomes in People With Haemophilia A Receiving Emicizumab: A Post Hoc Analysis of the HAVEN 1, 3 and 4 and STASEY Studies.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Trial
- Effect of emicizumab prophylaxis on bone and joint health markers in people with haemophilia A without factor VIII inhibitors in the HAVEN 3 study.Haemophilia : the official journal of the World Federation of Hemophilia · 2022Trial
- A novel humanized FIX/FX hemophilia A mouse model for preclinical evaluation of FVIIIa-mimetic bispecific antibodies.Blood advances · 2026Article
- Association of Patient-Reported Outcomes with Hemophilia A Inhibitor Status and Treatment Product Type.Journal of clinical medicine · 2026Article
- Paediatric Subanalysis of TSUBASA, Assessing Physical Activity, Bleeding, Quality of Life and Safety in People with Haemophilia A Receiving Emicizumab.TH open : companion journal to thrombosis and haemostasis · 2026Article
- Twelve-month follow-up of a real-world, noninterventional study evaluating the impact of emicizumab on bleeding episodes, joint health, and quality of life in people with hemophilia A.Therapeutic advances in hematology · 2026Article
- Rebalancing agents in hemophilia: knowns, unknowns, and uncertainties.Haematologica · 2025Review
- Emicizumab prophylaxis in current haemophilia A care in the Czech Republic-data from the Czech National Haemophilia Programme Registry.Research and practice in thrombosis and haemostasis · 2025Article
- Emicizumab utilization, safety, and outcomes in people with severe hemophilia and no inhibitors: 3-year follow-up. A report from the UK Haemophilia Centre Doctors' Organisation.Research and practice in thrombosis and haemostasis · 2025Article
- Article
- Haemophilia Prophylaxis in the Age of Innovation: Exploring Opportunities for Personalized Treatment.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Review
- TSUBASA Study: Evaluating Association of Physical Activity and Bleeding Events in People With Haemophilia A Without Factor VIII Inhibitors Receiving Emicizumab.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Observational
- TSUBASA study: evaluation of the quality and content of daily life of people with hemophilia A without factor VIII inhibitors on prophylactic treatment with emicizumab.Research and practice in thrombosis and haemostasis · 2025Article
- Mental Health, Work Productivity, and Quality of Life in People with Severe Haemophilia A Receiving Prophylaxis: Findings from the CHESS Data Platform.TH open : companion journal to thrombosis and haemostasis · 2025Article
- Contemporary approaches to treat people with hemophilia: what's new and what's not?Research and practice in thrombosis and haemostasis · 2025Review
- A Prospective, Noninterventional Study to Evaluate the Impact of Emicizumab in the Management of Hemophilia A.Cureus · 2024Article
- Concizumab prophylaxis in persons with hemophilia A or B with inhibitors: patient-reported outcome results from the phase 3 explorer7 study.Research and practice in thrombosis and haemostasis · 2024Article
- Emicizumab is well tolerated and effective in people with congenital hemophilia A regardless of age, severity of disease, or inhibitor status: a scoping review.Research and practice in thrombosis and haemostasis · 2024Article
Corrections and comments
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Authors and funding
16 authors at 12 institutions in 8 countries.
Funding
Abstract
introductionSevere haemophilia A (HA) has a major impact on health-related quality of life (HRQoL).
aimAssess the impact of emicizumab on HRQoL in persons with severe HA (PwHA) without factor VIII (FVIII) inhibitors in the phase 3 HAVEN 3 and 4 studies.
methodsThis pooled analysis examines the HRQoL of PwHA aged ≥ 18 years treated with emicizumab prophylaxis via Haemophilia-Specific Quality of Life Questionnaire for Adults (Haem-A-QoL) and EuroQoL 5-Dimensions 5-levels (EQ-5D-5L). In particular, changes from baseline in Haem-A-QoL 'Physical Health' (PH) domain and 'Total Score' (TS) are evaluated.
resultsAmong 176 evaluable participants, 96 (55%) had received prior episodic treatment and 80 (45%) prophylaxis; 70% had ≥ 1 target joint and 51% had experienced ≥ 9 bleeds in the previous 24 weeks. Mean Haem-A-QoL PH and TS improved after emicizumab initiation. Mean (standard deviation) -12.0 (21.26)- and -8.6 (12.57)-point improvements were observed in PH and TS from baseline to Week 73; Week 73 scores were 27.9 (24.54) and 22.0 (14.38), respectively. Fifty-four percent of participants reported a clinically meaningful improvement in PH scores (≥ 10 points) by Week 73. Subgroups with poorer HRQoL prior to starting emicizumab (i.e. receiving episodic treatment, ≥ 9 bleeds, target joints) had the greatest improvements in PH scores, and corresponding reductions in missed workdays; change was not detected among those previously taking prophylaxis. No change over time was detected by the EQ-5D-5L questionnaire.
conclusionsEmicizumab prophylaxis in PwHA without FVIII inhibitors resulted in persistent and meaningful improvements in Haem-A-QoL PH and less work disruption than previous treatment.
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