Trial reportBlood2024
Emicizumab prophylaxis in infants with hemophilia A (HAVEN 7): primary analysis of a phase 3b open-label trial.
Trial report in Blood, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04431726 (A Phase IIIb, Multicenter, Open-Label, Single-Arm Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Emicizumab in Patients From Birth to 12 Months of Age With Hemophilia A Without Inhibitors), which is not on this map. Cited by 46 papers, 5 of them syntheses that pooled 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.
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.
A Phase IIIb, Multicenter, Open-Label, Single-Arm Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Emicizumab in Patients From Birth to 12 Months of Age With Hemophilia A Without Inhibitors
Who cites it
46 citing papers in PubMed, 5 syntheses or guidelines pooled it, 69 citations in OpenAlex.
- Updated Egyptian national guidelines for management of hemophilia A in children & adolescents.Annals of hematology · 2025Guideline
- [Chinese guidelines on the treatment of hemophilia (2025)].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2025Guideline
- Efanesoctocog Alfa Versus Emicizumab in Adolescent and Adult Patients With Haemophilia A Without Inhibitors.Advances in therapy · 2025Pooled it
- Outcomes of Emicizumab Treatment for Haemophilia A Paediatric Patients: A Systematic Review With Meta-Analysis.Haemophilia : the official journal of the World Federation of HemophiliaPooled it
- Outcomes of Emicizumab in Acquired Hemophilia Patients: A Systematic Review.Clinical and applied thrombosis/hemostasis : official journal of the International Academy of Clinical and Applied Thrombosis/HemostasisPooled 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
- Personalized Treatment of Hemophilia: Matching Therapies to Patient Needs in a Rapidly Evolving Landscape.Drugs · 2026Review
- Kunihiro Hattori, Tomoyuki Igawa, and Takehisa Kitazawa share Lasker Award honors for revolutionary hemophilia A therapy.The Journal of clinical investigation · 2026Article
- Long-term outcomes with emicizumab prophylaxis for haemophilia A in China: A multicentre, large-cohort retrospective study.British journal of haematology · 2026Observational
- Non-Factor Therapies in Haemophilia: The Era of Factor VIII Mimetics and Targeted Rebalancing Agents.Transfusion medicine and hemotherapy : offizielles Organ der Deutschen Gesellschaft fur Transfusionsmedizin und Immunhamatologie · 2026Review
- When and How to Start Prophylaxis in Children with Hemophilia.Transfusion medicine and hemotherapy : offizielles Organ der Deutschen Gesellschaft fur Transfusionsmedizin und Immunhamatologie · 2026Review
- FVIII-containing platelets modulate immune responses and attenuate inhibitor development in hemophilia A mice.Haematologica · 2026Article
- Article
- Hemophilia in Mexico: Updated Consensus Recommendations on Diagnosis, Treatment and Gene Therapy.Diseases (Basel, Switzerland) · 2026Review
- Maternal-fetal transfer of emicizumab in a pregnant mouse model.Blood advances · 2026Article
- Non-factor Therapies in Hemophilia: Mechanisms, Clinical Evidence, Patient Management, and Future Perspectives.Advances in therapy · 2026Review
- Myeloid Cell Function and Cytokine Profiles in Paediatric Haemophilia A: Insights From FVIII and Emicizumab Prophylaxis.Journal of cellular and molecular medicine · 2026Article
- [Evaluation of the efficacy and safety of reduced-dose emicizumab in infants with severe hemophilia A: a single-center real-world study].Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics · 2026Article
- Development of a novel hemophilia A mouse model to study emicizumab and factor VIII inhibitor risk.Blood advances · 2026Article
- Foods for special medical purposes for the dietary therapy of rare diseases: Current status and future prospects.Intractable & rare diseases research · 2026Review
Corrections and comments
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Authors and funding
18 authors at 13 institutions in 9 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
abstractSubcutaneous emicizumab enables prophylaxis for people with hemophilia A (HA) from birth, potentially reducing risk of bleeding and intracranial hemorrhage (ICH). HAVEN 7 (NCT04431726) is the first clinical trial of emicizumab dedicated to infants, designed to investigate the efficacy, safety, pharmacokinetics, and pharmacodynamics of emicizumab in those aged ≤12 months with severe HA without factor VIII (FVIII) inhibitors. Participants in this phase 3b trial received emicizumab 3 mg/kg maintenance dose every 2 weeks for 52 weeks and are continuing emicizumab during the 7-year long-term follow-up. Efficacy end points included annualized bleed rate (ABR): treated, all, treated spontaneous, and treated joint bleeds. Safety end points included adverse events (AEs), thromboembolic events (TEs), thrombotic microangiopathies (TMAs), and immunogenicity (anti-emicizumab antibodies [ADAs] and FVIII inhibitors). At primary analysis, 55 male participants had received emicizumab (median treatment duration: 100.3; range, 52-118 weeks). Median age at informed consent was 4.0 months (range, 9 days to 11 months 30 days). Model-based ABR for treated bleeds was 0.4 (95% confidence interval, 0.30-0.63), with 54.5% of participants (n = 30) having zero treated bleeds. No ICH occurred. All 42 treated bleeds in 25 participants (45.5%) were traumatic. Nine participants (16.4%) had ≥1 emicizumab-related AE (all grade 1 injection-site reactions). No AE led to treatment changes. No deaths, TEs, or TMAs occurred. No participant tested positive for ADAs. Two participants were confirmed positive for FVIII inhibitors. This primary analysis of HAVEN 7 indicates that emicizumab is efficacious and well tolerated in infants with severe HA without FVIII inhibitors.
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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.